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

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

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

Study Overview

• Status: Recruiting
• Conditions: Genetic Rare Disease
• Intervention / Treatment: Drug: Busulfan

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 Type: Interventional
• Enrollment (Anticipated): 5
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Ji Won Lee, MD; Phone Number: 82 2 2072 4192; Email: agnesjw@hanmail.net

Study Locations

• Korea, Republic of
  • Daehangno, Jongno-gu
    • Seoul, Daehangno, Jongno-gu, Korea, Republic of
      • Recruiting
      • Seoul National University Hospital
      • Contact: Ji Won Lee, MD; Phone Number: 82 2 2072 3452; Email: agnesjw@hanmail.net

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 21 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate engraftment rate	To evaluate engraftment rate	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate treatment related mortality	To evaluate treatment related mortality	1 year

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate survival rate	To evaluate survival rate	1, 3, 6 and 12 months after transplantation
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
To evaluate acute GVHD	To evaluate acute GVHD	1, 3, 6 and 12 months after transplantation
To evaluate chronic GVHD	To evaluate chronic GVHD	1, 3, 6 and 12 months after transplantation

Collaborators and Investigators

• Sponsor: Seoul National University Hospital

Study record dates

Study Major Dates

• Study Start (ACTUAL): July 21, 2014
• Primary Completion (ANTICIPATED): December 1, 2023
• Study Completion (ANTICIPATED): December 1, 2023

Study Registration Dates

• First Submitted: January 9, 2014
• First Submitted That Met QC Criteria: January 10, 2014
• First Posted (ESTIMATE): January 13, 2014

Study Record Updates

• Last Update Posted (ACTUAL): February 8, 2023
• Last Update Submitted That Met QC Criteria: February 3, 2023
• Last Verified: January 1, 2023

More Information

Terms related to this study

• Keywords: hematopoietic stem cell transplantation
• Additional Relevant MeSH Terms: Pathologic Processes; Disease Attributes; Rare Diseases; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Antineoplastic Agents, Alkylating; Alkylating Agents; Myeloablative Agonists; Busulfan
• Other Study ID Numbers: SNUCH_GRDBuFlu