Promising antibody trial aims to halt rare bone disease in kids
NCT ID NCT07559513
First seen Jun 25, 2026 · Last updated Aug 25, 2026 · Updated 3 times
Summary
This Phase 3 trial tests garetosmab, an antibody that blocks activin A, in 18 children and teens with fibrodysplasia ossificans progressiva (FOP), a rare disease where muscle and tissue turn into bone. The study will check safety, side effects, and whether the drug reduces new abnormal bone growth and flare-ups. Participants receive the drug by infusion, and the trial is not yet recruiting.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- garetosmab (a lab-made antibody that blocks activin A)
- What this could lead to
- If it works, this could slow or prevent the formation of extra bone in children with FOP, reducing painful flare-ups and preserving mobility.
- What could go wrong
- This is an early Phase 3 trial with only 18 participants, so results may not apply to everyone. The drug may cause side effects or fail to show benefit.
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 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 18 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Feb 2027
An estimate. Start dates often move.
- Expected to finish
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Dec 2029
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
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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2 to 18 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.
Key Inclusion Criteria: 1. For USA participants, age criteria are 4 to \< 18 years old, at the time of the administration of the first dose of study intervention. Non-USA participants age criteria are 2 to \< 18 years old 2. Must have a confirmation of FOP diagnosis, as described in the protocol 3. At the time of enrollment, participants must weight: 1. Cohort 1 \> 30 kg 2. Cohort 2 \> 30 kg 3. Cohort 3 ≤ 30 kg Key Exclusion Criteria: 1. Cumulative Analog Joint Involvement Scale (CAJIS) score \> 19 at the time of screening 2. Participant has significant concomitant illness or history of significant illness, as described in the protocol 3. Previous history or diagnosis of cancer 4. Ongoing significant viral or bacterial illness, within 2 weeks of the first study drug administration 5. History of severe respiratory compromise requiring oxygen, respiratory support 6. Known history of cerebral vascular malformation 7. Participants with a history of severe, non-traumatic bleeding requiring transfusion or hospitalization for hemodynamic compromise 8. Participants with a known pre-existing medical history of a bleeding diathesis, as described in the protocol NOTE: Other Protocol-defined Inclusion/Exclusion Criteria Apply
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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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