An International Observational Registry Study to Further Describe Long-term Safety and Effectiveness of Palovarotene in Patients with Fibrodysplasia Ossificans Progressiva (FOP) (FOPal)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/1000000060
• EU PAS number:
  EUPAS1000000060
• Study ID:
  1000000060
• Official title and acronym:
  An International Observational Registry Study to Further Describe Long-term Safety and Effectiveness of Palovarotene in Patients with Fibrodysplasia Ossificans Progressiva (FOP) (FOPal)
• DARWIN EU® study:
  No
• Study countries:
  Australia

  Brazil

  Canada

  United States
• Study description:
  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.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Ipsen Pharma

Study timelines

Date when funding contract was signed

Actual: 09/05/2020

Study start date

Planned: 30/05/2024

Actual: 05/12/2024

Data analysis start date

Planned: 30/05/2026

Date of final study report

Planned: 30/08/2036

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Approximate 100% funding: Ipsen Pharma

Study protocol

Initial protocol

CLIN-60120-453_16.1.1 Protocol 2023SEP07_Redacted (1).pdf

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  Non-EU RMP only

Other study registration identification numbers and links

NCT06089616

Link to ClinicalTrials.gov