Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

Study Overview
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Brief Summary
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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.

Official Title
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Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

Conditions
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Mucopolysaccharidosis

Intervention / Treatment
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* Drug: Conditioning regimen
* Drug: Graft-versus-host disease (GVHD) prophylaxis
* Procedure: Stem Cell Transplantation


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Maximum Age: 8 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Transplantation<br> | Drug: Conditioning regimen<br>* 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<br>* Other names: Antithymocyte globulin (ATG) horse : Atgam;Drug: Graft-versus-host disease (GVHD) prophylaxis<br>* 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)<br>* Other names: MTX;Procedure: Stem Cell Transplantation<br>* 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<br>* Other names: HSCT;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic Hematopoietic stem cell transplantation (HSCT) |  | 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| One year overall survival after allogeneic hematopoietic stem cell transplantation (HSCT) |  | 1 year | 
| One year Progressive Free Survival after allogeneic hematopoietic stem cell transplantation (HSCT) |  | 1 year | 
| Transplantation Related Mortality (TRM) after allogeneic hematopoietic stem cell transplantation (HSCT) |  | 1 year | 
| Acute and chronic Graft-versus-host disease (GVHD) rate after allogeneic hematopoietic stem cell transplantation (HSCT) |  | 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Mucopolysaccharidosis, HSCT