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

• Sponsor: Seoul National University Hospital (Other)
• Overall Status: Recruiting
• CT.gov ID: NCT02034630
• Collaborator: (none)
• Enrollment: 5
• Location: 1
• Arm: 1
• Anticipated Duration (Months): 87.4
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Genetic Rare Disease	Drug: Busulfan	Phase 1/Phase 2

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 Design

• Study Type: Interventional
• Anticipated Enrollment: 5 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Genetic Rare Disease
• Actual Study Start Date: Jul 21, 2014
• Anticipated Primary Completion Date: Nov 1, 2021
• Anticipated Study Completion Date: Nov 1, 2021

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 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	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

Outcome Measures

Primary Outcome Measures

1. To evaluate engraftment rate [1 year]

   To evaluate engraftment rate

Secondary Outcome Measures

1. To evaluate treatment related mortality [1 year]

   To evaluate treatment related mortality

Other Outcome Measures

1. To evaluate survival rate [1, 3, 6 and 12 months after transplantation]

   To evaluate survival rate

2. To evaluate toxicities associated with hematopoietic stem cell transplantation [1, 3, 6 and 12 months after transplantation]

   To evaluate toxicities associated with hematopoietic stem cell transplantation

3. To evaluate acute GVHD [1, 3, 6 and 12 months after transplantation]

   To evaluate acute GVHD

4. To evaluate chronic GVHD [1, 3, 6 and 12 months after transplantation]

   To evaluate chronic GVHD

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 21 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Seoul National University Hospital	Seoul	Daehangno, Jongno-gu	Korea, Republic of

Sponsors and Collaborators

• Seoul National University Hospital

Investigators

• Principal Investigator: Hyoung Jin Kang, M.D., Ph.D, Seoul National University Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Seoul National University Hospital
• ClinicalTrials.gov Identifier: NCT02034630
• Other Study ID Numbers: SNUCH_GRDBuFlu
• First Posted: Jan 13, 2014
• Last Update Posted: Mar 30, 2021
• Last Verified: Mar 1, 2021

• Keywords provided by Seoul National University Hospital: hematopoietic stem cell transplantation
• Additional relevant MeSH terms: Rare Diseases; Busulfan

Study Results

No Results Posted as of Mar 30, 2021