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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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 |
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| Measure | Description | Time Frame |
|---|---|---|
| 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). | From Baseline up to 30 days after the last palovarotene dose. |
| 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. | From Baseline up to 30 days after the last palovarotene dose. |
| 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. | From Baseline up to 30 days after the last palovarotene dose. |
| 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. | From Baseline up to 30 days after the last palovarotene dose. |
| Measure | Description | Time Frame |
|---|---|---|
| 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). | From Baseline and every six months up to eleven years. |
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Inclusion Criteria :
Exclusion Criteria :
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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).
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Ipsen Clinical Study Enquiries | Contact | see email | clinical.trials@ipsen.com |
| Name | Affiliation | Role |
|---|---|---|
| Ipsen Medical, Director | Ipsen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Regents of the University of California | Recruiting | San Francisco | California | 94103 | United States | |
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications.
Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.
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Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.
Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).
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| 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. | From Baseline and every six months up to eleven years. |
| 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. | From Baseline and every six months up to eleven years. |
| 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. | From Baseline and every six months up to eleven years. |
| Change from Baseline in observed and percent predicted Forced Expiratory Volume in one second (FEV1) | The lung function parameters of observed FEV1 (liters) and percent predicted FEV1 are obtained by spirometry. | From Baseline and every six months up to eleven years. |
| Change from Baseline in absolute and percent predicted FEV1/FVC ratio | The lung function parameters of the absolute and percent predicted FEV1/FVC ratio are obtained by spirometry. | From Baseline and every six months up to eleven years. |
| Change from Baseline in observed and percent predicted Diffusion Capacity of the Lung for Carbon Monoxide (DLCO) | 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. | From Baseline and every six months up to eleven years. |
| 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 | PROMIS Global Health Scale for the adult version, the global physical health and global mental health scores will be calculated as follows:
| From Baseline and every six months up to eleven years. |
| Change from Baseline in PROMIS overall quality of life (QoL) (mean total score converted into T-scores) for participants <15 years old | 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. | From Baseline and every six months up to eleven years. |
| Change from Baseline in annualized number of Investigator-reported flare-ups by body location and overall | The annualized number of flare-ups will be derived from the number of flare-ups the participant experienced since last visit. | From Baseline and every six months up to eleven years. |
| Number of flare-up outcomes | Number of flare-up outcomes as measured by new bone growth, restricted movement at each visit will be reported. | From Baseline and every six months up to eleven years. |
| Flare-up Duration | Flare-up duration will be assessed by body location and overall, at each visit. | From Baseline and every six months up to eleven years. |
| Number of flare-ups | From 12 months before inclusion Baseline up to eleven years. |
| Movement mobility change | Assessed by key body location: better movement/the same movement/slightly worse movement/moderately worse movement/severely worse movement) | From Baseline and annually up to eleven years. |
| Number of locations impacted per participant | From Baseline and annually up to eleven years. |
| Evolution of impacted location (how many times a location is impacted) per location | From Baseline and annually up to eleven years. |
| Movement mobility outcomes: Percentage of participants with new bone growth overall and by flare-up status | 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. | From Baseline and every six months up to eleven years. |
| Percentage of participants with any fractures overall | Assessed by radiological imaging and clinical examination. | From baseline up to 30 days after the last palovarotene dose. |
| Change from Baseline in annualized number of fractures by body location and overall | Assessed by radiological imaging and clinical examination. | From baseline up to 30 days after the last palovarotene dose. |
| Incidence of pregnancy | A pregnant participant with FOP exposed to palovarotene will be followed throughout the pregnancy and the health status of the baby will be verified | From Baseline and every month up to 30 days after the last palovarotene dose. |
| Description of pregnancy characteristics and outcomes | A pregnant participant with FOP exposed to palovarotene will be followed throughout the pregnancy. | From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age. |
| Description of pregnancy related Adverse Events (AEs) | A pregnant participant with FOP exposed to palovarotene will be followed throughout the pregnancy. | From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age. |
| Description of birth and developmental outcomes through the first year after birth | A pregnant participant with FOP exposed to palovarotene will be followed throughout the pregnancy. | From the signing of the ICF and the participant will be followed throughout the pregnancy (From Baseline and every month up to 30 days after the last palovarotene dose) and the health status of the baby will be verified up until one year of age. |
| Description of birth and developmental outcomes through the first year after birth | Linear height and knee height velocity will be derived for growing children with FOP exposed and unexposed to palovarotene until skeletal maturity or final adult height is reached. | From Baseline and every six months up to eleven years. |
| Change from Baseline in height z-score | Linear height z-score will be derived for growing children with FOP exposed and unexposed to palovarotene until skeletal maturity or final adult height is reached. | From Baseline and every six months up to eleven years. |
| Mean difference between chronological age and bone age | Bone age (assessment as per the SmPC/PI) and chronological age will be collected for growing children with FOP exposed and unexposed to palovarotene until skeletal maturity or final adult height is reached. | From Baseline and every six months up to eleven years. |
| Frequency of premature physeal closure (PPC) overall | Epiphyseal status ("open" or "closed") (assessment as per the SmPC/PI) will be collected for growing children with FOP exposed and unexposed to palovarotene until skeletal maturity or final adult height is reached. | From baseline up to 30 days after the last palovarotene dose |
| Mean dose/year of palovarotene for participants with FOP exposed and unexposed to palovarotene for chronic treatment | From Baseline and every six months up to eleven years. |
| Mean dose/cycle of palovarotene for participants with FOP exposed and unexposed to palovarotene for flare-up treatment | From Baseline and every six months up to eleven years. |
| The Trustees of the University of Pennsylvania, Office of Clinical Research-Legal Services, Perelman School of Medicine |
| Recruiting |
| Philadelphia |
| Pennsylvania |
| 19104 |
| United States |
| Edmonton Clinic Health Academy (ECHA)- University of Alberta | Recruiting | Edmonton | Canada |
| Bone Research and Education Centre | Active, not recruiting | Oakville | Canada |
| University Health Network (UHN) - Toronto General | Recruiting | Toronto | Canada |
| ID | Term |
|---|---|
| D009221 | Myositis Ossificans |
| ID | Term |
|---|---|
| D009220 | Myositis |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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