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

  • STATUS
    Recruiting
  • End date
    Nov 7, 2021
  • participants needed
    5
  • sponsor
    Seoul National University Hospital
Updated on 7 April 2021

Summary

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

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.

Details
Condition Genetic Rare Disease
Treatment busulfan
Clinical Study IdentifierNCT02034630
SponsorSeoul National University Hospital
Last Modified on7 April 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Patients who are diagnosed as genetic rare disease
Patients who need hematopoietic stem cell transplantation
Age: up to 21 years
Performance status: ECOG 0-2
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
Patients must lack any active viral infections or active fungal infection
Appropriate donor is available: Matched in 6/6 of A, B, DR loci
Patients (or one of parents if patients age < 19) should sign informed consent

Exclusion Criteria

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