A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP)
Key facts
- Phase
- Phase 3
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 2 and 18 years old?
Source: NCT07559513, who can take part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Regeneron Pharmaceuticals
- Trial code
- NCT07559513EU 2024-518415-19-01
- Record updated
- 12 November 2026
- Original description
This study is researching an experimental drug called garetosmab, referred to as "study drug". The study is focused on children and adolescent participants with FOP. For United States: Ages 4 to less than 18 years; for non-US sites only: Ages 2 to less than 18 years.Read full original descriptionShow less
Phase 3 Evaluation of the Safety, Pharmacokinetics, and Efficacy of Garetosmab (Anti-Activin A Monoclonal Antibody) in Children and Adolescents With Fibrodysplasia Ossificans Progressiva
This study is researching an experimental drug called garetosmab, referred to as "study drug". The study is focused on children and adolescent participants with FOP. For United States: Ages 4 to less than 18 years; for non-US sites only: Ages 2 to less than 18 years.
The aim of the study is to see how safe, tolerable, and effective the study drug is.
The study is looking at several other research questions, including:
* What side effects may happen from taking the study drug * How much study drug is in the blood at different times * Whether the body makes antibodies against the study drug (which could make the study drug less effective or could lead to side effects)
Inclusion: 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; Must have a confirmation of FOP diagnosis, as described in the protocol; At the time of enrollment, participants must weight:; Cohort 1: more than 30 kg (or 66.1 pounds); Cohort 2: more than 30 kg (or 66.1 pounds); Cohort 3: less than or equal to 30 kg (or 66.1 pounds).
Exclusion: Participant has significant concomitant illness or history of significant illness, as described in the protocol; Previous history or diagnosis of cancer; Participants who have used palovarotene in the last 30 days or within 5 half-lives of the therapy, whichever is longer from the day of randomization; NOTE: Other Protocol-defined Inclusion/Exclusion Criteria Apply; Ongoing significant viral or bacterial illness, within 2 weeks of the first study drug administration; History of severe respiratory compromise requiring oxygen, respiratory support; Known history of cerebral vascular malformation; Participants with a history of severe, non-traumatic bleeding requiring transfusion or hospitalization for hemodynamic compromise; Participants with a known pre-existing medical history of a bleeding diathesis, as described in the protocol; Cumulative Analog Joint Involvement Scale (CAJIS) score > 19 at the time of screening.