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

Conditions

• Fibrodysplasia Ossificans Progressiva

Eligibility Criteria

Sex

ALL

Age

8 Years - N/A

Healthy Volunteers

Not accepted

Study Details

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.

Key Dates

Start date

Dec 5, 2024

Status verified

May 2026

Primary completion

Dec 5, 2035

Completion

Dec 5, 2035

Study Design

Enrollment

100 participants (estimated)

Arms

• Arm: Exposed Cohort
  Participants treated with palovarotene (i.e already be receiving palovarotene as prescribed by their treating physician according to locally approved product information)
• Arm: Unexposed Cohort
  Participants untreated with palovarotene

Primary Outcome Measure

Percentage of Participants With Treatment-emergent Adverse Events (TEAEs), whether or not they are considered as related to palovarotene [ Time Frame: From Baseline up to 30 days after the last palovarotene dose. ]

Central Contacts

• Ipsen Clinical Study Enquiries
  see email
  [email protected]

Locations (2)

Find similar trials in San Francisco, CA