Study identification

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
First published:
01/02/2024
Institution

Contact details

Ipsen Medical Director

Primary lead investigator

Study timelines

Date when funding contract was signed

Actual:

Study start date

Planned:
Actual:

Data analysis start date

Planned:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Approximate 100% funding: Ipsen Pharma
Regulatory

Was the study required by a regulatory body?

Yes

Is the study required by a Risk Management Plan (RMP)?

Non-EU RMP only