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

NCT02034630 | Unknown Phase 1 DMC

• 1 other identifier: SNUCH_GRDBuFlu
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Genetic Rare Disease

Keywords

hematopoietic stem cell transplantation

Outcome Measures

Primary Outcomes (1)

• To evaluate engraftment rate

  To evaluate engraftment rate

  1 year

Secondary Outcomes (1)

• To evaluate treatment related mortality

  1 year

Other Outcomes (4)

• To evaluate survival rate

  1, 3, 6 and 12 months after transplantation

• To evaluate toxicities associated with hematopoietic stem cell transplantation

  1, 3, 6 and 12 months after transplantation

• To evaluate acute GVHD

  1, 3, 6 and 12 months after transplantation

Study Arms (1)

Busulfan

EXPERIMENTAL

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

Interventions

Busulfan DRUG

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

Busulfan

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Seoul National University Hospital: lead

Study Sites (1)

Seoul National University Hospital

Seoul, Daehangno, Jongno-gu, South Korea

RECRUITING

MeSH Terms

Interventions

Busulfan

Intervention Hierarchy (Ancestors)

Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds

Study Officials

• Hyoung Jin Kang, M.D., Ph.D

  Seoul National University Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Hyoung Jin Kang, MD, PhD

CONTACT

82 2 2072 3304; kanghj@snu.ac.kr

Ji Won Lee, MD

CONTACT

82 2 2072 4192; agnesjw@hanmail.net

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 9, 2014
• First Posted: January 13, 2014
• Study Start: July 21, 2014
• Primary Completion: December 1, 2023
• Study Completion: December 1, 2023
• Last Updated: February 8, 2023

Record last verified: 2023-01

Locations

KR (1)