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

Last updated: April 30, 2025
Sponsor: Ipsen
Overall Status: Active - Recruiting

Phase

N/A

Condition

Myositis

Treatment

N/A

Clinical Study ID

NCT06089616
CLIN-60120-453
  • Ages > 8
  • All Genders

Study Summary

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 already be receiving palovarotene as prescribed by their treating physician according to locally approved product information.

The main aim of this study will be to collect and assess real-world safety data on children and adult participants with FOP treated with palovarotene.

This study will also describe the effectiveness of this treatment, including the effect on everyday activities and physical performance

Eligibility Criteria

Inclusion

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).

Study Design

Total Participants: 70
Study Start date:
December 05, 2024
Estimated Completion Date:
January 31, 2034

Connect with a study center

  • Edmonton Clinic Health Academy (ECHA)- University of Alberta

    Edmonton,
    Canada

    Site Not Available

  • Bone Research and Education Centre

    Oakville,
    Canada

    Site Not Available

  • University Health Network (UHN) - Toronto General

    Toronto,
    Canada

    Active - Recruiting

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