Trial Outcomes & Findings for JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure (NCT NCT05998408)
NCT ID: NCT05998408
Last Updated: 2026-08-18
Results Overview
Numbers of participants who complete a full course of ruxolitinib without discontinuation due to hematologic toxicity in the 6 months following treatment initiation. Discontinuation due to hematologic toxicity is defined as those participants that remain off drug for 6 consecutive weeks due to ongoing hematologic toxicity. Hematologic toxicity for this study will be defined as follows: * Greater than 50% increase in transfusion needs in participants who were transfusion dependent prior to ruxolitinib therapy, lasting for more than 12 weeks * Need for any transfusion for more than 12 weeks in participants who were transfusion independent prior to ruxolitinib therapy. This excludes transfusions given for Hb \>7g/dL or platelets \>10 x 109 or those given for procedures. * Worsening in peripheral cytopenias \>50% compared to pre-treatment levels in participants with a pre-treatment ANC \>500 or platelets \>50 Drop in ANC to \<200 in participants with a pre-treatment ANC \<500
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE1/PHASE2
Target enrollment
13 participants
Primary outcome timeframe
6 months
Results posted on
2026-08-18
Participant Flow
Participants who completed Month 6 of the clinical trial and achieved a predefined response will be eligible for enrollment in extension phase.
Participant milestones
| Measure |
Cohort 1: Participants With Severe Aplastic Anemia (SAA)
Participants diagnosed with Severe Aplastic Anemia (SAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 2: Participants With Moderate Aplastic Anemia (MAA)
Participants diagnosed with Moderate Aplastic Anemia (MAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 3: Participants With Unilineage Bone Marrow Failure Disorder
Participants diagnosed with Unilineage Bone Marrow Failure Disorder will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 4: Participants With T-cell Large Granular Lymphocyte (T-LGL) Leukemia
Participants diagnosed with T-cell large granular lymphocyte (T-LGL) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 5: Participants With Hypoplastic Myelodysplastic Syndrome (hMDS)
Participants diagnosed with hypoplastic myelodysplastic syndrome (hMDS) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
|---|---|---|---|---|---|
|
Day 0 to Month 6
STARTED
|
5
|
0
|
1
|
7
|
0
|
|
Day 0 to Month 6
Population for Month 6 Response Assessment
|
1
|
0
|
0
|
6
|
0
|
|
Day 0 to Month 6
COMPLETED
|
1
|
0
|
0
|
6
|
0
|
|
Day 0 to Month 6
NOT COMPLETED
|
4
|
0
|
1
|
1
|
0
|
|
Extension Phase: Month 6 to Year 3
STARTED
|
0
|
0
|
0
|
1
|
0
|
|
Extension Phase: Month 6 to Year 3
COMPLETED
|
0
|
0
|
0
|
1
|
0
|
|
Extension Phase: Month 6 to Year 3
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
| Measure |
Cohort 1: Participants With Severe Aplastic Anemia (SAA)
Participants diagnosed with Severe Aplastic Anemia (SAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 2: Participants With Moderate Aplastic Anemia (MAA)
Participants diagnosed with Moderate Aplastic Anemia (MAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 3: Participants With Unilineage Bone Marrow Failure Disorder
Participants diagnosed with Unilineage Bone Marrow Failure Disorder will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 4: Participants With T-cell Large Granular Lymphocyte (T-LGL) Leukemia
Participants diagnosed with T-cell large granular lymphocyte (T-LGL) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 5: Participants With Hypoplastic Myelodysplastic Syndrome (hMDS)
Participants diagnosed with hypoplastic myelodysplastic syndrome (hMDS) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
|---|---|---|---|---|---|
|
Day 0 to Month 6
Adverse Event
|
1
|
0
|
1
|
0
|
0
|
|
Day 0 to Month 6
Requires Alternate Disease Treatment
|
1
|
0
|
0
|
0
|
0
|
|
Day 0 to Month 6
Withdrawal by Subject
|
2
|
0
|
0
|
1
|
0
|
Baseline Characteristics
JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure
Baseline characteristics by cohort
| Measure |
Cohort 1: Participants With Severe Aplastic Anemia (SAA)
n=5 Participants
Participants diagnosed with Severe Aplastic Anemia (SAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 2: Participants With Moderate Aplastic Anemia (MAA)
Participants diagnosed with Moderate Aplastic Anemia (MAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 3: Participants With Pure Red Cell Aplasia (PRCA)
n=1 Participants
Participants diagnosed with Pure Red Cell Aplasia (PRCA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 4: Participants With T-cell Large Granular Lymphocyte (T-LGL) Leukemia
n=7 Participants
Participants diagnosed with T-cell large granular lymphocyte (T-LGL) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 5: Participants With Hypoplastic Myelodysplastic Syndrome (hMDS)
Participants diagnosed with hypoplastic myelodysplastic syndrome (hMDS) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Total
n=13 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=298 Participants
|
—
|
0 Participants
n=400 Participants
|
0 Participants
n=30 Participants
|
—
|
0 Participants
n=44 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=298 Participants
|
—
|
0 Participants
n=400 Participants
|
3 Participants
n=30 Participants
|
—
|
4 Participants
n=44 Participants
|
|
Age, Categorical
>=65 years
|
4 Participants
n=298 Participants
|
—
|
1 Participants
n=400 Participants
|
4 Participants
n=30 Participants
|
—
|
9 Participants
n=44 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=298 Participants
|
—
|
0 Participants
n=400 Participants
|
4 Participants
n=30 Participants
|
—
|
7 Participants
n=44 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=298 Participants
|
—
|
1 Participants
n=400 Participants
|
3 Participants
n=30 Participants
|
—
|
6 Participants
n=44 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=298 Participants
|
—
|
0 Participants
n=400 Participants
|
0 Participants
n=30 Participants
|
—
|
0 Participants
n=44 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=298 Participants
|
—
|
1 Participants
n=400 Participants
|
7 Participants
n=30 Participants
|
—
|
12 Participants
n=44 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=298 Participants
|
—
|
0 Participants
n=400 Participants
|
0 Participants
n=30 Participants
|
—
|
1 Participants
n=44 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=298 Participants
|
—
|
0 Participants
n=400 Participants
|
0 Participants
n=30 Participants
|
—
|
0 Participants
n=44 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=298 Participants
|
—
|
0 Participants
n=400 Participants
|
0 Participants
n=30 Participants
|
—
|
0 Participants
n=44 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=298 Participants
|
—
|
0 Participants
n=400 Participants
|
0 Participants
n=30 Participants
|
—
|
0 Participants
n=44 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=298 Participants
|
—
|
0 Participants
n=400 Participants
|
0 Participants
n=30 Participants
|
—
|
0 Participants
n=44 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=298 Participants
|
—
|
1 Participants
n=400 Participants
|
7 Participants
n=30 Participants
|
—
|
12 Participants
n=44 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=298 Participants
|
—
|
0 Participants
n=400 Participants
|
0 Participants
n=30 Participants
|
—
|
0 Participants
n=44 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=298 Participants
|
—
|
0 Participants
n=400 Participants
|
0 Participants
n=30 Participants
|
—
|
1 Participants
n=44 Participants
|
|
Region of Enrollment
United States
|
5 participants
n=298 Participants
|
—
|
1 participants
n=400 Participants
|
7 participants
n=30 Participants
|
—
|
13 participants
n=44 Participants
|
PRIMARY outcome
Timeframe: 6 monthsPopulation: No participants were enrolled in Cohort 2 and Cohort 5
Numbers of participants who complete a full course of ruxolitinib without discontinuation due to hematologic toxicity in the 6 months following treatment initiation. Discontinuation due to hematologic toxicity is defined as those participants that remain off drug for 6 consecutive weeks due to ongoing hematologic toxicity. Hematologic toxicity for this study will be defined as follows: * Greater than 50% increase in transfusion needs in participants who were transfusion dependent prior to ruxolitinib therapy, lasting for more than 12 weeks * Need for any transfusion for more than 12 weeks in participants who were transfusion independent prior to ruxolitinib therapy. This excludes transfusions given for Hb \>7g/dL or platelets \>10 x 109 or those given for procedures. * Worsening in peripheral cytopenias \>50% compared to pre-treatment levels in participants with a pre-treatment ANC \>500 or platelets \>50 Drop in ANC to \<200 in participants with a pre-treatment ANC \<500
Outcome measures
| Measure |
Cohort 1: Participants With Severe Aplastic Anemia (SAA)
n=5 Participants
Participants diagnosed with Severe Aplastic Anemia (SAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 2: Participants With Moderate Aplastic Anemia (MAA)
Participants diagnosed with Moderate Aplastic Anemia (MAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 3: Participants With Unilineage Bone Marrow Failure Disorder
n=1 Participants
Participants diagnosed with Unilineage Bone Marrow Failure Disorder will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 4: Participants With T-cell Large Granular Lymphocyte (T-LGL) Leukemia
n=7 Participants
Participants diagnosed with T-cell large granular lymphocyte (T-LGL) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 5: Participants With Hypoplastic Myelodysplastic Syndrome (hMDS)
Participants diagnosed with hypoplastic myelodysplastic syndrome (hMDS) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
|---|---|---|---|---|---|
|
Number of Participants Who Completed a Full Course of Ruxolitinib Without Discontinuation Due to Hematologic Toxicity
Discontinued for other reasons than hematologic toxicity
|
3 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Who Completed a Full Course of Ruxolitinib Without Discontinuation Due to Hematologic Toxicity
Completed full course
|
1 Participants
|
0 Participants
|
0 Participants
|
6 Participants
|
0 Participants
|
|
Number of Participants Who Completed a Full Course of Ruxolitinib Without Discontinuation Due to Hematologic Toxicity
Discontinued for hematologic toxicity
|
1 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: 6 monthsPopulation: No participants were enrolled in Cohort 2 and Cohort 5
Participants who had a CR at 3 months and discontinued study drug were considered responders, even if they subsequently relapsed. • Cohort 1: Response: No Camitta SAA criteria; ≥2 of ANC ≥0.5 × 10⁹/L, platelets ≥20 × 10⁹/L, reticulocytes ≥60 × 10⁹/L on 2 counts ≥1 week apart Complete Response (CR): ANC ≥1 × 10⁹/L, platelets ≥100 × 10⁹/L, Hb ≥10 g/dL Partial Response (PR): Response but not CR • Cohorts 3: Response: ≥1 evaluable lineage response Erythroid: Hb ↑ \>1.5 g/dL, ≥4 fewer RBC transfusions/8 weeks, or reticulocytes \>60 × 10⁹/L Platelet: ↑ ≥30 × 10⁹/L if baseline ≥20 × 10⁹/L, or \<20 to \>20 × 10⁹/L and ≥100% ↑ Neutrophil: ≥100% ↑ and absolute ↑ \>0.5 × 10⁹/L CR: ANC ≥1 × 10⁹/L, platelets ≥100 × 10⁹/L, Hb ≥10 g/dL • Cohort 4: Response: ≥1 evaluable lineage response CHR: ANC \>1.5 × 10⁹/L, platelets \>150 × 10⁹/L, lymphocytes \<4 × 10⁹/L PHR: Improvement in ≥1 affected parameter but not CHR CMR: No clonal T-cell detection and CHR CR: CHR and CMR
Outcome measures
| Measure |
Cohort 1: Participants With Severe Aplastic Anemia (SAA)
n=5 Participants
Participants diagnosed with Severe Aplastic Anemia (SAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 2: Participants With Moderate Aplastic Anemia (MAA)
Participants diagnosed with Moderate Aplastic Anemia (MAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 3: Participants With Unilineage Bone Marrow Failure Disorder
n=1 Participants
Participants diagnosed with Unilineage Bone Marrow Failure Disorder will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 4: Participants With T-cell Large Granular Lymphocyte (T-LGL) Leukemia
n=7 Participants
Participants diagnosed with T-cell large granular lymphocyte (T-LGL) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 5: Participants With Hypoplastic Myelodysplastic Syndrome (hMDS)
Participants diagnosed with hypoplastic myelodysplastic syndrome (hMDS) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
|---|---|---|---|---|---|
|
Number of Participants Who Achieved an Overall Response
No Response
|
5 Participants
|
0 Participants
|
1 Participants
|
6 Participants
|
0 Participants
|
|
Number of Participants Who Achieved an Overall Response
Complete Response
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Who Achieved an Overall Response
Partial Response
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 3, 12 months, and yearly thereafterOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 3, 6 monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: VariableOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: VariableOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: VariableOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: VariableOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: VariableOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: VariableOutcome measures
Outcome data not reported
Adverse Events
Cohort 1: Participants With Severe Aplastic Anemia (SAA)
Serious events: 5 serious events
Other events: 2 other events
Deaths: 0 deaths
Cohort 2: Participants With Moderate Aplastic Anemia (MAA)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Cohort 3: Participants With Pure Red Cell Aplasia (PRCA)
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Cohort 4: Participants With T-cell Large Granular Lymphocyte (T-LGL) Leukemia
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
Cohort 5: Participants With Hypoplastic Myelodysplastic Syndrome (hMDS)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1: Participants With Severe Aplastic Anemia (SAA)
n=5 participants at risk
Participants diagnosed with Severe Aplastic Anemia (SAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 2: Participants With Moderate Aplastic Anemia (MAA)
Participants diagnosed with Moderate Aplastic Anemia (MAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 3: Participants With Pure Red Cell Aplasia (PRCA)
n=1 participants at risk
Participants diagnosed with Pure Red Cell Aplasia (PRCA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 4: Participants With T-cell Large Granular Lymphocyte (T-LGL) Leukemia
n=7 participants at risk
Participants diagnosed with T-cell large granular lymphocyte (T-LGL) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 5: Participants With Hypoplastic Myelodysplastic Syndrome (hMDS)
Participants diagnosed with hypoplastic myelodysplastic syndrome (hMDS) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
|---|---|---|---|---|---|
|
Blood and lymphatic system disorders
Anemia
|
20.0%
1/5 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/7 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Gastrointestinal disorders
Lower gastrointestinal hemorrhage
|
0.00%
0/5 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
14.3%
1/7 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Gastrointestinal disorders
Oral hemorrhage
|
20.0%
1/5 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/7 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Infections and infestations
Lung infection
|
20.0%
1/5 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/7 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
20.0%
1/5 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/7 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Renal and urinary disorders
Chronic kidney disease
|
20.0%
1/5 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/7 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Reproductive system and breast disorders
Vaginal hemorrhage
|
20.0%
1/5 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/7 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
20.0%
1/5 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/7 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
20.0%
1/5 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/7 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
General disorders
Fever
|
0.00%
0/5 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
100.0%
1/1 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/7 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
0.00%
0/5 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
100.0%
1/1 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/7 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
General disorders
Fatigue
|
0.00%
0/5 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
14.3%
1/7 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Infections and infestations
Upper respiratory infection
|
0.00%
0/5 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
14.3%
1/7 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/5 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
14.3%
1/7 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Injury, poisoning and procedural complications
Fracture
|
0.00%
0/5 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
28.6%
2/7 • Number of events 2 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Nervous system disorders
Syncope
|
0.00%
0/5 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
14.3%
1/7 • Number of events 2 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Vascular disorders
Hematoma
|
0.00%
0/5 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
14.3%
1/7 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
Other adverse events
| Measure |
Cohort 1: Participants With Severe Aplastic Anemia (SAA)
n=5 participants at risk
Participants diagnosed with Severe Aplastic Anemia (SAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 2: Participants With Moderate Aplastic Anemia (MAA)
Participants diagnosed with Moderate Aplastic Anemia (MAA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 3: Participants With Pure Red Cell Aplasia (PRCA)
n=1 participants at risk
Participants diagnosed with Pure Red Cell Aplasia (PRCA) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 4: Participants With T-cell Large Granular Lymphocyte (T-LGL) Leukemia
n=7 participants at risk
Participants diagnosed with T-cell large granular lymphocyte (T-LGL) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
Cohort 5: Participants With Hypoplastic Myelodysplastic Syndrome (hMDS)
Participants diagnosed with hypoplastic myelodysplastic syndrome (hMDS) will be administered ruxolitinib 5 mg by mouth twice daily. The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
Ruxolitinib: Participants will be instructed to take ruxolitinib at up to 20mg (total) BID for up to 6 months (with or without food). The dose will be increased weekly in 5 mg twice-daily increments, up to a maximum dose of 20 mg by mouth twice daily.
|
|---|---|---|---|---|---|
|
Blood and lymphatic system disorders
Blood blisters on tongue and bleeding from gums
|
20.0%
1/5 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/7 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Infections and infestations
Skin infection
|
20.0%
1/5 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/7 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin lesion
|
20.0%
1/5 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/7 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Gastrointestinal disorders
Colitis
|
0.00%
0/5 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
100.0%
1/1 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/7 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Infections and infestations
Eye infection
|
0.00%
0/5 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
14.3%
1/7 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Infections and infestations
Lung infection
|
0.00%
0/5 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
28.6%
2/7 • Number of events 2 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Infections and infestations
Upper respiratory infection
|
0.00%
0/5 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
14.3%
1/7 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
|
Musculoskeletal and connective tissue disorders
Muscle cramp
|
0.00%
0/5 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
0.00%
0/1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
14.3%
1/7 • Number of events 1 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
—
0/0 • Up to 6 months
Adverse events included all Grade 2 or higher AEs, including local and systemic reactions not meeting SAE criteria, and all SAEs, regardless of relationship to study intervention. Clinically significant non-hematologic laboratory abnormalities included Grade 3 or 4 toxicities or abnormalities requiring intervention or study discontinuation. Events were identified through study visits, participant interviews, medical care encounters, monitor review, and safety laboratory assessments.
|
Additional Information
Emma M. Groarke, M.D.
National Heart, Lung, and Blood Institute
Phone: 301.451.7129
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place