Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

Mucopolysaccharidosis Clinical Trial

Official title:

Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01238328
• Other study ID #: HORCSCT-0906
• Status: Recruiting
• Phase: Phase 2/Phase 3
• First received: November 8, 2010
• Last updated: May 31, 2012
• Start date: September 2009
• Est. completion date: December 2012

Study information

• Verified date: May 2012
• Source: Tehran University of Medical Sciences
• Contact: Amir Ali Hamidieh, MD
• Phone: 84902645
• Email: aahamidieh[@]sina.tums.ac.ir
• Is FDA regulated: No
• Health authority: Iran: Ministry of Health
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. completion date: December 2012
• Est. primary completion date: September 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 8 Years

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis

Intervention

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
• 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: Sibling full match Other related full match Sibling or other related with 1 mismatch antigen Cord Blood Haploidentical

Locations

Country	Name	City	State
Iran, Islamic Republic of	Hematology-Oncology & SCT Research Center	Teharn	Tehran

Sponsors (1)

Lead Sponsor	Collaborator
Tehran University of Medical Sciences

Country where clinical trial is conducted

Iran, Islamic Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic Hematopoietic stem cell transplantation (HSCT)		1 year	No
Secondary	One year overall survival after allogeneic hematopoietic stem cell transplantation (HSCT)		1 year	No
Secondary	One year Progressive Free Survival after allogeneic hematopoietic stem cell transplantation (HSCT)		1 year	No
Secondary	Transplantation Related Mortality (TRM) after allogeneic hematopoietic stem cell transplantation (HSCT)		1 year	No
Secondary	Acute and chronic Graft-versus-host disease (GVHD) rate after allogeneic hematopoietic stem cell transplantation (HSCT)		1 year	No

External Links

•   Hematology-Oncology and Stem Cell Transplantation Research Center