Trial record 2 of 68 for:    Open Studies | "Rare Diseases"

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

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified July 2014 by Seoul National University Hospital
Sponsor:
Information provided by (Responsible Party):
Seoul National University Hospital
ClinicalTrials.gov Identifier:
NCT02034630
First received: January 9, 2014
Last updated: July 11, 2014
Last verified: July 2014
  Purpose

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


Condition Intervention Phase
Genetic Rare Disease
Drug: Busulfan
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Genetic Rare Disease

Resource links provided by NLM:


Further study details as provided by Seoul National University Hospital:

Primary Outcome Measures:
  • To evaluate engraftment rate [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    To evaluate engraftment rate


Secondary Outcome Measures:
  • To evaluate treatment related mortality [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    To evaluate treatment related mortality


Other Outcome Measures:
  • To evaluate survival rate [ Time Frame: 1, 3, 6 and 12 months after transplantation ] [ Designated as safety issue: No ]
    To evaluate survival rate

  • To evaluate toxicities associated with hematopoietic stem cell transplantation [ Time Frame: 1, 3, 6 and 12 months after transplantation ] [ Designated as safety issue: No ]
    To evaluate toxicities associated with hematopoietic stem cell transplantation

  • To evaluate acute GVHD [ Time Frame: 1, 3, 6 and 12 months after transplantation ] [ Designated as safety issue: No ]
    To evaluate acute GVHD

  • To evaluate chronic GVHD [ Time Frame: 1, 3, 6 and 12 months after transplantation ] [ Designated as safety issue: No ]
    To evaluate chronic GVHD


Estimated Enrollment: 5
Study Start Date: January 2014
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   up to 21 Years
Genders Eligible for Study:   Both
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02034630

Contacts
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
Korea, Republic of
Seoul National University Hospital Not yet 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
Principal Investigator: Hyoung Jin Kang, M.D., Ph.D Seoul National University Hospital
  More Information

No publications provided

Responsible Party: Seoul National University Hospital
ClinicalTrials.gov Identifier: NCT02034630     History of Changes
Other Study ID Numbers: SNUCH_GRDBuFlu
Study First Received: January 9, 2014
Last Updated: July 11, 2014
Health Authority: Korea: Food and Drug Administration

Keywords provided by Seoul National University Hospital:
hematopoietic stem cell transplantation

Additional relevant MeSH terms:
Rare Diseases
Disease Attributes
Pathologic Processes
Fludarabine
Busulfan
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antineoplastic Agents, Alkylating
Myeloablative Agonists

ClinicalTrials.gov processed this record on September 18, 2014