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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02851407




Registration number
NCT02851407
Ethics application status
Date submitted
27/07/2016
Date registered
1/08/2016
Date last updated
5/08/2020

Titles & IDs
Public 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
Scientific 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
Secondary ID [1] 0 0
15-007
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Veno-occlusive Disease 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Defibrotide
Other interventions - Best Supportive Care

Experimental: Defibrotide - 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

Other: Best Supportive Care - 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


Treatment: Drugs: Defibrotide


Other interventions: Best Supportive Care


Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Veno-Occlusive Disease (VOD)-Free Survival at Day 30 Post-Hemotopoietic Stem Cell Transplant (HSCT) - Comparison of efficacy of defibrotide vs Best Supportive Care (BSC) for the prevention of VOD
Timepoint [1] 0 0
30 Days Post-Transplant
Secondary outcome [1] 0 0
Veno-Occlusive Disease (VOD)-Free Survival at Day 100 Post-Hemotopoietic Stem Cell Transplant (HSCT) - Comparison of efficacy of defibrotide vs Best Supportive Care (BSC) for the prevention of VOD
Timepoint [1] 0 0
100 Days Post-Transplant

Eligibility
Key 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.
Minimum age
1 Month
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Prevention
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA,VIC
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
Royal Children's Hospital Melbourne - Melbourne
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
3052 - Melbourne
Recruitment outside Australia
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Jazz Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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 website
https://clinicaltrials.gov/show/NCT02851407
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Jazz Pharmaceuticals
Address 0 0
Jazz Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications