A Study to Document and to Further Describe Long-term Safety and Effectiveness of Palovarotene in Participants With Fibrodysplasia Ossificans Progressiva (FOP)

NCT06089616 | Recruiting

• 1 other identifier: CLIN-60120-453
• Study Type: observational
• Target: 100
• Locations: 2 countries
• Sites: 5

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 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.

Conditions

Fibrodysplasia Ossificans Progressiva

Outcome Measures

Primary Outcomes (4)

• 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.

Secondary Outcomes (29)

• Change from Baseline in Cumulative Analogue Joint Involvement Scale (CAJIS) total score

  From Baseline and every six months up to eleven years.

• Change from Baseline in use of assistive devices

  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

  From Baseline and every six months up to eleven years.

• Change from Baseline in observed and percent predicted Forced Vital Capacity (FVC)

  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)

  From Baseline and every six months up to eleven years.

Study Arms (2)

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

Eligibility Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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).

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Ipsen: lead

Study Sites (5)

The Regents of the University of California

San Francisco, California, 94103, United States

RECRUITING

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

Philadelphia, Pennsylvania, 19104, United States

ACTIVE NOT RECRUITING

Edmonton Clinic Health Academy (ECHA)- University of Alberta

Edmonton, Canada

RECRUITING

Bone Research and Education Centre

Oakville, Canada

ACTIVE NOT RECRUITING

University Health Network (UHN) - Toronto General

Toronto, Canada

RECRUITING

MeSH Terms

Conditions

Myositis Ossificans

Condition Hierarchy (Ancestors)

Myositis; Muscular Diseases; Musculoskeletal Diseases

Study Officials

• Ipsen Medical, Director

  Ipsen

  STUDY DIRECTOR

Central Study Contacts

Ipsen Clinical Study Enquiries

CONTACT

see email; clinical.trials@ipsen.com

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 6, 2023
• First Posted: October 18, 2023
• Study Start: December 5, 2024
• Primary Completion (Estimated): December 5, 2035
• Study Completion (Estimated): December 5, 2035
• Last Updated: April 30, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Time Frame: 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.
• Access Criteria: Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).

Locations

CA (3); US (2)