Mesenchymal Cell Therapy in Osteogenesis Imperfecta (OI)
Trial statusNot yet recruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age3-10
SponsorEmory University
About this trial
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.
Eligibility criteria
Qualifiers
1. Parent/legal guardian must be willing to sign consent forms to participate in this trial 2. Participants must be >3 years of age and <10 years of age at time of enrollment 3. Must carry mutation in either COL1A1 or COL1A2 genes and based on clinical assessment have severe Type 3 OI* 4. Must be pre-pubertal to minimize potential influence of hormonal effects on growth velocity and BMD; for children who may be entering puberty at or near upper end of this age bracket, puberty assessment will be based on clinical and laboratory findings 5. Must have received IV pamidronate therapy for at least one year prior to study initiation.
Blue/grey sclerae
Presence of prenatal fractures (on ultrasound when available)
Deformities present at birth (confirming prenatal fractures)
Disqualifiers
Lacking confirmation of mutation in either COLA1A1 or COL1A2 genes
Other pathological types of OI
Any concurrent medical issue(s) known to decrease BMD (e.g., malabsorption conditions, glucocorticoid use)
Participation in other clinical trial
Trial design
Treatments tested in this trial
- Bone marrow-derived mesenchymal stromal cells (MSCs)
Treatment groups
12 Participants
are divided into 1 treatment groupLocations
Sponsors and collaborators
Source ClinicalTrials.gov