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

Genetic Rare Disease Clinical Trial

Official title:

Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Genetic Rare Disease

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02034630
• Other study ID #: SNUCH_GRDBuFlu
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: July 21, 2014
• Est. completion date: December 2023

Study information

• Verified date: January 2023
• Source: Seoul National University Hospital
• Contact: Hyoung Jin Kang, MD, PhD
• Phone: 82 2 2072 3304
• Email: kanghj[@]snu.ac.kr
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 5
• Est. completion date: December 2023
• Est. primary completion date: December 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 21 Years

Eligibility

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.

Related Conditions & MeSH terms

• Genetic Rare Disease
• Rare Diseases

Intervention

Drug:
• 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

Locations

Country	Name	City	State
Korea, Republic of	Seoul National University Hospital	Seoul	Daehangno, Jongno-gu

Sponsors (1)

Lead Sponsor	Collaborator
Seoul National University Hospital

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	To evaluate survival rate	To evaluate survival rate	1, 3, 6 and 12 months after transplantation
Other	To evaluate toxicities associated with hematopoietic stem cell transplantation	To evaluate toxicities associated with hematopoietic stem cell transplantation	1, 3, 6 and 12 months after transplantation
Other	To evaluate acute GVHD	To evaluate acute GVHD	1, 3, 6 and 12 months after transplantation
Other	To evaluate chronic GVHD	To evaluate chronic GVHD	1, 3, 6 and 12 months after transplantation
Primary	To evaluate engraftment rate	To evaluate engraftment rate	1 year
Secondary	To evaluate treatment related mortality	To evaluate treatment related mortality	1 year