A Study to Document and to Further Describe Long-term Safety and Effectiveness of Palovarotene in Participants With Fibrodysplasia Ossificans Progressiva (FOP)
Recruiting now
Conditions studied: Fibrodysplasia Ossificans Progressiva
In brief
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.
Key facts
- Study ID
- NCT06089616
- Run by
- Ipsen
- People needed
- 100
- Starts
- 2024-12-05
- Expected to finish
- 2035-12-05
- Last updated by the study team
- 2026-07-02
Who can join
Age: 8 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Adult or child with FOP who have been prescribed palovarotene (prior to and independently of the decision to enroll the patient in this registry study and as per local label) by their treating physician according to the locally approved product information;
- Signed informed consent as per local regulations must be obtained and maintained. Consent/assent from the participant should be obtained as appropriate before any registry study data collection are conducted. If applicable, parents or legally authorized representatives must give signed informed consent.
You may not qualify if…
- Currently participating in a palovarotene clinical trial;
- Currently participating in any interventional clinical trial for FOP;
- Have any contraindication to palovarotene as per the locally approved label (except for pregnant women who have previously received and discontinued palovarotene at any time during the pregnancy and who will be included for safety follow-up).
Where it is running
- The Regents of the University of California — San Francisco, California, United States (enrolling)
- The Trustees of the University of Pennsylvania, Office of Clinical Research-Legal Services, Perelman School of Medicine — Philadelphia, Pennsylvania, United States (enrolling)
- Edmonton Clinic Health Academy (ECHA)- University of Alberta — Edmonton, Canada (enrolling)
- University Health Network (UHN) - Toronto General — Toronto, Canada (enrolling)
- Bone Research and Education Centre — Oakville, Canada
Full record on ClinicalTrials.gov
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