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Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in GRD

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Seoul National University

Status and phase

Enrolling
Phase 2
Phase 1

Conditions

Genetic Rare Disease

Treatments

Drug: Busulfan

Study type

Interventional

Funder types

Other

Identifiers

NCT02034630
SNUCH_GRDBuFlu

Details and patient eligibility

About

To evaluate the outcome of hematopoietic stem cell transplantation using targeted busulfan, fludarabine conditioning regimen in genetic rare disease

Full description

Busulfan is a highly toxic drug with narrow therapeutic window used for the conditioning of hematopoietic stem cell transplantation. High exposure is associated with systemic toxicity such as veno-occlusive disease (VOD) and underexposure is associated with graft failure or relapse. In this study the investigators plan to improve the outcome of hematopoietic stem cell transplantation by using optimal busulfan dose through pharmacokinetic study.

Enrollment

5 estimated patients

Sex

All

Ages

Under 21 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  1. Patients who are diagnosed as genetic rare disease.
  2. Patients who need hematopoietic stem cell transplantation
  3. Age: up to 21 years
  4. Performance status: ECOG 0-2.
  5. Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases. 1. Heart: a shortening fraction > 30% and ejection fraction > 45%. 2. Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of normal. 3. Kidney: creatinine <2 × normal or a creatinine clearance (GFR) > 60 ml/min/1.73m2.
  6. Patients must lack any active viral infections or active fungal infection.
  7. Appropriate donor is available: Matched in 6/6 of A, B, DR loci.
  8. Patients (or one of parents if patients age < 19) should sign informed consent.

Exclusion criteria

  1. Pregnant or nursing women.
  2. Malignant or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.
  3. Psychiatric disorder that would preclude compliance.

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

5 participants in 1 patient group

Busulfan
Experimental group
Description:
First dose: busulfan (120 mg/m2 ivs once daily) (if age\<1 yr: 80 mg/ m2) Second to forth dose: according to the daily pharmacokinetic study
Treatment:
Drug: Busulfan

Trial contacts and locations

1

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Central trial contact

Ji Won Lee, MD; Hyoung Jin Kang, MD, PhD

Data sourced from clinicaltrials.gov

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