Trial Outcomes & Findings for Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function (NCT NCT06145282)
NCT ID: NCT06145282
Last Updated: 2026-04-29
Results Overview
Number of participants at 1 year post stem cell transplant who have not experienced graft failure and who are without severe graft-versus-host disease (defined as grade 3 and higher acute GVHD). Graft failure is defined as the absence of or insufficient donor chimerism associated with the return of acute complications of sickle cell disease. As defined by CIMBTR criteria for Organ Stages of Acute GVHD: Grade III: Skin = Rash on \>50% of body surface; Liver = Total Bilirubin 6.1 - 15.0 mg/dL; Lower GI = Diarrhea \> 1500 mL/day. Grade IV: Skin = Generalized erythroderma plus bullous formation; Liver = Total Bilirubin \>15 mg/dL; Lower GI = Severe abdominal pain with or without ileus. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE1/PHASE2
Target enrollment
6 participants
Primary outcome timeframe
Up to 1 year
Results posted on
2026-04-29
Participant Flow
Participant milestones
| Measure |
PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
Stem cell recipients with Sickle Cell Disease will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. This group will receive a total of two doses of PT-Cy; 50mg/kg, on days +3 and +4 (total 100 mg/kg) post-HCT
|
Human Leukocyte Antigens (HLA) Haploidentical Related Stem Cell Donor
A haploidentical relative donor will receive filgrastim (G-CSF) 10 to 16 µg/kg/d subcutaneously or intravenously for up to 6 days with apheresis collections of peripheral blood hematopoietic progenitor cells (PBPC) after the 5th day (and after the 6th day if required).
|
PBSC Transplant With 50 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
Stem cell recipients with Sickle Cell Disease will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. This group will receive a single dose of PT-Cy; 50 mg/kg, on day +3 post-HCT
|
|---|---|---|---|
|
Overall Study
STARTED
|
0
|
3
|
3
|
|
Overall Study
COMPLETED
|
0
|
3
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
1
|
Reasons for withdrawal
| Measure |
PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
Stem cell recipients with Sickle Cell Disease will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. This group will receive a total of two doses of PT-Cy; 50mg/kg, on days +3 and +4 (total 100 mg/kg) post-HCT
|
Human Leukocyte Antigens (HLA) Haploidentical Related Stem Cell Donor
A haploidentical relative donor will receive filgrastim (G-CSF) 10 to 16 µg/kg/d subcutaneously or intravenously for up to 6 days with apheresis collections of peripheral blood hematopoietic progenitor cells (PBPC) after the 5th day (and after the 6th day if required).
|
PBSC Transplant With 50 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
Stem cell recipients with Sickle Cell Disease will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. This group will receive a single dose of PT-Cy; 50 mg/kg, on day +3 post-HCT
|
|---|---|---|---|
|
Overall Study
Death
|
0
|
0
|
1
|
Baseline Characteristics
Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function
Baseline characteristics by cohort
| Measure |
PBSC Transplant With 50 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
n=3 Participants
Stem cell recipients with Sickle Cell Disease will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. This group will receive a single dose of PT-Cy; 50 mg/kg, on day +3 post-HCT
|
PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
Stem cell recipients with Sickle Cell Disease will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. This group will receive a total of two doses of PT-Cy; 50mg/kg, on days +3 and +4 (total 100 mg/kg) post-HCT
|
Human Leukocyte Antigens (HLA) Haploidentical Related Stem Cell Donor
n=3 Participants
A haploidentical relative donor will receive filgrastim (G-CSF) 10 to 16 µg/kg/d subcutaneously or intravenously for up to 6 days with apheresis collections of peripheral blood hematopoietic progenitor cells (PBPC) after the 5th day (and after the 6th day if required).
|
Total
n=6 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Region of Enrollment
United States
|
3 participants
n=9 Participants
|
—
|
3 participants
n=23 Participants
|
6 participants
n=73 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=9 Participants
|
—
|
1 Participants
n=23 Participants
|
1 Participants
n=73 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=9 Participants
|
—
|
2 Participants
n=23 Participants
|
5 Participants
n=73 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=9 Participants
|
—
|
0 Participants
n=23 Participants
|
0 Participants
n=73 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=9 Participants
|
—
|
2 Participants
n=23 Participants
|
4 Participants
n=73 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=9 Participants
|
—
|
1 Participants
n=23 Participants
|
2 Participants
n=73 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=9 Participants
|
—
|
0 Participants
n=23 Participants
|
0 Participants
n=73 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=9 Participants
|
—
|
3 Participants
n=23 Participants
|
6 Participants
n=73 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
—
|
0 Participants
n=23 Participants
|
0 Participants
n=73 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=9 Participants
|
—
|
0 Participants
n=23 Participants
|
0 Participants
n=73 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=9 Participants
|
—
|
0 Participants
n=23 Participants
|
0 Participants
n=73 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=9 Participants
|
—
|
0 Participants
n=23 Participants
|
0 Participants
n=73 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=9 Participants
|
—
|
3 Participants
n=23 Participants
|
6 Participants
n=73 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=9 Participants
|
—
|
0 Participants
n=23 Participants
|
0 Participants
n=73 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=9 Participants
|
—
|
0 Participants
n=23 Participants
|
0 Participants
n=73 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
—
|
0 Participants
n=23 Participants
|
0 Participants
n=73 Participants
|
PRIMARY outcome
Timeframe: Up to 1 yearPopulation: Clinical trial closed before enrollment into arm: PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
Number of participants at 1 year post stem cell transplant who have not experienced graft failure and who are without severe graft-versus-host disease (defined as grade 3 and higher acute GVHD). Graft failure is defined as the absence of or insufficient donor chimerism associated with the return of acute complications of sickle cell disease. As defined by CIMBTR criteria for Organ Stages of Acute GVHD: Grade III: Skin = Rash on \>50% of body surface; Liver = Total Bilirubin 6.1 - 15.0 mg/dL; Lower GI = Diarrhea \> 1500 mL/day. Grade IV: Skin = Generalized erythroderma plus bullous formation; Liver = Total Bilirubin \>15 mg/dL; Lower GI = Severe abdominal pain with or without ileus. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.
Outcome measures
| Measure |
PBSC Transplant With 50 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
n=3 Participants
Stem cell recipients with Sickle Cell Disease will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. This group will receive a single dose of PT-Cy; 50 mg/kg, on day +3 post-HCT
|
PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
Stem cell recipients with Sickle Cell Disease will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. This group will receive a total of two doses of PT-Cy; 50mg/kg, on days +3 and +4 (total 100 mg/kg) post-HCT
|
|---|---|---|
|
Number of Participants With Successful Engraftment and Absence of Acute Grade 3 or Higher GVHD
|
2 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Up to 1 yearPopulation: Clinical trial closed before enrollment into arm: PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
Number of participants at 1 year post stem cell transplant who have not experienced graft failure and who are without moderate to severe chronic graft-versus-host disease (GVHD) Graft failure is defined as the absence of or insufficient donor chimerism associated with the return of acute complications of sickle cell disease. CIMBTR criteria definition for Chronic GVHD: Moderate chronic GVHD involves EITHER 3 organs/sites with no clinically significant functional impairment OR a less than or equal to 1 organ/site with clinically significant functional impairment, but no major disability. Severe GVHD is associated with a major disability caused by chronic GVHD.
Outcome measures
| Measure |
PBSC Transplant With 50 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
n=3 Participants
Stem cell recipients with Sickle Cell Disease will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. This group will receive a single dose of PT-Cy; 50 mg/kg, on day +3 post-HCT
|
PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
Stem cell recipients with Sickle Cell Disease will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. This group will receive a total of two doses of PT-Cy; 50mg/kg, on days +3 and +4 (total 100 mg/kg) post-HCT
|
|---|---|---|
|
Number of Participants With Successful Engraftment and Absence of Moderate to Severe Chronic Graft-versus-host Disease
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: length of studyOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: length of studyOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: length of studyOutcome measures
Outcome data not reported
Adverse Events
PBSC Transplant With 50 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
Serious events: 2 serious events
Other events: 3 other events
Deaths: 1 deaths
PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Human Leukocyte Antigens (HLA) Haploidentical Related Stem Cell Donor
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
PBSC Transplant With 50 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
n=3 participants at risk
Stem cell recipients with Sickle Cell Disease will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. This group will receive a single dose of PT-Cy; 50 mg/kg, on day +3 post-HCT
|
PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
Stem cell recipient participants will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. Cohort 2 will receive a total of two doses of PT-Cy; 50mg/kg, on days +3 and +4 (total 100 mg/kg) post-HCT
|
Human Leukocyte Antigens (HLA) Haploidentical Related Stem Cell Donor
n=3 participants at risk
A haploidentical relative donor will receive filgrastim (G-CSF) 10 to 16 µg/kg/d subcutaneously or intravenously for up to 6 days with apheresis collections of peripheral blood hematopoietic progenitor cells (PBPC) after the 5th day (and after the 6th day if required).
|
|---|---|---|---|
|
Immune System Disorders
Hyperinflammatory Syndrome
|
33.3%
1/3 • Number of events 2 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Infections and Infestations
Bacteremia
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Vascular Disorders
Deep Vein Thrombosis
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
General Disorders and Administration Site Conditions
Pain
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Cardiac Disorders
Dilated cardiomyopathy
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Gastrointestinal Disorders
Diarrhea
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Hepatobiliary Disorders
Hepatolithiasis
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Hepatobiliary Disorders
Cholangitis
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Hepatobiliary Disorders
Choledocholithiasis
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
Other adverse events
| Measure |
PBSC Transplant With 50 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
n=3 participants at risk
Stem cell recipients with Sickle Cell Disease will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. This group will receive a single dose of PT-Cy; 50 mg/kg, on day +3 post-HCT
|
PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy)
Stem cell recipient participants will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. Cohort 2 will receive a total of two doses of PT-Cy; 50mg/kg, on days +3 and +4 (total 100 mg/kg) post-HCT
|
Human Leukocyte Antigens (HLA) Haploidentical Related Stem Cell Donor
n=3 participants at risk
A haploidentical relative donor will receive filgrastim (G-CSF) 10 to 16 µg/kg/d subcutaneously or intravenously for up to 6 days with apheresis collections of peripheral blood hematopoietic progenitor cells (PBPC) after the 5th day (and after the 6th day if required).
|
|---|---|---|---|
|
Eye disorders
Dry eye
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Gastrointestinal disorders
Anal pain
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Gastrointestinal disorders
Diarrhea
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Gastrointestinal disorders
Mucositis oral
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Gastrointestinal disorders
Nausea
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
General disorders
Chills
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
General disorders
Fever
|
66.7%
2/3 • Number of events 3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
General disorders
Generalized edema
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Hepatobiliary disorders
Gallbladder obstruction
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Immune system disorders
GVHD (GI GR-2)
|
66.7%
2/3 • Number of events 2 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Infections and Infestations
COVID infection
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Infections and Infestations
Sepsis
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Injury, Poisoning and Procedural Complications
Infusion related reaction
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Investigations
Alanine aminotransferase increased
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Investigations
Aspartate aminotransferase increased
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Investigations
Blood bilirubin increased
|
66.7%
2/3 • Number of events 2 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Investigations
Neutrophil count decreased
|
100.0%
3/3 • Number of events 3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Investigations
Platelet count decreased
|
100.0%
3/3 • Number of events 3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Metabolism and Nutrition Disorders
Hyperglycemia
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Musculoskeletal and Connective Tissue Disorders
Bone pain
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Nervous System Disorders
Headache
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Renal and Urinary Disorders
Proteinuria
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Reproductive System and Breast Disorders
Testicular disorder
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
|
Vascular Disorders
Hypertension
|
33.3%
1/3 • Number of events 1 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
—
0/0 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
0.00%
0/3 • Up to 22 months
Adverse Events and Serious Adverse Events whether volunteered by the patient, discovered by study personnel during questioning, or detected through physical examination, clinically significant laboratory test, upon review by a study monitor or other means will be recorded. The clinical trial concluded enrollment prior to recruiting any participants for PBSC Transplant With 100 mg/kg Post-transplant Cyclophosphamide (PT- Cy) cohort.
|
Additional Information
Dr. Courtney Fitzhugh
National Heart, Lung, and Blood Institute (NHLBI)
Phone: 301.402.6496
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place