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
This trial does not accept healthy volunteersQualifiers
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
Design model
Single group
Treatments tested in this trial
Bone marrow-derived mesenchymal stromal cells (MSCs)
DrugMesenchymal stromal cells (MSCs) are cells of non-hematopoietic stromal origin that reside in bone marrow and a variety of tissues. MSCs will be prepared in a GMP Cell Production facility and administered to children age 3-10 years (at time of enrollment) with Osteogenesis Imperfecta Type III.
Treatment groups
12 Participants
are divided into 1 treatment groupGroup A: Children with OI receiving Bone marrow-derived MSCs infusionExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Recruitment rate of participants
Number of participants consented at the end of the trial
Time frame
Up to 24 months post-intervention2
Number of participants with correctly collected data for the study outcomes
Number of participants with data correctly collected for the study outcomes
Time frame
Up to 24 months post-intervention3
Total number of visits with protocol deviation
Total number of visits with protocol deviations at the end of the trial
Time frame
Up to 24 months post-intervention4
Visit attendance by participants
Percentage of visits completed by participants
Time frame
Up to 1 year post last infusion interventionSecondary outcomes
1
Number of participants with changes in bone metabolism markers
Markers including osteocalcin, BAP, CTX, PICP, Calcium/Creatinine Ratio (urine), and NTX/Creatinine Ratio (urine) will be assessed for both bone health as well as bone formation/resorption.
Time frame
Baseline, every 12 months post-intervention, and at the conclusion of the study (up to 36 months post-intervention)2
Change in Limb Deformity Modified EOS Questionnaire (LD-EOSQ-22) from baseline
This is a validated survey that will serve as a patient-reported outcome measure evaluating health status for these patients and the burden of their caregivers. The scores can range from 0-100 with a higher score correlating with a better outcome.
Time frame
Baseline, annually until 1 year after last infusion3
Change in BAMF (Brief Assessment of Motor Function) from baseline
This validated survey contains 10 point ordinal scales that outline rapid descriptions for gross and fine motor performance. The higher you score on the BAMF survey, the more developed your gross and fine motor skills are.
Time frame
Baseline, annually until 1 year after last infusion4
Change in Pediatric Orthopedic Data Collection Instrument (PODCI) from baseline
The PODCI questionnaire will used to quantify function abilities in our studied population. The scores can range from 0-100, with higher scores indicating higher levels of disability, and lower scores indicating better functioning.
Time frame
Baseline, annually until 1 year after last interventionLocations
Sponsors and contacts
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Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: