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

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ClinicalTrials.gov Identifier: NCT02034630
Recruitment Status : Recruiting
First Posted : January 13, 2014
Last Update Posted : March 30, 2021
Sponsor:
Information provided by (Responsible Party):
Seoul National University Hospital

Tracking Information
First Submitted Date  ICMJE January 9, 2014
First Posted Date  ICMJE January 13, 2014
Last Update Posted Date March 30, 2021
Actual Study Start Date  ICMJE July 21, 2014
Estimated Primary Completion Date November 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 10, 2014)
To evaluate engraftment rate [ Time Frame: 1 year ]
To evaluate engraftment rate
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 10, 2014)
To evaluate treatment related mortality [ Time Frame: 1 year ]
To evaluate treatment related mortality
Original Secondary Outcome Measures  ICMJE 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  ICMJE Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in GRD
Official Title  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Genetic Rare Disease
Intervention  ICMJE 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  ICMJE 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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 10, 2014)
5
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 2021
Estimated Primary Completion Date November 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 21 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Hyoung Jin Kang, MD, PhD 82 2 2072 3304 kanghj@snu.ac.kr
Contact: Ji Won Lee, MD 82 2 2072 4192 agnesjw@hanmail.net
Listed Location Countries  ICMJE Korea, Republic of
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02034630
Other Study ID Numbers  ICMJE SNUCH_GRDBuFlu
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Seoul National University Hospital
Study Sponsor  ICMJE Seoul National University Hospital
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Hyoung Jin Kang, M.D., Ph.D Seoul National University Hospital
PRS Account Seoul National University Hospital
Verification Date March 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP