- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00186914
Stromal Therapy of Osteodysplasia After Allogeneic Bone Marrow Transplantation
Stromal Therapy of Osteodysplasia After Allogeneic Bone Marrow Transplantation: A Phase I Study
Osteodysplasia or poorly formed bones, "brittle bones" is a genetic disease with no known proven treatments. Some forms of osteodysplasia may cause severe disability and even death.
Eligible patients were limited to those children with Osteodysplasia who had undergone a previous allogeneic bone marrow transplant at St. Jude. The study intervention involved an infusion of a specified number of ex vivo expanded stromal cells obtained from the bone marrow of the same donor from whom they received their primary transplant procedure. These bone marrow stromal cells can become bone-forming cells, called osteoblasts. Participants then received 2 infusions of ex vivo expanded, gene marked cells not less than 6 months after bone marrow transplantation. The second cell infusion occurred between 14 to 21 days after the first infusion in the absence of toxicity. The goal of the study was to evaluate the safety and toxicity of these infusions.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
- To determine whether these ex vivo expanded, gene marked marrow stromal cells will engraft in the recipient's bone, bone marrow, and/or skin.
- To begin to investigate whether high proliferative mesenchymal progenitor cells can be expanded ex vivo and retain their progenitor potential in vivo.
- To begin to investigate whether ex vivo expanded bone marrow stromal cells can ameliorate the skeletal dysplasias associated with various genetic disorders.
Study Type
Enrollment (Actual)
Phase
- Phase 1
Contacts and Locations
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
Eligibility Criteria
Ages Eligible for Study
- ADULT
- OLDER_ADULT
- CHILD
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Patients with an appropriate genetic disorder who have had an allogeneic bone marrow transplant and currently maintain complete or mixed hematopoietic chimerism
- Patient must be < or equal to 18Kg
Exclusion Criteria:
- Presence of infection
- Presence of GVHD (graft versus host disease)or the need for prophylaxis
- Concurrent participation in any investigational study that could potentially confound the interpretation of the safety parameters being investigated in this study.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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OTHER: 1
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This is a pilot study of infusions of ex vivo expanded, gene marked donor bone marrow stromal cells following allogeneic bone marrow transplantation. The study is a within patient dose escalation safety evaluation. It is believed that this patient population may benefit from these donor stromal cell infusions. As the stromal cells will be obtained from the original stem cell donor, no conditioning is required. Patients will receive two infusions of mesenchymal cells (MSC) approximately 14 to 21 days apart unless there is unacceptable toxicity after the first infusion. The first dose of cells to be given will be 1x10^6 MSC/kg and the second dose of cells will be 5x10^6 MSC/kg. The patients will be followed for approximately 28 days following the second infusion for any toxicity. Only after all six patients have safely completed both infusions will we consider 5 x 10^6 MSC/kg a safe dose of allogeneic mesenchymal stem cells for infusion.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
To evaluate the safety of the stromal cell infusion
Time Frame: June 2001
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June 2001
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Collaborators and Investigators
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start
Primary Completion (ACTUAL)
Study Completion (ACTUAL)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ESTIMATE)
Study Record Updates
Last Update Posted (ESTIMATE)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- STOD
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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