FOPal: A Study to Document and to Further Describe Long-term Safety and Effectiveness of Palovarotene in Participants With Fibrodysplasia Ossificans Progressiva (FOP)
Study Details
Study Description
Brief 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
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Study Design
Outcome Measures
Primary Outcome Measures
- Percentage of Participants With Treatment-emergent Adverse Events (TEAEs), whether or not they are considered as related to palovarotene [From Baseline up to 30 days after the last palovarotene dose.]
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 [From Baseline up to 30 days after the last palovarotene dose.]
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 [From Baseline up to 30 days after the last palovarotene dose.]
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. [From Baseline up to 30 days after the last palovarotene dose.]
Adverse events will be coded according to MedDRA and will be classified by PT and SOC.
Secondary Outcome Measures
- Change from Baseline in Cumulative Analogue Joint Involvement Scale (CAJIS) total score [From Baseline and every six months up to eleven years.]
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 [From Baseline and every six months up to eleven years.]
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 [From Baseline and every six months up to eleven years.]
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) [From Baseline and every six months up to eleven years.]
The lung function parameters of observed FVC (liters) and percent predicted FVC are obtained by spirometry.
- Change from Baseline in observed and percent predicted Forced Expiratory Volume in one second (FEV1) [From Baseline and every six months up to eleven years.]
The lung function parameters of observed FEV1 (liters) and percent predicted FEV1 are obtained by spirometry.
- Change from Baseline in absolute and percent predicted FEV1/FVC ratio [From Baseline and every six months up to eleven years.]
The lung function parameters of the absolute and percent predicted FEV1/FVC ratio are obtained by spirometry.
- Change from Baseline in observed and percent predicted Diffusion Capacity of the Lung for Carbon Monoxide (DLCO) [From Baseline and every six months up to eleven years.]
The lung function parameters of observed (traditional unit of mL/min/mmHg or SI unit of mmol/min/kPa) and percent predicted DLCO is obtained by the DLCO test.
- Change from Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) physical and mental function (mean global physical and mental health score converted into T-scores) for participants ≥15 years old [From Baseline and every six months up to eleven years.]
PROMIS Global Health Scale for the adult version, the global physical health and global mental health scores will be calculated as follows: Global physical health scores will be calculated as the sum of scores from Questions 3, 6, 7 and 8 and will range from 4 (worse health) to 20 (better health); Global mental health scores will be calculated as the sum of scores from Questions 2, 4, 5 and 10 and will range from 4 (worse health) to 20 (better health).
- Change from Baseline in PROMIS overall quality of life (QoL) (mean total score converted into T-scores) for participants <15 years old [From Baseline and every six months up to eleven years.]
PROMIS Global Health Scale for the paediatric version, the total score will be calculated as the sum of scores from the first 7 questions and will range from 7 (worse health) to 35 (better health). If more than 3 questions contributing to the total score are missing, the total score will not be calculated. It will be considered as missing.
- Change from Baseline in annualized number of Investigator-reported flare-ups by body location and overall [From Baseline and every six months up to eleven years.]
The annualized number of flare-ups will be derived from the number of flare-ups the participant experienced since last visit.
- Number of flare-up outcomes [From Baseline and every six months up to eleven years.]
Number of flare-up outcomes as measured by new bone growth, restricted movement at each visit will be reported.
- Flare-up Duration [From Baseline and every six months up to eleven years.]
Flare-up duration will be assessed by body location and overall, at each visit.
- Number of flare-ups [From 12 months before inclusion Baseline up to eleven years.]
- Percentage of participants with new bone growth overall and by flare-up status [From Baseline and every six months up to eleven years.]
The number of extra bone growths, location of the extra bone growth, whether it was preceded by injury, illness, vaccination and/or other events, whether it was preceded by pain, soft tissue swelling, decreased range of motion, stiffness, redness, or warmth, extra bone growth start and stop date and whether it is ongoing, will be collected.
- Movement mobility change [From Baseline and annualy up to eleven years.]
Assessed by key body location: better movement/the same movement/slightly worse movement/moderately worse movement/severely worse movement)
- Number of locations impacted per participant [From Baseline and annualy up to eleven years.]
- Evolution of impacted location (how many times a location is impacted) per location [From Baseline and annualy up to eleven years.]
- Movement mobility outcomes [From Baseline and annualy up to eleven years.]
- Mean dose/year of palovarotene for chronic treatment [From Baseline and every six months up to eleven years.]
- Mean dose/cycle of palovarotene for flare-up treatment [From Baseline and every six months up to eleven years.]
- Incidence of pregnancy [From Baseline and every month up to 30 days of the last palovarotene dose.]
A pregnant participant will be followed throughout the pregnancy and the health status of the baby will be verified
- Description of pregnancy outcomes [From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days of the last palovarotene dose) and the health status of the baby will be verified up until one year of age]
A pregnant participant will be followed throughout the pregnancy.
- Change from Baseline in height velocity [From Baseline and every six months up to eleven years.]
Height velocity will be derived for growing children.
- Mean difference between chronological age and bone age [From Baseline and every six months up to eleven years.]
Bone age (assessment as per the SmPC/PI) will be collected for growing children. Chronological age will be derived for growing children.
- Frequency of premature physeal closure overall [From baseline up to 30 days of the last palovarotene dose]
Epiphyseal status ("open" or "closed") (assessment as per the SmPC/PI) will be collected for growing children.
- Percentage of participants with any fractures overall [From baseline up to 30 days of the last palovarotene dose]
Assessed by radiological imaging and clinical examination.
Eligibility Criteria
Criteria
Inclusion Criteria :
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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;
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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 :
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Currently participating in a palovarotene clinical trial;
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Currently participating in any interventional clinical trial for FOP;
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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).
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Ipsen
Investigators
- Study Director: Ipsen Medical, Director, Ipsen
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CLIN-60120-453