Repeated Infusions of Mesenchymal Stromal Cells in Children With Osteogenesis Imperfecta (STOD3)
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Purpose
This is a study to evaluate the safety and effectiveness of repeated Mesenchymal Stromal Cells (MSC) infusions to patients with Type II or III osteogenesis imperfecta (OI).
Participants will receive MSC infusions approximately every 4 months to complete a total of 6 infusions over 20 months. Participants will be followed for 4 months post their last MSC infusion.
| Condition | Intervention | Phase |
|---|---|---|
|
Osteogenesis Imperfecta Type II Osteogenesis Imperfecta Type III |
Biological: Mesenchymal Stromal Cells |
Phase 1 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Pilot Study to Assess the Safety and Feasibility of Repeated Infusions of Mesenchymal Stromal Cells (MSC) in Children With Osteogenesis Imperfecta |
- To determine the safety or repeated infusions of donor-derived and MSCs in subjects with severe osteogenesis imperfecta > 5years after an allogeneic bone marrow transplant and no prior bone marrow transplant. [ Time Frame: Completion of study ] [ Designated as safety issue: Yes ]
- To determine if MSCs elicit an immune response after repeated infusions. [ Time Frame: Completion of study ] [ Designated as safety issue: Yes ]
- To determine the change in clinical course (growth, bone mineral content, fracture rate, development/activities) of subjects after experimental MSC intervention therapy as compared with each subject's own pre-MSC intervention therapy. [ Time Frame: Completion of Study ] [ Designated as safety issue: Yes ]
| Estimated Enrollment: | 12 |
| Study Start Date: | February 2010 |
| Estimated Study Completion Date: | September 2013 |
| Estimated Primary Completion Date: | September 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Active Comparator: Stratum A
Subjects with a diagnosis of Type II or Type III osteogenesis imperfecta who have previously undergone a bone marrow transplant.
|
Biological: Mesenchymal Stromal Cells
Both cohorts will receive multiple infusions of ex-vivo expanded MSCs. Stratum A may use previously harvested and cryopreserved bone marrow mononuclear cells from original BMT donor or freshly harvested or cryopreserved bone marrow mononuclear cells obtained from a haploidentical healthy parent or sibling. Stratum B will only receive freshly harvested or cryopreserved bone marrow
Other Names:
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Active Comparator: Stratum B
Subjects with Type II or III osteogenesis imperfecta who have not undergone a bone marrow transplant
|
Biological: Mesenchymal Stromal Cells
Both cohorts will receive multiple infusions of ex-vivo expanded MSCs. Stratum A may use previously harvested and cryopreserved bone marrow mononuclear cells from original BMT donor or freshly harvested or cryopreserved bone marrow mononuclear cells obtained from a haploidentical healthy parent or sibling. Stratum B will only receive freshly harvested or cryopreserved bone marrow
Other Names:
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Detailed Description:
This is a pilot study to evaluate the safety and efficacy of repeated MSC infusions to subjects with OI. This study will evaluate subjects on two separate strata. Stratum A will include subjects with a diagnosis of Type II or Type III osteogenesis imperfecta who have previously undergone a bone marrow transplant. Stratum A may use previously harvested and cryopreserved bone marrow mononuclear cells from original BMT donor or freshly harvested or cryopreserved bone marrow mononuclear cells obtained from a haploidentical healthy parent or sibling. Stratum B will include subjects with Type II or III osteogenesis imperfecta who have not undergone a bone marrow transplant. Stratum B will only receive freshly harvested or cryopreserved bone marrow mononuclear cells from a haploidentical healthy parent or sibling.
Participants will receive MSC infusions approximately every 4 months to complete a total of 6 infusions over 20 months. Participants will be followed for 12 months post their last MSC infusion.
Eligibility| Ages Eligible for Study: | up to 19 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Subjects less than or equal to 19 years of age at the time of enrollment
- Children with a diagnosis of severe of Type II or III osteogenesis imperfecta
- Parent or sibling greater than or equal to 18 years of age, donor willing to or has already undergone HLA typing, and willing and able to provide bone marrow
- BMT greater than 5 years ago for Stratum A
Exclusion Criteria:
- Dependent on supplemental oxygen
- Concurrent Infection
Contacts and Locations| United States, Pennsylvania | |
| The Children's Hospital of Philadelphia | |
| Philadelphia, Pennsylvania, United States, 19105 | |
| Principal Investigator: | Ed Horwitz, MD | Children's Hospital of Philadelphia |
More Information
Publications:
| Responsible Party: | Edwin Horwitz, Attending Physician, Children's Hospital of Philadelphia |
| ClinicalTrials.gov Identifier: | NCT01061099 History of Changes |
| Other Study ID Numbers: | 2008-4-5947 |
| Study First Received: | February 1, 2010 |
| Last Updated: | August 24, 2012 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Children's Hospital of Philadelphia:
|
Type II or Type III Osteogenesis imperfecta OI Mesenchymal Stromal Cells MSC |
Additional relevant MeSH terms:
|
Osteogenesis Imperfecta Osteochondrodysplasias Bone Diseases, Developmental Bone Diseases |
Musculoskeletal Diseases Genetic Diseases, Inborn Collagen Diseases Connective Tissue Diseases |
ClinicalTrials.gov processed this record on May 23, 2013