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Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01238328
Recruitment Status : Unknown
Verified May 2012 by Tehran University of Medical Sciences.
Recruitment status was:  Recruiting
First Posted : November 10, 2010
Last Update Posted : June 4, 2012
Sponsor:
Information provided by (Responsible Party):
Tehran University of Medical Sciences

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

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated 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 : September 2009
Estimated Primary Completion Date : September 2012
Estimated Study Completion Date : December 2012

Resource links provided by the National Library of Medicine


Arms and Interventions
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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 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


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

Secondary Outcome Measures :
  1. One year overall survival after allogeneic hematopoietic stem cell transplantation (HSCT) [ Time Frame: 1 year ]
  2. One year Progressive Free Survival after allogeneic hematopoietic stem cell transplantation (HSCT) [ Time Frame: 1 year ]
  3. Transplantation Related Mortality (TRM) after allogeneic hematopoietic stem cell transplantation (HSCT) [ Time Frame: 1 year ]
  4. Acute and chronic Graft-versus-host disease (GVHD) rate after allogeneic hematopoietic stem cell transplantation (HSCT) [ Time Frame: 1 year ]

Eligibility Criteria
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Ages Eligible for Study:   up to 8 Years   (Child)
Sexes Eligible for Study:   All
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
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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): NCT01238328


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

Locations
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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
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Responsible Party: Tehran University of Medical Sciences
ClinicalTrials.gov Identifier: NCT01238328    
Other Study ID Numbers: HORCSCT-0906
First Posted: November 10, 2010    Key Record Dates
Last Update Posted: June 4, 2012
Last Verified: May 2012
Keywords provided by Tehran University of Medical Sciences:
Mucopolysaccharidosis
HSCT
Additional relevant MeSH terms:
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Mucopolysaccharidoses
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
 Metabolic Diseases
Thymoglobulin
Antilymphocyte Serum
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs