Clinical trial NCT06089616: A Study to Document and to Further Describe Long-term Safety and Effectiveness of Palovarotene in Participants With Fibrodysplasia Ossificans Progressiva (FOP)
Official title: An International Observational Registry Study to Further Describe Long-term Safety and Effectiveness of Palovarotene in Patients With Fibrodysplasia Ossificans Progressiva (FOP)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Fibrodysplasia Ossificans Progressiva
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2023-10-18
Enrollment: 70
Sex: ALL
Minimum age (years): 8
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 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).

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


Eligibility 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).

Primary outcomes:
measure: Percentage of Participants With Treatment-emergent Adverse Events (TEAEs), whether or not they are considered as related to palovarotene
timeFrame: From Baseline up to 30 days after the last palovarotene dose.
measure: Percentage of Participants With Serious and Non-serious treatment-related TEAEs
timeFrame: From Baseline up to 30 days after the last palovarotene dose.
measure: Percentage of Participants With all serious TEAEs, whether or not they are considered as related to the palovarotene
timeFrame: From Baseline up to 30 days after the last palovarotene dose.
measure: Percentage of Participants With nonserious TEAEs whether or not they are considered as related to the palovarotene.
timeFrame: From Baseline up to 30 days after the last palovarotene dose.
Secondary outcomes:
measure: Change from Baseline in Cumulative Analogue Joint Involvement Scale (CAJIS) total score
timeFrame: From Baseline and every six months up to eleven years.
measure: Change from Baseline in use of assistive devices
timeFrame: From Baseline and every six months up to eleven years.
measure: Change from Baseline in percent of worst score for Fibrodysplasia Ossificans Progressiva-Physical Function Questionnaire (FOP-PFQ) total score
timeFrame: From Baseline and every six months up to eleven years.
measure: Change from Baseline in observed and percent predicted Forced Vital Capacity (FVC)
timeFrame: From Baseline and every six months up to eleven years.
measure: Change from Baseline in observed and percent predicted Forced Expiratory Volume in one second (FEV1)
timeFrame: From Baseline and every six months up to eleven years.
measure: Change from Baseline in absolute and percent predicted FEV1/FVC ratio
timeFrame: From Baseline and every six months up to eleven years.
measure: Change from Baseline in observed and percent predicted Diffusion Capacity of the Lung for Carbon Monoxide (DLCO)
timeFrame: From Baseline and every six months up to eleven years.
measure: 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
timeFrame: From Baseline and every six months up to eleven years.
measure: Change from Baseline in PROMIS overall quality of life (QoL) (mean total score converted into T-scores) for participants <15 years old
timeFrame: From Baseline and every six months up to eleven years.
measure: Change from Baseline in annualized number of Investigator-reported flare-ups by body location and overall
timeFrame: From Baseline and every six months up to eleven years.
measure: Number of flare-up outcomes
timeFrame: From Baseline and every six months up to eleven years.
measure: Flare-up Duration
timeFrame: From Baseline and every six months up to eleven years.
measure: Number of flare-ups
timeFrame: From 12 months before inclusion Baseline up to eleven years.
measure: Percentage of participants with new bone growth overall and by flare-up status
timeFrame: From Baseline and every six months up to eleven years.
measure: Movement mobility change
timeFrame: From Baseline and annualy up to eleven years.
measure: Number of locations impacted per participant
timeFrame: From Baseline and annualy up to eleven years.
measure: Evolution of impacted location (how many times a location is impacted) per location
timeFrame: From Baseline and annualy up to eleven years.
measure: Movement mobility outcomes
timeFrame: From Baseline and annualy up to eleven years.
measure: Mean dose/year of palovarotene for chronic treatment
timeFrame: From Baseline and every six months up to eleven years.
measure: Mean dose/cycle of palovarotene for flare-up treatment
timeFrame: From Baseline and every six months up to eleven years.
measure: Incidence of pregnancy
timeFrame: From Baseline and every month up to 30 days of the last palovarotene dose.
measure: Description of pregnancy outcomes
timeFrame: 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
measure: Change from Baseline in height velocity
timeFrame: From Baseline and every six months up to eleven years.
measure: Mean difference between chronological age and bone age
timeFrame: From Baseline and every six months up to eleven years.
measure: Frequency of premature physeal closure overall
timeFrame: From baseline up to 30 days of the last palovarotene dose
measure: Percentage of participants with any fractures overall
timeFrame: From baseline up to 30 days of the last palovarotene dose
Other outcomes:
Locations:
city: Edmonton
country: Canada
facility: Edmonton Clinic Health Academy (ECHA)- University of Alberta
city: Oakville
country: Canada
facility: Bone Research and Education Centre
city: Toronto
country: Canada
facility: University Health Network (UHN) - Toronto General
Study officials:
affiliation: Ipsen
name: Ipsen Medical, Director
role: STUDY_DIRECTOR
References: