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A Phase 1/2 Study to Examine the Safety and Preliminary Efficacy of Mesenchymal Stromal Cells on Linear Growth and Bone Health Parameters in Children With Type 3 Osteogenesis Imperfecta (OI)
This is a Phase 1/2 study to determine the safety and efficacy of allogeneic (third party), bone-marrow derived mesenchymal stromal cells (MSCs) for the treatment of Osteogenesis Imperfecta (OI) Type 3. It will evaluate this by looking at whether there are treatment related infusion reactions, and assessing linear growth rates and bone health, both of which are impaired in patients ages 3-10 with Osteogenesis Imperfecta Type 3. This is a single-site non-randomized clinical trial, that will take place at Children's Healthcare of Atlanta (CHOA) at Egleston and Emory Children's Center.
Details
| Lead sponsor | Emory University |
|---|---|
| Phase | Phase 1/Phase 2 |
| Status | NOT_YET_RECRUITING |
| Enrolment | 12 |
| Start date | 2027-01 |
| Completion | 2029-01 |
Conditions
- Osteogenesis Imperfecta
- Osteogenesis Imperfecta Type III
Interventions
- Bone marrow-derived mesenchymal stromal cells (MSCs)
Primary outcomes
- Recruitment rate of participants — Up to 24 months post-intervention
Number of participants consented at the end of the trial - Number of participants with correctly collected data for the study outcomes — Up to 24 months post-intervention
Number of participants with data correctly collected for the study outcomes - Total number of visits with protocol deviation — Up to 24 months post-intervention
Total number of visits with protocol deviations at the end of the trial - Visit attendance by participants — Up to 1 year post last infusion intervention
Percentage of visits completed by participants - Patient retention rate — Up to 1 year post last infusion intervention
Percentage of patients that complete the study intervention and all follow up study visits - Patient Primary Clinical Outcome Retention Rate — Up to 1 year post last infusion intervention
Percentage of participants that completed the primary clinical outcome measurements