Overview

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

Status:
Recruiting

Trial end date:
2021-11-01

Target enrollment:
0

Participant gender:
All

Summary

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

Phase:

Phase 1/Phase 2

Accepts Healthy Volunteers?

Details

Lead Sponsor:

Seoul National University Hospital

Treatments:

Busulfan
Fludarabine
Fludarabine phosphate

Criteria

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.