Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis
NCT ID: NCT01238328
Last Updated: 2012-06-04
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
PHASE2/PHASE3
10 participants
INTERVENTIONAL
2009-09-30
2012-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Transplantation
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
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)
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
Interventions
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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
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)
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 Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Age up to 8 year old
* Have suitable donor
Exclusion Criteria
* Bilirubin ≥ 3mg/dL
* SGPT ≥ 500 U/L
* Current severe infection
* Evidence of CNS involvement
* Morbidity such as blindness or deafness
8 Years
ALL
No
Sponsors
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Tehran University of Medical Sciences
OTHER
Responsible Party
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Locations
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Hematology-Oncology & SCT Research Center
Teharn, Tehran Province, Iran
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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HORCSCT-0906
Identifier Type: -
Identifier Source: org_study_id
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