Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

Tehran University of Medical Sciences

Status and phase: Unknown; Phase 3; Phase 2

Conditions: Mucopolysaccharidosis

Treatments: Procedure: Stem Cell Transplantation; Drug: Conditioning regimen; Drug: Graft-versus-host disease (GVHD) prophylaxis

Study type: Interventional

Funder types: Other

Identifiers

NCT01238328

HORCSCT-0906

Details and patient eligibility

About

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.

Enrollment: 10 estimated patients

Sex: All

Ages: Under 8 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

---

10 participants in 1 patient group

Transplantation

Experimental group

Treatment:

Procedure: Stem Cell Transplantation

Drug: Graft-versus-host disease (GVHD) prophylaxis

Drug: Conditioning regimen