Mucopolysaccharidosis
Conditions
Keywords
Mucopolysaccharidosis, HSCT
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.
Interventions
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
DRUGGraft-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)
PROCEDUREStem 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
Sponsors
Tehran University of Medical Sciences
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 8 Years
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Overall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic Hematopoietic stem cell transplantation (HSCT) | 1 year |
Secondary
| Measure | 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 |
Countries
Iran
Contacts
Primary ContactAmir Ali Hamidieh, MD
Outcome results
None listed