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Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

NCT01238328 · Status: UNKNOWN · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2012-06-04

No results posted yet for this study

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.

Conditions

  • Mucopolysaccharidosis

Interventions

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

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)

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

Sponsors & Collaborators

  • Tehran University of Medical Sciences

    lead OTHER

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-09-30
Primary Completion
2012-09-30
Completion
2012-12-31

Countries

  • Iran

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01238328 on ClinicalTrials.gov