Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

The recruitment status of this study is unknown because the information has not been verified recently.
Verified May 2012 by Tehran University of Medical Sciences.
Recruitment status was  Recruiting
Sponsor:
Information provided by (Responsible Party):
Tehran University of Medical Sciences
ClinicalTrials.gov Identifier:
NCT01238328
First received: November 8, 2010
Last updated: May 31, 2012
Last verified: May 2012

November 8, 2010
May 31, 2012
September 2009
September 2012   (final data collection date for primary outcome measure)
Overall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic Hematopoietic stem cell transplantation (HSCT) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
Overall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic HSCT [ Time Frame: 1 year ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT01238328 on ClinicalTrials.gov Archive Site
  • One year overall survival after allogeneic hematopoietic stem cell transplantation (HSCT) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • One year Progressive Free Survival after allogeneic hematopoietic stem cell transplantation (HSCT) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • Transplantation Related Mortality (TRM) after allogeneic hematopoietic stem cell transplantation (HSCT) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • Acute and chronic Graft-versus-host disease (GVHD) rate after allogeneic hematopoietic stem cell transplantation (HSCT) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • One year overall survival after allogeneic HSCT [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • One year Progressive Free Survival after allogeneic HSCT [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • Transplantation Related Mortality (TRM) after allogeneic HSCT [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • Acute and chronic GVHD rate after allogeneic HSCT [ Time Frame: 1 year ] [ Designated as safety issue: No ]
Not Provided
Not Provided
 
Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis
Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

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.

Not Provided
Interventional
Phase 2
Phase 3
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Mucopolysaccharidosis
  • 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 Name: MTX
  • Procedure: Stem Cell Transplantation

    Patients undergoing Hematopoietic Stem Cell Transplantation from one of below source:

    1. Sibling full match
    2. Other related full match
    3. Sibling or other related with 1 mismatch antigen
    4. Cord Blood
    5. Haploidentical
    Other Name: HSCT
Experimental: Transplantation
Interventions:
  • Drug: Conditioning regimen
  • Drug: Graft-versus-host disease (GVHD) prophylaxis
  • Procedure: Stem Cell Transplantation
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
10
December 2012
September 2012   (final data collection date for primary outcome measure)

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
Both
up to 8 Years
No
Contact: Amir Ali Hamidieh, MD 84902645 ext +98 21 aahamidieh@sina.tums.ac.ir
Iran, Islamic Republic of
 
NCT01238328
HORCSCT-0906
Yes
Tehran University of Medical Sciences
Tehran University of Medical Sciences
Not Provided
Not Provided
Tehran University of Medical Sciences
May 2012

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