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

• ClinicalTrials.gov Identifier: NCT02034630
• Recruitment Status: Unknown
• First Posted: January 13, 2014
• Last Update Posted: April 8, 2019
• Sponsor: Seoul National University Hospital
• Information provided by (Responsible Party): Seoul National University Hospital

Tracking Information

• First Submitted Date: January 9, 2014
• First Posted Date: January 13, 2014
• Last Update Posted Date: April 8, 2019
• Study Start Date: January 2014
• Estimated Primary Completion Date: April 2019 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: January 10, 2014)

To evaluate engraftment rate [ Time Frame: 1 year ]

To evaluate engraftment rate

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: January 10, 2014)

To evaluate treatment related mortality [ Time Frame: 1 year ]

To evaluate treatment related mortality

• Original Secondary Outcome Measures: Same as current

Current Other Pre-specified Outcome Measures (submitted: January 10, 2014)

• To evaluate survival rate [ Time Frame: 1, 3, 6 and 12 months after transplantation ]
  To evaluate survival rate
• To evaluate toxicities associated with hematopoietic stem cell transplantation [ Time Frame: 1, 3, 6 and 12 months after transplantation ]
  To evaluate toxicities associated with hematopoietic stem cell transplantation
• To evaluate acute GVHD [ Time Frame: 1, 3, 6 and 12 months after transplantation ]
  To evaluate acute GVHD
• To evaluate chronic GVHD [ Time Frame: 1, 3, 6 and 12 months after transplantation ]
  To evaluate chronic GVHD

• Original Other Pre-specified Outcome Measures: Same as current

Descriptive Information

• Brief Title: Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in GRD
• Official Title: Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Genetic Rare Disease
• Brief Summary: To evaluate the outcome of hematopoietic stem cell transplantation using targeted busulfan, fludarabine conditioning regimen in genetic rare disease
• 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
• Study Phase: Phase 1; Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Genetic Rare Disease

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

Study Arms

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

Intervention: Drug: Busulfan

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Unknown status
• Estimated Enrollment (submitted: January 10, 2014): 5
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 2019
• Estimated Primary Completion Date: April 2019 (Final data collection date for primary outcome measure)

Eligibility 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: up to 21 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Korea, Republic of

Administrative Information

• NCT Number: NCT02034630
• Other Study ID Numbers: SNUCH_GRDBuFlu
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Seoul National University Hospital
• Study Sponsor: Seoul National University Hospital
• Collaborators: Not Provided

Investigators

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

• PRS Account: Seoul National University Hospital
• Verification Date: April 2018