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Purpose

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.

Condition

Eligibility

Eligible Ages
Over 8 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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

Phase
Study Type
Observational
Observational Model
Other
Time Perspective
Prospective

Arm Groups

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

Recruiting Locations

The Regents of the University of California
San Francisco, California 94103

The Trustees of the University of Pennsylvania, Office of Clinical Research-Legal Services, Perelman School of Medicine
Philadelphia, Pennsylvania 19104

More Details

Status
Recruiting
Sponsor
Ipsen

Study Contact

Ipsen Clinical Study Enquiries
see email
clinical.trials@ipsen.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.