Marrow Mesenchymal Cell Therapy for Osteogenesis Imperfecta: A Pilot Study
March 3, 2015 updated by: St. Jude Children's Research Hospital
Osteogenesis imperfecta (OI) is a genetic disease for which there is currently no known cure.
OI causes the osteoblasts (bone-forming cells in the body) to grow poorly, which slows the growth of children with the disease and causes their bones to bend and break easily.
Some forms of osteogenesis imperfecta may cause severe disability and even death.
In previous research studies performed at St. Jude, it was found that children treated with bone marrow transplant (infusion of healthy immature blood-forming cells) began to grow faster, had more minerals (material that helps make the bones strong) in their bones, and broke their bones less often than before the bone marrow transplant.
Several months after the bone marrow transplant however, body growth once again began to slow down.
In this research study, children with osteogenesis imperfecta will receive another infusion of bone marrow cells but without any chemotherapy.
The marrow cells will come from the same bone marrow donor as their previous bone marrow transplant.
It is hoped that by removing the CD3+ cells (a type of white blood cells that attack other cells that are not like themselves) from the donated bone marrow, the subject's body will be infused quite safely and that body growth and bone strength will increase.
The CD3+ cells will be removed from the donor bone marrow by use of a machine called the CliniMACS System.
This machine has not been approved for use in the United States by the Food and Drug Administration (FDA).
The use of this device is considered experimental.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
9
Phase
- Not Applicable
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Tennessee
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Memphis, Tennessee, United States, 38105
- St. Jude Children's Research Hospital
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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
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Must have been previously enrolled on TOIT protocol at St. Jude Children's Research Hospital
- Must have original bone marrow donor available and willing to participate as a donor
- Normal liver function
- Hemoglobin >10gm/dl
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: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
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To find out the effects (good and bad) of bone marrow cell infusions using donor bone marrow that has had CD3+ cells removed
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To find out if there is any effect on the growth rate of children with osteogenesis imperfecta who receive donor bone marrow which has had CD3+ cells removed
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To find out if there is any effect on the total bone mineral content of children with OI
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who receive donor bone marrow which has had CD3+ cells removed
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Secondary Outcome Measures
Outcome Measure |
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To find out the effect of the CD3 washed-out marrow cell therapy on the growth rate of the children
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Investigators
- Principal Investigator: Gregory Hale, M.D., St. Jude Children's Research Hospital
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
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
March 1, 2004
Primary Completion (Actual)
August 1, 2007
Study Completion (Actual)
August 1, 2007
Study Registration Dates
First Submitted
September 12, 2005
First Submitted That Met QC Criteria
September 12, 2005
First Posted (Estimate)
September 16, 2005
Study Record Updates
Last Update Posted (Estimate)
March 4, 2015
Last Update Submitted That Met QC Criteria
March 3, 2015
Last Verified
May 1, 2007
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- STOD2
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.