Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis

NCT01238328 | Unknown Phase 2 DMC

• 1 other identifier: HORCSCT-0906
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Mucopolysaccharidosis

Keywords

Mucopolysaccharidosis; HSCT

Outcome Measures

Primary Outcomes (1)

• Overall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic Hematopoietic stem cell transplantation (HSCT)

  1 year

Secondary Outcomes (4)

• 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

Study Arms (1)

Transplantation

EXPERIMENTAL

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

Interventions

Conditioning regimen DRUG

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

Also known as: Endoxan, Antithymocyte globulin (ATG) rabbit : Thymoglobulin, Antithymocyte globulin (ATG) horse : Atgam

Transplantation

Graft-versus-host disease (GVHD) prophylaxis DRUG

* 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)

Also known as: MTX

Transplantation

Stem Cell Transplantation PROCEDURE

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

Also known as: HSCT

Transplantation

Eligibility Criteria

• Age: Up to 8 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Diagnosis of Mucopolysaccharidosis type 1, 6 and 7 confirm by leukocyte enzyme assay
• Age up to 8 year old
• Have suitable donor

You may not qualify if:

• 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

Sponsors & Collaborators

• Tehran University of Medical Sciences: lead

Study Sites (1)

Hematology-Oncology & SCT Research Center

Teharn, Tehran Province, 14114, Iran

RECRUITING

MeSH Terms

Conditions

Mucopolysaccharidoses

Interventions

Transplantation Conditioning; Cyclophosphamide; Antilymphocyte Serum; Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Immunosuppression Therapy; Immunotherapy; Immunomodulation; Biological Therapy; Therapeutics; Immunologic Techniques; Investigative Techniques; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures; Cell Transplantation; Cell- and Tissue-Based Therapy; Transplantation; Surgical Procedures, Operative

Central Study Contacts

Amir Ali Hamidieh, MD

CONTACT

84902645; aahamidieh@sina.tums.ac.ir

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 8, 2010
• First Posted: November 10, 2010
• Study Start: September 1, 2009
• Primary Completion: September 1, 2012
• Study Completion: December 1, 2012
• Last Updated: June 4, 2012

Record last verified: 2012-05

Locations

IR (1)