Clincosm LogoSign in Get a demo

Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

Sponsor
Tehran University of Medical Sciences (Other)
Overall Status
Unknown status
CT.gov ID
NCT01238328
Collaborator
(none)
10
Enrollment
1
Location
1
Arm
39
Duration (Months)
0.3
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to evaluate the efficacy and side effects of donor hematopoietic cells using chemotherapy regimen without total-body irradiation in children undergoing a hematopoietic stem cell transplant for Mucopolysaccharidosis. The blood stem cells will be derived from either related donor or unrelated umbilical cord blood or haploidentical donor.

Condition or Disease Intervention/Treatment Phase
  • Drug: Conditioning regimen
  • Drug: Graft-versus-host disease (GVHD) prophylaxis
  • Procedure: Stem Cell Transplantation
 Phase 2/Phase 3

Study Design

Study Type:
Interventional
Anticipated Enrollment :
10 participants
Allocation:
Non-Randomized
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis
Study Start Date :
Sep 1, 2009
Anticipated Primary Completion Date :
Sep 1, 2012
Anticipated Study Completion Date :
Dec 1, 2012

Arms and Interventions

Arm Intervention/Treatment
Experimental: Transplantation

Drug: Conditioning regimen
For sibling full match: Busulfan 16 mg/kg >5year - 20 mg/kg <5year po Cyclophosphamide 200 mg/kg iv For other related full match, sibling or other related with one antigen mismatch and umbilical cord blood: Busulfan 16 mg/kg >5year - 20 mg/kg <5year po Cyclophosphamide 200 mg/kg iv Antithymocyte globulin (ATG) rabbit (Thymoglobulin) 10 mg/kg or ATG horse (Atgam) 40 mg/kg For haploidentical: Busulfan 16 mg/kg >5year - 20 mg/kg <5year po Cyclophosphamide 200 mg/kg iv Fludarabine 160 mg/m^2
Other Names:
  • Endoxan
  • Antithymocyte globulin (ATG) rabbit : Thymoglobulin
  • Antithymocyte globulin (ATG) horse : Atgam

    • Drug: Graft-versus-host disease (GVHD) prophylaxis
    Cyclosporin A 1.5 mg/kg/day iv from -2, then 3 mg/kg/day iv (from +7 in peripheral blood stem cell transplant (PBSCT) and +11 in Bone marrow transplantation or Umbilical Cord Blood (UCB) transplantation) then 9 mg/kg/day po 10 mg/m^2 iv day +1 then 6 mg/m^ iv day +3 and +6 (Not for UCBT)
    Other Names:
  • MTX

    • Procedure: Stem Cell Transplantation
    Patients undergoing Hematopoietic Stem Cell Transplantation from one of below source: Sibling full match Other related full match Sibling or other related with 1 mismatch antigen Cord Blood Haploidentical
    Other Names:
  • HSCT

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Overall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic Hematopoietic stem cell transplantation (HSCT) [1 year]

    Secondary Outcome Measures

    1. One year overall survival after allogeneic hematopoietic stem cell transplantation (HSCT) [1 year]

    2. One year Progressive Free Survival after allogeneic hematopoietic stem cell transplantation (HSCT) [1 year]

    3. Transplantation Related Mortality (TRM) after allogeneic hematopoietic stem cell transplantation (HSCT) [1 year]

    4. Acute and chronic Graft-versus-host disease (GVHD) rate after allogeneic hematopoietic stem cell transplantation (HSCT) [1 year]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A to 8 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Diagnosis of Mucopolysaccharidosis type 1, 6 and 7 confirm by leukocyte enzyme assay

    • Age up to 8 year old

    • Have suitable donor

    Exclusion Criteria:

    • Creatinine clearance ≤ 40ml/min/1.73m^2

    • Bilirubin ≥ 3mg/dL

    • SGPT ≥ 500 U/L

    • Current severe infection

    • Evidence of CNS involvement

    • Morbidity such as blindness or deafness

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Hematology-Oncology & SCT Research Center Teharn Tehran Iran, Islamic Republic of 14114

    Sponsors and Collaborators

    • Tehran University of Medical Sciences

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Tehran University of Medical Sciences
    ClinicalTrials.gov Identifier:
    NCT01238328
    Other Study ID Numbers:
    • HORCSCT-0906
    First Posted:
    Nov 10, 2010
    Last Update Posted:
    Jun 4, 2012
    Last Verified:
    May 1, 2012
    Keywords provided by Tehran University of Medical Sciences
    Mucopolysaccharidosis
    HSCT
    Additional relevant MeSH terms:
    Mucopolysaccharidoses
    Thymoglobulin
    Antilymphocyte Serum

    Study Results

    No Results Posted as of Jun 4, 2012