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Clinical Trials in the USA / NCT06089616
Recruiting Observational

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

NCT06089616 · tracked via the Priya Life Science USA tracker
Sponsor
Ipsen
Phase
Observational
Started
2024-12-05
Last updated
2026-10-23

Condition(s) studied

Fibrodysplasia Ossificans Progressiva

Study 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.

Eligibility

Sex
ALL
Min age
8 Years
Max age
—
Healthy volunteers
No
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 outcome measure(s)

  • 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.

Trial sites (5)

FacilityCityRegionStatus
The Regents of the University of California San Francisco California Recruiting
The Trustees of the University of Pennsylvania, Office of Clinical Research-Legal Services, Perelman School of Medicine Philadelphia Pennsylvania 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

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A Study to Assess the Effectiveness and Safety of IPN10200 in Adults With Moderate to Severe Wrinkle-like Lines Between the Eyebrows Active, not recruiting

Other trials for the same condition

A Study of Andecaliximab in Participants With Fibrodysplasia Ossificans Progressiva (FOP) Active, not recruiting
A Study to Assess Safety, Tolerability and Efficacy of Garetosmab Versus Placebo Administered Intravenously (IV) in Adult Participants With Fibrodysplasia Ossificans Progressiva (FOP) Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06089616 on ClinicalTrials.gov ↗ ← All trials in the USA