Documenting long-term safety and effectiveness of palovarotene in patients with fibrodysplasia ossificans progressiva

An International Observational Registry Study to Further Describe Long-term Safety and Effectiveness of Palovarotene in Patients With Fibrodysplasia Ossificans Progressiva (FOP)

This study is trying to see how safe and effective the medication palovarotene is for children and adults with fibrodysplasia ossificans progressiva who are already using it.

Quick facts

• Study type: Observational
• Enrollment: 100 (estimated)
• Ages: 8 Years and up
• Sex: All
• Sponsor: Ipsen (industry)
• Locations: 5 sites (San Francisco, California and 4 other locations)
• Trial ID: NCT06089616 on ClinicalTrials.gov

What this trial studies

This observational registry study aims to collect and assess real-world safety data on children and adults with fibrodysplasia ossificans progressiva (FOP) who are being treated with palovarotene. Participants will already be receiving palovarotene as prescribed by their treating physician, and the study will evaluate the treatment's effectiveness on everyday activities and physical performance. The study will take place in countries where palovarotene is approved for use, focusing on the long-term outcomes of this treatment in managing FOP.

Who should consider this trial

Good fit: Ideal candidates for this study are adults and children diagnosed with fibrodysplasia ossificans progressiva who have been prescribed palovarotene.

Not a fit: Patients currently participating in a palovarotene clinical trial or those with contraindications to palovarotene may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the long-term safety and effectiveness of palovarotene, potentially improving the management of FOP.

How similar studies have performed: While this study focuses on real-world data collection, similar studies assessing the effectiveness of treatments for rare diseases have shown promising results.

Eligibility criteria

Inclusion Criteria :

* 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.

Exclusion Criteria :

* 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 this trial is running

San Francisco, California and 4 other locations

• The Regents of the University of California — San Francisco, California, United States (RECRUITING)
• The Trustees of the University of Pennsylvania, Office of Clinical Research-Legal Services, Perelman School of Medicine — Philadelphia, Pennsylvania, United States (ACTIVE_NOT_RECRUITING)
• Edmonton Clinic Health Academy (ECHA)- University of Alberta — Edmonton, Canada (RECRUITING)
• Bone Research and Education Centre — Oakville, Canada (ACTIVE_NOT_RECRUITING)
• University Health Network (UHN) - Toronto General — Toronto, Canada (RECRUITING)

Study contacts

• Study coordinator: Ipsen Clinical Study Enquiries

Conditions: Fibrodysplasia Ossificans Progressiva