Osteogenesis Imperfecta Type III Clinical Trial
Official title:
A Pilot Study to Assess the Safety and Feasibility of Repeated Infusions of Mesenchymal Stromal Cells (MSC) in Children With Osteogenesis Imperfecta
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.
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.
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Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Status | Clinical Trial | Phase | |
---|---|---|---|
Not yet recruiting |
NCT05559801 -
Mesenchymal Cell Therapy in Osteogenesis Imperfecta (OI)
|
Phase 1/Phase 2 | |
Completed |
NCT03118570 -
A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804
|
Phase 2 |