- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01238328
Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis
May 31, 2012 updated by: Tehran University of Medical Sciences
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.
Study Overview
Status
Unknown
Conditions
Study Type
Interventional
Enrollment (Anticipated)
10
Phase
- Phase 2
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Amir Ali Hamidieh, MD
- Phone Number: +98 21 84902645
- Email: aahamidieh@sina.tums.ac.ir
Study Locations
-
-
Tehran
-
Teharn, Tehran, Iran, Islamic Republic of, 14114
- Recruiting
- Hematology-Oncology & SCT Research Center
-
Sub-Investigator:
- Kamran Alimoghaddam, MD
-
Sub-Investigator:
- Mahdi Jalili, MD
-
Contact:
- Amir Ali Hamidieh, MD
- Phone Number: +9821 84902645
- Email: aahamidieh@sina.tums.ac.ir
-
Principal Investigator:
- Amir Ali Hmidieh, MD
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
No older than 8 years (CHILD)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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 Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NON_RANDOMIZED
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: Transplantation
|
Patients undergoing Hematopoietic Stem Cell Transplantation from one of below source:
Other Names:
For sibling full match:
For other related full match, sibling or other related with one antigen mismatch and umbilical cord blood:
For haploidentical:
Other Names:
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Overall Survival and Progressive Free Survival in patient with Mucopolysaccharidosis who receive allogeneic Hematopoietic stem cell transplantation (HSCT)
Time Frame: 1 year
|
1 year
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
One year overall survival after allogeneic hematopoietic stem cell transplantation (HSCT)
Time Frame: 1 year
|
1 year
|
One year Progressive Free Survival after allogeneic hematopoietic stem cell transplantation (HSCT)
Time Frame: 1 year
|
1 year
|
Transplantation Related Mortality (TRM) after allogeneic hematopoietic stem cell transplantation (HSCT)
Time Frame: 1 year
|
1 year
|
Acute and chronic Graft-versus-host disease (GVHD) rate after allogeneic hematopoietic stem cell transplantation (HSCT)
Time Frame: 1 year
|
1 year
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
September 1, 2009
Primary Completion (ANTICIPATED)
September 1, 2012
Study Completion (ANTICIPATED)
December 1, 2012
Study Registration Dates
First Submitted
November 8, 2010
First Submitted That Met QC Criteria
November 8, 2010
First Posted (ESTIMATE)
November 10, 2010
Study Record Updates
Last Update Posted (ESTIMATE)
June 4, 2012
Last Update Submitted That Met QC Criteria
May 31, 2012
Last Verified
May 1, 2012
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Metabolic Diseases
- Genetic Diseases, Inborn
- Connective Tissue Diseases
- Carbohydrate Metabolism, Inborn Errors
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Mucinoses
- Mucopolysaccharidoses
- Physiological Effects of Drugs
- Immunosuppressive Agents
- Immunologic Factors
- Thymoglobulin
- Antilymphocyte Serum
Other Study ID Numbers
- HORCSCT-0906
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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