Fibrodysplasia Ossificans Progressiva

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Review clinical trials related to Fibrodysplasia Ossificans Progressiva. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Not yet recruiting

A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP)

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)

Participants needed: 18
Trial details
Phase: Phase 3Age: 2-18Biological sex: AllType: InterventionalSponsor: Regeneron PharmaceuticalsUpdated: Oct 8, 2026
Eligibility criteria

For USA participants, age criteria are 4 to < 18 years old, at the time of the a... [+4]

Cumulative Analog Joint Involvement Scale (CAJIS) score > 19 at the time of scre... [+8]

Status: Recruiting

A Study to Document and to Further Describe Long-term Safety and Effectiveness of Palovarotene in Participants With Fibrodysplasia Ossificans Progressiva (FOP)

The participants in this registry study will have fibrodysplasia ossificans progressiva (FOP). FOP is an ultra-rare, severely disabling disease characterized by new bone formation in areas of the body where bone is not normally present (heterotopic ossification (HO)). HO is often preceded by painful, recurrent episodes of soft tissue swelling (flare-ups). This registry study will take place in countries where the treatment, known as palovarotene, has been approved for use. Participants will either be treated with palovarotene (i.e already be receiving palovarotene as prescribed by their treating physician according to locally approved product information) or untreated with palovarotene. The main aim of this registry study will be to collect and assess real-world safety data on children and adult participants with FOP treated with palovarotene. This registry study will also describe the effectiveness of palovarotene in exposed participants, including the effect on everyday activities and physical performance. In addition, this registry study aims to descriptively compare key safety outcomes (i.e. flare-up episodes, growth outcomes, and bone fractures) between participants exposed and unexposed to palovarotene.

Participants needed: 100
Trial details
Age: 8+Biological sex: AllType: ObservationalSponsor: IpsenUpdated: Oct 1, 2026Locations: 5
Eligibility criteria

Adult or child with FOP who have been prescribed palovarotene (prior to and inde... [+1]

Currently participating in a palovarotene clinical trial; [+2]