Could donor stem cells help kids with brittle bones grow taller and stronger?
NCT ID NCT05559801
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tests whether infusions of donor bone marrow stem cells (MSCs) are safe and can improve growth and bone health in 12 children aged 3–10 with severe osteogenesis imperfecta type 3. The study will monitor side effects, growth rates, fracture healing, and bone density over one year. Because it is a small, non-randomized safety trial, results will be preliminary and may not lead to a proven treatment.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- mesenchymal stromal cells (MSCs) from donor bone marrow
- What this could lead to
- If it works, this could point toward a treatment that helps children with severe brittle bone disease grow taller and have stronger bones.
- What could go wrong
- This is a very early, small trial (12 children) focused mainly on safety. It may not show clear benefit, and there are risks like infusion reactions or unknown side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 12 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Jan 2027
An estimate. Start dates often move.
- Expected to finish
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Jan 2029
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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3 to 10 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 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 Exclusion Criteria: 1. Lacking confirmation of mutation in either COLA1A1 or COL1A2 genes 2. Other pathological types of OI 3. Any concurrent medical issue(s) known to decrease BMD (e.g., malabsorption conditions, glucocorticoid use) 4. Participation in other clinical trial 5. Vitamin D deficiency (\<20 ng/dL) despite treatment 6. 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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
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Other studies related to the condition(s) this trial covers.
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