Trial Outcomes & Findings for A Study to Compare T-Guard vs Ruxolitinib for Treatment of Steroid-Refractory Acute Graft-vs-Host Disease (BMT CTN 2002) (NCT NCT04934670)
NCT ID: NCT04934670
Last Updated: 2024-08-27
Results Overview
The primary objective of this trial is to assess the rate of CR on Day 28 post-randomization in Grades III and IV SR-aGVHD patients treated with T-Guard treatment in comparison to ruxolitinib. Participants were classified as Day 28 CR if these three conditions were satisfied: 1\. Stage 0 aGVHD (no remaining symptoms) in the three target organs skin, bowel and liver, 2 . The participant is still alive at Day 28, 3 . No additional systemic treatment for aGVHD has been administered through Day 28.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
12 participants
Primary outcome timeframe
Day 28
Results posted on
2024-08-27
Participant Flow
Participant milestones
| Measure |
T-Guard
Participants will be administered four doses of T-Guard intravenously for a 4-hour period every other day
T-Guard: T-Guard will be administered intravenously inpatient over 4 hours every 2 calendar days on Days 0, 2, 4, and 6, at a dose of 4mg/m2 Body Surface Area (BSA).
|
Ruxolitinib
Participants will take ruxolitinib twice daily for continuous daily dosing
Ruxolitinib: Ruxolitinib will be administered orally twice a day starting on Day 0 through Day 56, at a dose of 10mg. Ruxolitinib taper can be initiated starting on Day 56 for participants responding to treatment, tapering will be done according to institutional practices.
|
|---|---|---|
|
Overall Study
STARTED
|
7
|
5
|
|
Overall Study
COMPLETED
|
2
|
1
|
|
Overall Study
NOT COMPLETED
|
5
|
4
|
Reasons for withdrawal
| Measure |
T-Guard
Participants will be administered four doses of T-Guard intravenously for a 4-hour period every other day
T-Guard: T-Guard will be administered intravenously inpatient over 4 hours every 2 calendar days on Days 0, 2, 4, and 6, at a dose of 4mg/m2 Body Surface Area (BSA).
|
Ruxolitinib
Participants will take ruxolitinib twice daily for continuous daily dosing
Ruxolitinib: Ruxolitinib will be administered orally twice a day starting on Day 0 through Day 56, at a dose of 10mg. Ruxolitinib taper can be initiated starting on Day 56 for participants responding to treatment, tapering will be done according to institutional practices.
|
|---|---|---|
|
Overall Study
Death
|
4
|
0
|
|
Overall Study
Lost to Follow-up
|
1
|
4
|
Baseline Characteristics
A Study to Compare T-Guard vs Ruxolitinib for Treatment of Steroid-Refractory Acute Graft-vs-Host Disease (BMT CTN 2002)
Baseline characteristics by cohort
| Measure |
T-Guard
n=7 Participants
Participants will be administered four doses of T-Guard intravenously for a 4-hour period every other day
T-Guard: T-Guard will be administered intravenously inpatient over 4 hours every 2 calendar days on Days 0, 2, 4, and 6, at a dose of 4mg/m2 Body Surface Area (BSA).
|
Ruxolitinib
n=5 Participants
Participants will take ruxolitinib twice daily for continuous daily dosing
Ruxolitinib: Ruxolitinib will be administered orally twice a day starting on Day 0 through Day 56, at a dose of 10mg. Ruxolitinib taper can be initiated starting on Day 56 for participants responding to treatment, tapering will be done according to institutional practices.
|
Total
n=12 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
3 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
10 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
|
Region of Enrollment
Netherlands
|
1 participants
n=99 Participants
|
0 participants
n=107 Participants
|
1 participants
n=206 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=99 Participants
|
0 participants
n=107 Participants
|
3 participants
n=206 Participants
|
|
Region of Enrollment
France
|
3 participants
n=99 Participants
|
3 participants
n=107 Participants
|
6 participants
n=206 Participants
|
|
Region of Enrollment
Germany
|
0 participants
n=99 Participants
|
2 participants
n=107 Participants
|
2 participants
n=206 Participants
|
|
Age 65 or above
|
3 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Grade IV steroid-refractory aGVHD (SR-aGVHD) at randomization
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Hematopoietic Cell Transplantation-specific Comorbidity Index (HCT-CI) above 3
|
3 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Baseline serum albumin levels below 2 mg/dL
|
5 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
7 Participants
n=206 Participants
|
|
Absolute neutrophil count (ANC) at or below 1000/µL
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Day 28Population: Intention to treat (ITT)
The primary objective of this trial is to assess the rate of CR on Day 28 post-randomization in Grades III and IV SR-aGVHD patients treated with T-Guard treatment in comparison to ruxolitinib. Participants were classified as Day 28 CR if these three conditions were satisfied: 1\. Stage 0 aGVHD (no remaining symptoms) in the three target organs skin, bowel and liver, 2 . The participant is still alive at Day 28, 3 . No additional systemic treatment for aGVHD has been administered through Day 28.
Outcome measures
| Measure |
T-Guard
n=7 Participants
Participants will be administered four doses of T-Guard intravenously for a 4-hour period every other day
T-Guard: T-Guard will be administered intravenously inpatient over 4 hours every 2 calendar days on Days 0, 2, 4, and 6, at a dose of 4mg/m2 Body Surface Area (BSA).
|
Ruxolitinib
n=5 Participants
Participants will take ruxolitinib twice daily for continuous daily dosing
Ruxolitinib: Ruxolitinib will be administered orally twice a day starting on Day 0 through Day 56, at a dose of 10mg. Ruxolitinib taper can be initiated starting on Day 56 for participants responding to treatment, tapering will be done according to institutional practices.
|
|---|---|---|
|
Complete Response (CR)
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 180Population: Participants randomized to the treatment arms were analyzed. For both treatment arms, the number of participants who died are reported.
OS is defined as survival of death from any cause. The time from randomization until death from any cause will be described for each arm.
Outcome measures
| Measure |
T-Guard
n=7 Participants
Participants will be administered four doses of T-Guard intravenously for a 4-hour period every other day
T-Guard: T-Guard will be administered intravenously inpatient over 4 hours every 2 calendar days on Days 0, 2, 4, and 6, at a dose of 4mg/m2 Body Surface Area (BSA).
|
Ruxolitinib
n=5 Participants
Participants will take ruxolitinib twice daily for continuous daily dosing
Ruxolitinib: Ruxolitinib will be administered orally twice a day starting on Day 0 through Day 56, at a dose of 10mg. Ruxolitinib taper can be initiated starting on Day 56 for participants responding to treatment, tapering will be done according to institutional practices.
|
|---|---|---|
|
Overall Survival (OS)
|
4 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Day 28DoCR will be evaluated only in the set of participants who are in CR at Day 28 post-randomization. The primary definition of DoCR is the time from Day 28 until an aGVHD target organ worsens by at least 1 stage and requires a significant escalation in treatment, or death.
Outcome measures
| Measure |
T-Guard
n=7 Participants
Participants will be administered four doses of T-Guard intravenously for a 4-hour period every other day
T-Guard: T-Guard will be administered intravenously inpatient over 4 hours every 2 calendar days on Days 0, 2, 4, and 6, at a dose of 4mg/m2 Body Surface Area (BSA).
|
Ruxolitinib
n=5 Participants
Participants will take ruxolitinib twice daily for continuous daily dosing
Ruxolitinib: Ruxolitinib will be administered orally twice a day starting on Day 0 through Day 56, at a dose of 10mg. Ruxolitinib taper can be initiated starting on Day 56 for participants responding to treatment, tapering will be done according to institutional practices.
|
|---|---|---|
|
Duration of Complete Response (DoCR)
|
2 Participants
|
0 Participants
|
Adverse Events
T-Guard
Serious events: 5 serious events
Other events: 7 other events
Deaths: 4 deaths
Ruxolitinib
Serious events: 2 serious events
Other events: 5 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
T-Guard
n=7 participants at risk
Participants will be administered four doses of T-Guard intravenously for a 4-hour period every other day
T-Guard: T-Guard will be administered intravenously inpatient over 4 hours every 2 calendar days on Days 0, 2, 4, and 6, at a dose of 4mg/m2 Body Surface Area (BSA).
|
Ruxolitinib
n=5 participants at risk
Participants will take ruxolitinib twice daily for continuous daily dosing
Ruxolitinib: Ruxolitinib will be administered orally twice a day starting on Day 0 through Day 56, at a dose of 10mg. Ruxolitinib taper can be initiated starting on Day 56 for participants responding to treatment, tapering will be done according to institutional practices.
|
|---|---|---|
|
Infections and infestations
Enterobacter infection
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Infections and infestations
Fungal infections
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Infections and infestations
Septic Shock
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Nervous system disorders
Subarachnoid Hemorrhage
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Gastrointestinal disorders
Lower gastrointestinal hemorrhage
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Gastrointestinal disorders
Small intestinal hemorrhage
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 2 • 180 days
|
|
General disorders
Multipel organ dysfunction syndrome
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
Other adverse events
| Measure |
T-Guard
n=7 participants at risk
Participants will be administered four doses of T-Guard intravenously for a 4-hour period every other day
T-Guard: T-Guard will be administered intravenously inpatient over 4 hours every 2 calendar days on Days 0, 2, 4, and 6, at a dose of 4mg/m2 Body Surface Area (BSA).
|
Ruxolitinib
n=5 participants at risk
Participants will take ruxolitinib twice daily for continuous daily dosing
Ruxolitinib: Ruxolitinib will be administered orally twice a day starting on Day 0 through Day 56, at a dose of 10mg. Ruxolitinib taper can be initiated starting on Day 56 for participants responding to treatment, tapering will be done according to institutional practices.
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
42.9%
3/7 • Number of events 4 • 180 days
|
0.00%
0/5 • 180 days
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Blood and lymphatic system disorders
Thrombotic microangiopathy
|
28.6%
2/7 • Number of events 2 • 180 days
|
0.00%
0/5 • 180 days
|
|
Congenital, familial and genetic disorders
Factor XIII deficiency
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Eye disorders
Conjunctival hemorrhage
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 2 • 180 days
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Gastrointestinal disorders
Duodenal ulcer
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Gastrointestinal disorders
Gastrointestinal hemorhage
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Gastrointestinal disorders
Jejunal ulcer
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Gastrointestinal disorders
Lower gastrointestinal hemorrhage
|
28.6%
2/7 • Number of events 5 • 180 days
|
0.00%
0/5 • 180 days
|
|
Gastrointestinal disorders
Small intestinal hemorrhage
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 2 • 180 days
|
|
General disorders
Multiple organ dysfunction syndrome
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
General disorders
Edema peripheral
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
General disorders
Physical deconditioning
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
General disorders
Pyrexia
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Hepatobiliary disorders
Bile duct stenosis
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Hepatobiliary disorders
Cholecystisis
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Hepatobiliary disorders
Hyperbilirubinemia
|
0.00%
0/7 • 180 days
|
40.0%
2/5 • Number of events 2 • 180 days
|
|
Infections and infestations
Enterobacter infection
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Infections and infestations
Fungal infection
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Infections and infestations
Pancreatic abscess
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Infections and infestations
Septic shock
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Infections and infestations
Sinusitis
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Investigations
Blood bilirubin increased
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Investigations
Blood lactate dehydrogenase increased
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Investigations
Lipase increased
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Investigations
Lymphocyte count decreased
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Investigations
Neutrophil count decreased
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Investigations
Platelet count decreased
|
28.6%
2/7 • Number of events 2 • 180 days
|
0.00%
0/5 • 180 days
|
|
Investigations
white blood cell count decreased
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Metabolism and nutrition disorders
Acidosis
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
28.6%
2/7 • Number of events 2 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Metabolism and nutrition disorders
Hypernatremia
|
14.3%
1/7 • Number of events 2 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Metabolism and nutrition disorders
Hypervolemia
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
14.3%
1/7 • Number of events 2 • 180 days
|
0.00%
0/5 • 180 days
|
|
Metabolism and nutrition disorders
Hypokalemia
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Musculoskeletal and connective tissue disorders
Athralgia
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Musculoskeletal and connective tissue disorders
Myopathy
|
0.00%
0/7 • 180 days
|
40.0%
2/5 • Number of events 2 • 180 days
|
|
Musculoskeletal and connective tissue disorders
Metabolic encephalopathy
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Nervous system disorders
Subarachnoid hemorrhage
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Psychiatric disorders
Delirium
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Renal and urinary disorders
Azotemia
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Renal and urinary disorders
Dysuria
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Renal and urinary disorders
Hematuria
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
14.3%
1/7 • Number of events 1 • 180 days
|
0.00%
0/5 • 180 days
|
|
Vascular disorders
Hypertension
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Vascular disorders
Hypotension
|
0.00%
0/7 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
|
Vascular disorders
Peripheral vain thrombosis
|
14.3%
1/7 • Number of events 1 • 180 days
|
20.0%
1/5 • Number of events 1 • 180 days
|
Additional Information
Ypke van Oosterhout, custodian of Sponsor
Xenikos BV (no longer in business)
Phone: +31 611 0177 611
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place