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.
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Recruitment rate of participants
Timeframe: Up to 24 months post-intervention
Number of participants with correctly collected data for the study outcomes
Timeframe: Up to 24 months post-intervention
Total number of visits with protocol deviation
Timeframe: Up to 24 months post-intervention
Visit attendance by participants
Timeframe: Up to 1 year post last infusion intervention
Patient retention rate
Timeframe: Up to 1 year post last infusion intervention
Patient Primary Clinical Outcome Retention Rate
Timeframe: Up to 1 year post last infusion intervention
Change in acceptability from baseline
Timeframe: Baseline, Up to 36 months post-intervention
Number of unexpected adverse events
Timeframe: Up to 1 year post last infusion intervention
Number of participants with change in vital signs from baseline
Timeframe: Baseline, 10 minutes pre-intervention, 15 minutes post-intervention, 30 minutes post-intervention, 1 hour post-intervention, 1 hour post completion of the infusion
Number of participants with changes in laboratory panels from baseline (CBC, CMP)
Timeframe: Baseline, every 4 months post-intervention
Change in number of participants with changes in radiological parameters
Timeframe: Baseline, 8, 20 and 32 months post-intervention
Change in annualized linear growth velocity
Timeframe: Baseline, every 4 months until 1 year after last infusion intervention
Change in fracture rate from baseline
Timeframe: Baseline, every 4 months post-intervention until 1 year after last infusion intervention
Change in bone mineral density from baseline
Timeframe: Baseline, yearly until 1 year after last infusion intervention
Change in bone age
Timeframe: Baseline, yearly until 1 year after last infusion intervention
Change in Vertebral Compression Fractures (VCFs)
Timeframe: Baseline, every 18 months until one year after last infusion intervention