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Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

This study is currently recruiting participants.
Verified May 2012 by Tehran University of Medical Sciences
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
History of Changes
  Purpose

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 Intervention Phase
Mucopolysaccharidosis
 Drug: Conditioning regimen
Drug: Graft-versus-host disease (GVHD) prophylaxis
Procedure: Stem Cell Transplantation
 Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Tehran University of Medical Sciences:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 10
Study Start Date: September 2009
Estimated Study Completion Date: December 2012
Estimated Primary Completion Date: September 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 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

  Eligibility

Ages Eligible for Study:   up to 8 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01238328

Contacts
Contact: Amir Ali Hamidieh, MD 84902645 ext +98 21 aahamidieh@sina.tums.ac.ir

Locations
Iran, Islamic Republic of
Hematology-Oncology & SCT Research Center Recruiting
Teharn, Tehran, Iran, Islamic Republic of, 14114
Contact: Amir Ali Hamidieh, MD     84902645 ext +9821     aahamidieh@sina.tums.ac.ir    
Principal Investigator: Amir Ali Hmidieh, MD            
Sub-Investigator: Ardeshir Ghavamzadeh, MD            
Sub-Investigator: Kamran Alimoghaddam, MD            
Sub-Investigator: Mahdi Jalili, MD            
Sponsors and Collaborators
Tehran University of Medical Sciences
  More Information

Additional Information:
Hematology-Oncology and Stem Cell Transplantation Research Center  This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party: Tehran University of Medical Sciences
ClinicalTrials.gov Identifier: NCT01238328     History of Changes
Other Study ID Numbers: HORCSCT-0906
Study First Received: November 8, 2010
Last Updated: May 31, 2012
Health Authority: Iran: Ministry of Health

Keywords provided by Tehran University of Medical Sciences:
Mucopolysaccharidosis
HSCT

Additional relevant MeSH terms:
Mucopolysaccharidoses
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
 Metabolic Diseases
Antilymphocyte Serum
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 22, 2013