Osteogenesis ImperfectaPhase 1/2NCT05559801
Mesenchymal Cell Therapy in Osteogenesis Imperfecta (OI)
A Phase 1/2 study testing Bone marrow-derived mesenchymal stromal cells (MSCs) in people with Osteogenesis Imperfecta and Osteogenesis Imperfecta Type III, run by Emory University.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 3 to 10 years
- Conditions the study is for
- Osteogenesis Imperfecta, Osteogenesis Imperfecta Type III
- What is being tested?
- Bone marrow-derived mesenchymal stromal cells (MSCs)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- No sites listed yet
- Study dates
- Jan 2027to Jan 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
- Type 3 OI will be confirmed with an Invitae Skeletal Dysplasia test, and clinical assessment including:
- Blue/grey sclerae
- Presence of prenatal fractures (on ultrasound when available)
- Deformities present at birth (confirming prenatal fractures)
- Severity of fractures and progressive deformities although no absolute 'number' of fractures is available
Who cannot join
- 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
- Vitamin D deficiency (<20 ng/dL) despite treatment
- Clinically significant thrombocytopenia as defined by a platelet count of < 150,000x103/microliter ; anemia as defined by hemoglobin < 5th percentile for age (<11.5g/dL); neutropenia as defined by absolute neutrophil count < 1.5 x103/microliter; or elevations in the white blood cell count as defined by 3-6 year old-WBC > 15.5WBC x 103/microliter; 6-9 year old WBC >13.5 x103/microliter (Flerlage 2015)
- 8. PRA screening positive for anti-HLA antibodies 9. Elevated LFT's greater than 2 times the upper limit of normal 10. Other genetic disorders 11. Other skeletal dysplasia disorders 12. Other primary or secondary bone disorders 13. History of acute or chronic infections 14. History of cancer 15. History of thrombosis or prothrombotic disorders 16. History of heart disease 17. History of diabetes 18. History of strokes 19. History of vascular conditions 20. History of lung disease
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: 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.
Read the detailed description
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.
MSCs will be infused through IV every 4 months for 6 total infusions. There will be a baseline visit before MSC therapy is initiated, and there will be a follow up visits every 4 months for 1 year after the final MSC infusion. These infusions will take place in between pamidronate infusions (i.e. 2 months after the last pamidronate infusion, and 2 months before the next one). Pamidronate aids in treatment of bone pain and bone mineral density but does not correct the underlying defect nor does it show substantial improvements in linear growth.
After the MSC infusions, patients will be provided an overnight at Ronald McDonald House, or a hospital affiliated hotel so they can be near to the hospital in case of any unanticipated effects, and for a follow up visit the next morning.
Labs will be collected during every visit to look at bone metabolism. Limb and bone age x-rays, pQCT scans, and dual-energy x-ray absorptiometry (DXA) scans will be completed annually, while spine films will be completed every 18 months. These images will directly examine bone health. Body measurements will be taken every visit as well to assess linear growth. The patients' parents will complete events diaries and submit them each infusion day to evaluate fractures that occurred between visits. Patients and their parents will also complete quality of life surveys once a year.
There will be financial compensation for each study visit. Subjects will also be provided a free lunch on days when radiology visits occur and will be reimbursed for parking.
Subjects will be identified and recruited mainly through Children's endocrinology clinic. There will also be advertisements to the OI foundation, and neighboring pediatric hospitals with OI programs such as University of Alabama at Birmingham, and Vanderbilt. If identified as eligible to participate, the study team will seek approval by the subjects' primary endocrinologist. The consent process will then take place in person during a baseline visit.
There is an optional part of the study that involves donation of a bone fragment that is taken out during a routine surgical rodding procedure. This piece of bone is removed and discarded, if it is not donated, making it minimal risk. Bone sample donation will be available to OI Type 3 subjects receiving MSCs, and to OI Type 3 subjects who are not receiving MSCs but want to participate in research.
Leftover blood samples, and bone fragments may be stored for future research by the sponsor of this study.
The purpose of this study is to help doctors and scientists learn if serial MSC infusions will safely and effectively aid in growth, bone health, and ultimately improve motoric function and quality of life in this population.
- Being tested
- Bone marrow-derived mesenchymal stromal cells (MSCs)
- Medical subject headings
- Osteogenesis Imperfecta, Osteogenesis imperfecta, type 3
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bone marrow-derived mesenchymal stromal cells (MSCs)Drug
Also called MSC infusions
Mesenchymal 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.
| Study group | Role | What they receive |
|---|---|---|
| Children with OI receiving Bone marrow-derived MSCs infusion | Experimental | Intravenously-infused allogeneic, bone marrow-derived mesenchymal stromal cells (MSCs) in children with Osteogenesis Imperfecta Type III that will be infused at 0 months, 4 months, 8 months, 12 months, 16 months, and 20 months, after enrollment. Receives: Drug: Bone marrow-derived mesenchymal stromal cells (MSCs) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recruitment rate of participants
Number of participants consented at the end of the trial
Measured: Up to 24 months post-intervention
Number of participants with correctly collected data for the study outcomes
Number of participants with data correctly collected for the study outcomes
Measured: Up to 24 months post-intervention
Total number of visits with protocol deviation
Total number of visits with protocol deviations at the end of the trial
Measured: Up to 24 months post-intervention
Visit attendance by participants
Percentage of visits completed by participants
Measured: Up to 1 year post last infusion intervention
Patient retention rate
Percentage of patients that complete the study intervention and all follow up study visits
Measured: Up to 1 year post last infusion intervention
Patient Primary Clinical Outcome Retention Rate
Percentage of participants that completed the primary clinical outcome measurements
Measured: Up to 1 year post last infusion intervention
Change in acceptability from baseline
Acceptability will be evaluated with an end of study structured, verbal interview. This interview will consist of a mix of closed ended (scaled questions from 1[worst]-5 [best]) and open-ended questions giving subjective measurements. Open-ended questions will address overall experience during the study and prompt expression of thoughts or perspectives after completing the study. The total possible score range is 18 to 90. Higher score correlates with better outcome.
Measured: Baseline, Up to 36 months post-intervention
Number of unexpected adverse events
Unexpected adverse events that occur from first study intervention to one year after last infusion.
Measured: Up to 1 year post last infusion intervention
Number of participants with change in vital signs from baseline
Number of participants with blood pressure, oxygen saturation, heart rate, respiratory rate, and temperature below or above each participants accepted mean reference range.
Measured: 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)
Number of patients with changes in CBC or CMP values from baseline CBC or CMP values. Any evidence of thrombocytopenia, anemia, neutropenia, or elevations in white blood counts will require consideration of stopping or pausing infusions by the PI. Slight changes in the ALT, AST, and calcium values are expected due to standard medication uses and will be monitored by PI.
Measured: Baseline, every 4 months post-intervention
Change in number of participants with changes in radiological parameters
Number of participants with changes in radiological parameters (xrays and DXA scan) from baseline will be monitored during the study follow up time. DXA scans and x-rays used to assess for any semi-triggers that may require considerations of stopping or pausing infusions.
Measured: Baseline, 8, 20 and 32 months post-intervention
Change in annualized linear growth velocity
This study will assess the annualized linear growth velocity at baseline and during cell therapy protocol in children with Type 3 OI (3-10 years of age and pre-pubertal at time of enrollment). Growth charts will be generated using growth measurements obtained every 4 months.
Growth charts will be referenced against sex- and age-specific Centers for Disease Control and Prevention (CDC) childhood OI growth reference charts.
Measured: Baseline, every 4 months until 1 year after last infusion intervention
Change in fracture rate from baseline
Overt and covert fracture rate will be measured in each child at baseline and at regular intervals during MSC therapy by using a caretaker events diary, limb films, and spine films to tally more objectively new and healing fractures.
Measured: Baseline, every 4 months post-intervention until 1 year after last infusion intervention
Change in bone mineral density from baseline
Bone mineral density will be determined by DXA (dual-energy absorptiometry) analysis for TBLH (total body less head), spine and distal radius sites.
Measured: Baseline, yearly until 1 year after last infusion intervention
Change in bone age
A bone age assessment is done using left hand radiograph. Bone age will be followed yearly in children of 8 years of age and older at time of enrollment in conjunction with exam and laboratory assessments to determine potential effects of puberty on bone density.
Measured: Baseline, yearly until 1 year after last infusion intervention
Change in Vertebral Compression Fractures (VCFs)
Due to abnormal collagen and the associated decrease in BMD, VCFs are common in Type 3 OI. These can be 'silent' and/or associated with back pain. PA TL film and LAT TL film will be obtained to assess VCF's that cannot be assessed by DXA in children. Spine radiographs are within standard of care for treatment of OI.
Measured: Baseline, every 18 months until one year after last infusion intervention
Secondary outcomes (4)
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.
Measured: Baseline, every 12 months post-intervention, and at the conclusion of the study (up to 36 months post-intervention)
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.
Measured: Baseline, annually until 1 year after last infusion
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.
Measured: Baseline, annually until 1 year after last infusion
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.
Measured: Baseline, annually until 1 year after last intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2022
- Study startJan 2027 (estimated)
- Primary completionJan 2029 (estimated)
- Study completionJan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Osteogenesis ImperfectaStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Doris Fadoju, Emory University
- Investigators
- Doris Fadoju, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05559801
- Sponsor's protocol ID
- STUDY00003434
- Other IDs
- 2025P010634
- Status last verified
- May 2026
- First submitted
- 8 Sep 2022
- Last updated
- 8 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All of the individual participant data collected during the trial, after de-identification and publication.
Study at a glance
From the registry record, last updated by the sponsor on 8 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT05559801
- Acronym / sponsor ID
- STUDY00003434
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Study start
- Jan 2027 (estimated)
- Primary completion
- Jan 2029 (estimated)
- Study completion
- Jan 2029 (estimated)
- First posted
- 29 Sep 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
MSCs will be infused through IV every 4 months for 6 total infusions. There will be a baseline visit before MSC therapy is initiated, and there will be a follow up visits every 4 months for 1 year after the final MSC infusion. These infusions will take place in between pamidronate infusions (i.e. 2 months after the last pamidronate infusion, and 2 months before the next one). Pamidronate aids in treatment of bone pain and bone mineral density but does not correct the underlying defect nor does it show substantial improvements in linear growth.
After the MSC infusions, patients will be provided an overnight at Ronald McDonald House, or a hospital affiliated hotel so they can be near to the hospital in case of any unanticipated effects, and for a follow up visit the next morning.
Labs will be collected during every visit to look at bone metabolism. Limb and bone age x-rays, pQCT scans, and dual-energy x-ray absorptiometry (DXA) scans will be completed annually, while spine films will be completed every 18 months. These images will directly examine bone health. Body measurements will be taken every visit as well to assess linear growth. The patients' parents will complete events diaries and submit them each infusion day to evaluate fractures that occurred between visits. Patients and their parents will also complete quality of life surveys once a year.
There will be financial compensation for each study visit. Subjects will also be provided a free lunch on days when radiology visits occur and will be reimbursed for parking.
Subjects will be identified and recruited mainly through Children's endocrinology clinic. There will also be advertisements to the OI foundation, and neighboring pediatric hospitals with OI programs such as University of Alabama at Birmingham, and Vanderbilt. If identified as eligible to participate, the study team will seek approval by the subjects' primary endocrinologist. The consent process will then take place in person during a baseline visit.
There is an optional part of the study that involves donation of a bone fragment that is taken out during a routine surgical rodding procedure. This piece of bone is removed and discarded, if it is not donated, making it minimal risk. Bone sample donation will be available to OI Type 3 subjects receiving MSCs, and to OI Type 3 subjects who are not receiving MSCs but want to participate in research.
Leftover blood samples, and bone fragments may be stored for future research by the sponsor of this study.
The purpose of this study is to help doctors and scientists learn if serial MSC infusions will safely and effectively aid in growth, bone health, and ultimately improve motoric function and quality of life in this population.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Children with OI receiving Bone marrow-derived MSCs infusion | Experimental | Drug: Bone marrow-derived mesenchymal stromal cells (MSCs)Intravenously-infused allogeneic, bone marrow-derived mesenchymal stromal cells (MSCs) in children with Osteogenesis Imperfecta Type III that will be infused at 0 months, 4 months, 8 months, 12 months, 16 months, and 20 months, after enrollment. |
Who may qualify
- Ages
- 3 to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (7 listed)
- 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
- Vitamin D deficiency (<20 ng/dL) despite treatment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Bone marrow-derived mesenchymal stromal cells (MSCs)Also: MSC infusionsMesenchymal 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. | Drug | Children with OI receiving Bone marrow-derived MSCs infusion |
Primary outcome
Recruitment rate of participants
Time frame: 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
Time frame: Up to 24 months post-intervention
Number of participants with data correctly collected for the study outcomes
Total number of visits with protocol deviation
Time frame: Up to 24 months post-intervention
Total number of visits with protocol deviations at the end of the trial
Visit attendance by participants
Time frame: Up to 1 year post last infusion intervention
Percentage of visits completed by participants
Patient retention rate
Time frame: 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
Time frame: Up to 1 year post last infusion intervention
Percentage of participants that completed the primary clinical outcome measurements
Change in acceptability from baseline
Time frame: Baseline, Up to 36 months post-intervention
Acceptability will be evaluated with an end of study structured, verbal interview. This interview will consist of a mix of closed ended (scaled questions from 1[worst]-5 [best]) and open-ended questions giving subjective measurements. Open-ended questions will address overall experience during the study and prompt expression of thoughts or perspectives after completing the study. The total possible score range is 18 to 90. Higher score correlates with better outcome.
Number of unexpected adverse events
Time frame: Up to 1 year post last infusion intervention
Unexpected adverse events that occur from first study intervention to one year after last infusion.
Number of participants with change in vital signs from baseline
Time frame: 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 blood pressure, oxygen saturation, heart rate, respiratory rate, and temperature below or above each participants accepted mean reference range.
Number of participants with changes in laboratory panels from baseline (CBC, CMP)
Time frame: Baseline, every 4 months post-intervention
Number of patients with changes in CBC or CMP values from baseline CBC or CMP values. Any evidence of thrombocytopenia, anemia, neutropenia, or elevations in white blood counts will require consideration of stopping or pausing infusions by the PI. Slight changes in the ALT, AST, and calcium values are expected due to standard medication uses and will be monitored by PI.
Change in number of participants with changes in radiological parameters
Time frame: Baseline, 8, 20 and 32 months post-intervention
Number of participants with changes in radiological parameters (xrays and DXA scan) from baseline will be monitored during the study follow up time. DXA scans and x-rays used to assess for any semi-triggers that may require considerations of stopping or pausing infusions.
Change in annualized linear growth velocity
Time frame: Baseline, every 4 months until 1 year after last infusion intervention
This study will assess the annualized linear growth velocity at baseline and during cell therapy protocol in children with Type 3 OI (3-10 years of age and pre-pubertal at time of enrollment). Growth charts will be generated using growth measurements obtained every 4 months. Growth charts will be referenced against sex- and age-specific Centers for Disease Control and Prevention (CDC) childhood OI growth reference charts.
Change in fracture rate from baseline
Time frame: Baseline, every 4 months post-intervention until 1 year after last infusion intervention
Overt and covert fracture rate will be measured in each child at baseline and at regular intervals during MSC therapy by using a caretaker events diary, limb films, and spine films to tally more objectively new and healing fractures.
Change in bone mineral density from baseline
Time frame: Baseline, yearly until 1 year after last infusion intervention
Bone mineral density will be determined by DXA (dual-energy absorptiometry) analysis for TBLH (total body less head), spine and distal radius sites.
Change in bone age
Time frame: Baseline, yearly until 1 year after last infusion intervention
A bone age assessment is done using left hand radiograph. Bone age will be followed yearly in children of 8 years of age and older at time of enrollment in conjunction with exam and laboratory assessments to determine potential effects of puberty on bone density.
Change in Vertebral Compression Fractures (VCFs)
Time frame: Baseline, every 18 months until one year after last infusion intervention
Due to abnormal collagen and the associated decrease in BMD, VCFs are common in Type 3 OI. These can be 'silent' and/or associated with back pain. PA TL film and LAT TL film will be obtained to assess VCF's that cannot be assessed by DXA in children. Spine radiographs are within standard of care for treatment of OI.
4 secondary outcomes
Number of participants with changes in bone metabolism markers
Time frame: Baseline, every 12 months post-intervention, and at the conclusion of the study (up to 36 months post-intervention)
Change in Limb Deformity Modified EOS Questionnaire (LD-EOSQ-22) from baseline
Time frame: Baseline, annually until 1 year after last infusion
Change in BAMF (Brief Assessment of Motor Function) from baseline
Time frame: Baseline, annually until 1 year after last infusion
Change in Pediatric Orthopedic Data Collection Instrument (PODCI) from baseline
Time frame: Baseline, annually until 1 year after last intervention
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 May 2026.
- Individual participant dataSharing planned: Yes. All of the individual participant data collected during the trial, after de-identification and publication.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Doris Fadoju, MD principal investigator
Emory University
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Bone marrow-derived mesenchymal stromal cells (MSCs) in people with Osteogenesis Imperfecta and Osteogenesis Imperfecta Type III, run by Emory University.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 8 May 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Doris Fadoju, MD (404-712-9839, doris.o.fadoju@emory.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Recruitment rate of participants (time frame: Up to 24 months post-intervention). The study lists 16 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 May 2026.