Description:
This study is to compare the efficacy and safety of defibrotide prophylaxis in addition to
best supportive care versus best supportive care alone in the prevention of hepatic veno-
occlusive disease (VOD) in adult and pediatric patients undergoing hematopoietic stem cell
transplant who are at high risk or very high risk of developing VOD.
Title
- Brief Title: Study Comparing Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients
- Official Title: A Phase 3, Randomized, Adaptive Study Comparing the Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients Undergoing Hematopoietic Stem Cell Transplant
Clinical Trial IDs
- ORG STUDY ID:
15-007
- NCT ID:
NCT02851407
Conditions
Interventions
Drug | Synonyms | Arms |
---|
Defibrotide | | Defibrotide |
Purpose
This study is to compare the efficacy and safety of defibrotide prophylaxis in addition to
best supportive care versus best supportive care alone in the prevention of hepatic veno-
occlusive disease (VOD) in adult and pediatric patients undergoing hematopoietic stem cell
transplant who are at high risk or very high risk of developing VOD.
Trial Arms
Name | Type | Description | Interventions |
---|
Defibrotide | Experimental | Defibrotide is administered intravenously at a dose of 25 mg/kg/day in addition to best supportive care on the day before the first day of the conditioning regimen and will continue (for those patients without a VOD diagnosis) for a recommended minimum of 21 days and end no later than Day +30 post HSCT | |
Best Supportive Care | Other | Best supportive care alone (without the addition of defibrotide) according to institutional guidelines and patient need, is administered on the first day of conditioning and will continue until Day +30 post HSCT or hospital discharge, whichever is sooner, or diagnosis of VOD, if applicable | |
Eligibility Criteria
Inclusion Criteria:
1. Patient must be above the age of 1 month as of the start date of study treatment.
2. Patient must be scheduled to undergo allogeneic hematopoietic stem cell transplant
(HSCT) (adults or pediatric patients) or autologous HSCT (pediatric patients only) and
be at high risk or very high risk of developing veno-occlusive disease (VOD).
3. Female patients (and female partners of male patients) of childbearing potential who
are sexually active must agree to use a highly effective method of contraception with
their partners during exposure to defibrotide and for 1 week after the last dose of
defibrotide.
4. Adult patients must be able to understand and sign a written informed consent. For
minor patients, the parent/legal guardian or representative must be able to understand
and sign a written informed consent. Assent, when appropriate, will be obtained
according to institutional guidelines.
Exclusion Criteria:
1. Patient has hemodynamic instability within 24 hours before the start of study
treatment.
2. Patient has acute bleeding that is clinically significant within 24 hours before the
start of study treatment.
3. Patient used any medication that increases the risk of bleeding within 24 hours before
the start of study treatment.
4. Patient is using or plans to use an investigational agent for the prevention or
treatment of VOD.
5. Patient, in the opinion of the investigator, may not be able to comply with the safety
monitoring requirements of the study.
6. Patient or parent/legal guardian or representative has a psychiatric illness that
would prevent the patient or parent/legal guardian or representative from giving
informed consent and/or assent.
7. Patient has a serious active disease or co-morbid medical condition, as judged by the
investigator, which would interfere with the conduct of this study.
8. Patient is pregnant or lactating and does not agree to stop breastfeeding.
9. Patient has a known history of hypersensitivity to defibrotide or any of the
excipients.
10. Patient or parent/legal guardian or representative lacks the full mental capacity to
understand and sign a written informed consent.
11. Patient is receiving or plans to receive other investigational therapy during study.
Maximum Eligible Age: | N/A |
Minimum Eligible Age: | 1 Month |
Eligible Gender: | All |
Healthy Volunteers: | No |
Primary Outcome Measures
Measure: | Veno-Occlusive Disease (VOD)-Free Survival at Day 30 Post-Hemotopoietic Stem Cell Transplant (HSCT) |
Time Frame: | 30 Days Post-Transplant |
Safety Issue: | |
Description: | Comparison of efficacy of defibrotide vs Best Supportive Care (BSC) for the prevention of VOD |
Secondary Outcome Measures
Measure: | Veno-Occlusive Disease (VOD)-Free Survival at Day 100 Post-Hemotopoietic Stem Cell Transplant (HSCT) |
Time Frame: | 100 Days Post-Transplant |
Safety Issue: | |
Description: | Comparison of efficacy of defibrotide vs Best Supportive Care (BSC) for the prevention of VOD |
Details
Phase: | Phase 3 |
Primary Purpose: | Interventional |
Overall Status: | Completed |
Lead Sponsor: | Jazz Pharmaceuticals |
Trial Keywords
- stem cell transplant
- hematopoietic stem cell transplant (HSCT)
- veno-occlusive disease (VOD)
- sinosoidial obstruction syndrome (SOS)
Last Updated
October 22, 2020