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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 : Unknown
Verified April 2018 by Seoul National University Hospital.
Recruitment status was:  Recruiting
First Posted : January 13, 2014
Last Update Posted : April 8, 2019
Sponsor:
Information provided by (Responsible Party):
Seoul National University Hospital

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.

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Study Type : Interventional  (Clinical Trial)
Estimated 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
Study Start Date : January 2014
Estimated Primary Completion Date : April 2019
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rare Diseases
Drug Information available for: Busulfan

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




Primary Outcome Measures :
  1. To evaluate engraftment rate [ Time Frame: 1 year ]
    To evaluate engraftment rate


Secondary Outcome Measures :
  1. To evaluate treatment related mortality [ Time Frame: 1 year ]
    To evaluate treatment related mortality


Other Outcome Measures:
  1. To evaluate survival rate [ Time Frame: 1, 3, 6 and 12 months after transplantation ]
    To evaluate survival rate

  2. 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

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

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



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02034630


Contacts
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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

Locations
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Korea, Republic of
Seoul National University Hospital Recruiting
Seoul, Daehangno, Jongno-gu, Korea, Republic of
Contact: Hyoung Jin Kang, MD, PhD    82 2 2072 3304    kanghj@snu.ac.kr   
Contact: Ji Won Lee, MD    82 2 2072 3452    agnesjw@hanmail.net   
Sponsors and Collaborators
Seoul National University Hospital
Investigators
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Principal Investigator: Hyoung Jin Kang, M.D., Ph.D Seoul National University Hospital
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Responsible Party: Seoul National University Hospital
ClinicalTrials.gov Identifier: NCT02034630    
Other Study ID Numbers: SNUCH_GRDBuFlu
First Posted: January 13, 2014    Key Record Dates
Last Update Posted: April 8, 2019
Last Verified: April 2018
Keywords provided by Seoul National University Hospital:
hematopoietic stem cell transplantation
Additional relevant MeSH terms:
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Rare Diseases
Disease Attributes
Pathologic Processes
Busulfan
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antineoplastic Agents, Alkylating
Antineoplastic Agents
Myeloablative Agonists