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

Ipsen

Status: Enrolling

Conditions: Fibrodysplasia Ossificans Progressiva

Study type: Observational

Funder types: Industry

Identifiers

NCT06089616

CLIN-60120-453

Details and patient eligibility

About

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.

Enrollment: 100 estimated patients

Sex: All

Ages: 8+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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).

Trial design

100 participants in 2 patient groups

Exposed Cohort

Description:

Participants treated with palovarotene (i.e already be receiving palovarotene as prescribed by their treating physician according to locally approved product information)

Unexposed Cohort

Description:

Participants untreated with palovarotene