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

Phase 1

Recruiting

• Conditions: Genetic Rare Disease
• Interventions: Drug: Busulfan

• Registration Number: NCT02034630
• Lead Sponsor: Seoul National University Hospital

Brief Summary

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

Detailed 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.

Study Timeline

• Study First Submitted: 2014-01-09
• Study First Submitted QC: 2014-01-10
• Study First Posted: 2014-01-13
• Study Start: 2014-07-21
• Status Verified: 2023-01
• Last Update Submitted: 2023-02-03
• Last Update Posted: 2023-02-08
• Primary Completion: 2023-12
• Study Completion: 2023-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 5

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Busulfan	Busulfan	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

Primary Outcome Measures

Name	Time	Method
To evaluate engraftment rate	1 year	To evaluate engraftment rate

Secondary Outcome Measures

Name	Time	Method
To evaluate treatment related mortality	1 year	To evaluate treatment related mortality

Trial Locations

Locations (1)

Seoul National University Hospital; 🇰🇷 Seoul, Daehangno, Jongno-gu, Korea, Republic of