Pediatric Trial Investigating the Incidence & Outcome of... | NCT00272948

Studies
Pediatric Trial Investigating the Incidence & Outcome of Veno-Occlusive Disease With the Prophylactic Use of Defibrotide

NCT00272948

Sponsor

European Society for Blood and Marrow Transplantation

Status

Completed

Last Update Posted

Jun 9, 2011Estimated

Enrollment

360Estimated

Phase

Phase 2Phase 3

Conditions

Hepatic Veno-Occlusive Disease

Interventions

Defibrotide
Defibrotide
Defibrotide

Countries

Austria
France
Germany
Ireland
Israel
Italy
Netherlands
Sweden
Switzerland
United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

ID	Type	Description	Link
EBMT-PD-200601

Status Module

Record Verification Date

Jun 2011

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Dec 2005

Primary Completion Date

Jan 2009Actual

Completion Date

Jul 2009Actual

First Submitted Date

Jan 4, 2006

First Submission Date that Met QC Criteria

Jan 4, 2006

First Posted Date

Jan 9, 2006Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jun 8, 2011

Last Update Posted Date

Jun 9, 2011Estimated

Sponsor/Collaborators Module

Responsible Party, by Official Title

Not provided

Lead Sponsor

European Society for Blood and Marrow TransplantationNETWORK

Collaborators

Name	Class
Jazz Pharmaceuticals	INDUSTRY
Deutsche Krebshilfe e.V., Bonn (Germany)	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

The aim of this trial is to evaluate whether the prophylactic use of Defibrotide (DF) in pediatric patients (age less than 18 years) undergoing stem cell transplantation and who are at high risk of developing hepatic Veno-occlusive Disease (VOD) will have an impact on the incidence and severity of the disease. Patients will be randomly assigned to one of two treatment arms: Those allocated to the Prophylactic Arm will receive the study drug (Defibrotide) from the day of conditioning onwards. Patients allocated to the Control Arm will receive the study drug (Defibrotide) from the day that VOD is diagnosed.

Detailed Description

Comparison/control intervention and duration of the intervention:

Patients will be assigned randomly to either the Defibrotide (DF) prophylaxis arm or the control arm. Those allocated to the DF prophylaxis arm (DF 25 mg/kg/d iv in 4 doses) will begin treatment at day of conditioning and stop at day +30 after Stem Cell Transplantation (SCT) or upon discharge from inpatient care. There is no dose adjustment for a patient of the study arm who developed VOD, they continue with the 25mg/kg/d iv.

Patients allocated to the control arm receive no prophylactic measures and will start DF (25 mg/kg/d iv in 4 doses) beginning at day of diagnosis of Veno-occlusive Disease (VOD) according to modified Seattle criteria. Treatment will be stopped at complete resolution of symptoms. In both arms patients who developed VOD will continue DF until:

complete resolution of the ascites and
reversion of the hepatopedal flow (if present) and
normalization of the total and direct bilirubin

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

360Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Prophylaxis Arm	Experimental		Drug: Defibrotide
Control Arm	Active Comparator		Drug: Defibrotide

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Defibrotide	Drug	Defibrotide 25 mg/kg/d	Control Arm Prophylaxis Arm

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
The primary objective is to evaluate if prophylactic DF has an impact on the incidence of VOD		Day + 30 post HSCT

Secondary Outcomes

Measure	Description	Time Frame
Occurrence of Multi-System Organ Failure and Survival (all causes of mortality)		day +100 post HSCT

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Age <18 years
myeloablative conditioning and autologous or allogeneic stem cell transplantation with at least one of the following risk factors for VOD:
1. Pre-existing liver disease
2. Second myeloablative HSCT
3. History of treatment with gemtuzumab ozogamicin (MYLOTARGÒ, GO, CMA-676, Wyeth)
4. Allogeneic HSCT for leukemia beyond the second relapse
5. Osteopetrosis (OP)
6. Conditioning with busulfan and melphalan
7. Macrophage activating syndromes (MAS, like hemophagocytic lymphohistiocytosis, Griscelli, Chediak-Higashi
8. Adrenoleukodystrophy (ALD)

Exclusion Criteria:

Pregnant patients
Patients who are transplanted but do not fulfill any of the above mentioned criteria

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

18 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Selim Corbacioglu, MD	University of Ulm, Germany	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University Hospital		Graz			Austria
St Anna Kinderspital

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
37231094	Derived	Corbacioglu S, Grupp SA, Richardson PG, Duarte R, Pagliuca A, Ruutu T, Mahadeo K, Carreras E. Prevention of veno-occlusive disease/sinusoidal obstruction syndrome: a never-ending story and no easy answer. Bone Marrow Transplant. 2023 Aug;58(8):839-841. doi: 10.1038/s41409-023-02007-2. Epub 2023 May 25. No abstract available.
22364685

ID	Term
D008107	Liver Diseases
D004066	Digestive System Diseases
D014652	Vascular Diseases
D002318	Cardiovascular Diseases