Overview

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

Status:
Completed

Trial end date:
2009-07-01

Target enrollment:
0

Participant gender:
All

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.

Phase:

Phase 2/Phase 3

Accepts Healthy Volunteers?

Details

Lead Sponsor:

European Group for Blood and Marrow Transplantation
European Society for Blood and Marrow Transplantation

Collaborators:

Deutsche Krebshilfe e.V., Bonn (Germany)
Jazz Pharmaceuticals

Treatments:

Defibrotide

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