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.
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.
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).
Adults and children with FOP who have been prescribed palovarotene, as per local label, by their treating physician for which informed consent has been obtained and maintained. Participants cannot be included if they are currently participating in a palovarotene clinical trial or an interventional clinical trial for FOP or if they have any contraindication for palovarotene (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).
Other
Prospective
Not listed
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs), whether or not they are considered as related to palovarotene
Adverse events will be coded according to MedDRA and will be classified by Preferred Term (PT) and system organ class (SOC).
Percentage of Participants With Serious and Non-serious treatment-related TEAEs
Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
Percentage of Participants With all serious TEAEs, whether or not they are considered as related to the palovarotene
Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
Percentage of Participants With nonserious TEAEs whether or not they are considered as related to the palovarotene.
Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
Change from Baseline in Cumulative Analogue Joint Involvement Scale (CAJIS) total score
The CAJIS is an objective measure of joint movement completed by the Investigator to document total joint involvement. The CAJIS total score is calculated as the sum of the scores of all joints/regions and ranges from 0 (no involvement) to 30 (maximally involved).
Change from Baseline in use of assistive devices
Assistive devices is a questionnaire to assess the assistive devices used by particpants and adaptations for their daily living.
Change from Baseline in percent of worst score for Fibrodysplasia Ossificans Progressiva-Physical Function Questionnaire (FOP-PFQ) total score
Fibrodysplasia Ossificans Progressiva-Physical Function Questionnaire (FOP-PFQ) consists of questions which are scored on a scale of 1 to 5, lower scores indicating that the participant has more difficulties.
Change from Baseline in observed and percent predicted Forced Vital Capacity (FVC)
The lung function parameters of observed FVC (liters) and percent predicted FVC are obtained by spirometry.
