Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

Phase 2

• Conditions: Mucopolysaccharidosis
• Interventions: Drug: Conditioning regimen; Drug: Graft-versus-host disease (GVHD) prophylaxis; Procedure: Stem Cell Transplantation

• Registration Number: NCT01238328
• Lead Sponsor: Tehran University of Medical Sciences

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2009-09
• Study First Submitted: 2010-11-08
• Study First Submitted QC: 2010-11-08
• Study First Posted: 2010-11-10
• Status Verified: 2012-05
• Last Update Submitted: 2012-05-31
• Last Update Posted: 2012-06-04
• Primary Completion: 2012-09
• Study Completion: 2012-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 10

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Transplantation	Conditioning regimen	-
Transplantation	Graft-versus-host disease (GVHD) prophylaxis	-
Transplantation	Stem Cell Transplantation	-

Primary Outcome Measures

Name	Time	Method
Overall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic Hematopoietic stem cell transplantation (HSCT)	1 year

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

Hematology-Oncology & SCT Research Center; 🇮🇷 Teharn, Tehran, Iran, Islamic Republic of