Phase 2 Study of a Reduced-toxicity Myeloablative Conditionning Regimen Using Fludarabine and Full Doses of iv Busulfan in Pediatric Patients Not Eligible for Standard Myeloablative Conditioning Regimens

Nantes University Hospital2 sites in 1 country50 target enrollmentApril 2012

ConditionsHematologic Malignancy

InterventionsFludarabine IV- Busulfan IV (Busilvex®) - Anti-thymocyte globulines (Thymoglobuline®)

DrugsFludarabine IV- Busulfan IV (Busilvex®) - Anti-thymocyte globulines (Thymoglobuline®)

Overview

Phase: Phase 2
Intervention: Fludarabine IV- Busulfan IV (Busilvex®) - Anti-thymocyte globulines (Thymoglobuline®)
Conditions: Hematologic Malignancy
Sponsor: Nantes University Hospital
Enrollment: 50
Locations: 2
Primary Endpoint: Transplant-related mortality (TRM)
Status: Completed
Last Updated: 8 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The purpose of this study is to assess transplant-related mortality (TRM) at one year after allogeneic hematopoietic stem cell transplantation (allo-HSCT) prepared by a "reduced toxicity myeloablative" conditioning regimen in young patients (children and adolescents) with hematologic malignancies.

Registry

clinicaltrials.gov

Start Date

April 2012

End Date

October 24, 2017

Last Updated

8 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Nantes University Hospital

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

Not provided

Exclusion Criteria

•Patient has been administered any other systemic chemotherapeutic drug (including Gemtuzumab) within 21 days prior to trial enrollment and start of the conditioning regimen. Hydroxyurea is permitted if indicated to control induction refractory disease, and IT chemotherapy is allowed if indicated as maintenance treatment for previously diagnosed leptomeningeal disease, that has been in remission for at least 3 months prior to enrollment on this study.
•Active infection. Protocol PI will be final arbiter if there is uncertainty regarding whether a previous infection is resolved.
•Children and adolescents who are not older than 12 months and under 25 years
•A donor who is HLA mismatched at the level of more than one locus.
•Poor performance status (Lansky \< 50%)
•Life expectancy is severely limited by concomitant illness and expected to be \<12 weeks.
•Left ventricular ejection fraction \< 30%. Uncontrolled arrhythmias or symptomatic cardiac disease.
•Symptomatic pulmonary disease. FEV1, FVC and DLCO \<30% of expected corrected for hemoglobin.
•Creatinine clearance less than 30 mL/m per 1.73 m2 or requiring dialysis
•Evidence of chronic active hepatitis or cirrhosis. If positive hepatitis serology, discuss with Study Chairman and consider liver biopsy.

Arms & Interventions

Drugs

Fludarabine IV- Busulfan IV (Busilvex®) - Anti-thymocyte globulines (Thymoglobuline®)

Intervention: Fludarabine IV- Busulfan IV (Busilvex®) - Anti-thymocyte globulines (Thymoglobuline®)

Outcomes

Primary Outcomes

Transplant-related mortality (TRM)

Time Frame: 12 months

Evaluation of the cumulative incidence of TRM at 12 months after transplantation

Secondary Outcomes

Incidence of engraftment(Day+42)
Evaluation of overall (OS) and disease-free survival (DFS)(12 months)
Cumulative incidence of relapse, death from disease, and non-relapse mortality (NRM)(12 months)
Cumulative incidences and severity of acute and chronic Graft-versus-Host disease(12 months)
Immune Recovery (to be determined in a subgroup of patients)(12 months)