PIVOINE
Research type
Research Study
Full title
Rollover Study; Multicentre, Phase III, Open-label Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged ≥14 Years with Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed Study PVO-1A-301 or PVO-1A-202/PVO-1A-204 and May Benefit from Palovarotene Therapy.
IRAS ID
306188
Contact name
Richard Keen
Contact email
Sponsor organisation
Ipsen Pharma SAS
Eudract number
2021-002244-70
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 5 months, 17 days
Research summary
Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare and severely disabling genetic disease characterised by bone formation in muscles, tendons, and ligaments. These bone formations accumulate, progressively restricting movement. The prognosis of patients with FOP is poor and life expectancy is low. At present, there are no approved medical treatment options to prevent the formation of bone in FOP.
This study CLIN-60120-452 is a multicentre, noncomparative rollover study with the aim of continuing to provide palovarotene to participants with FOP who have completed one of the parent studies (Study PVO-1A-301 or PVO-1A-202/PVO-1A-204) and, in the investigator’s judgement, may benefit from palovarotene therapy.
The study is designed primarily to further evaluate the safety of the study drug palovarotene and secondly, to collect efficacy data in male and female participants aged ≥14 years with FOP treated with palovarotene. In-depth qualitative interviews will be proposed to all participants and caregivers. Patient-reported questionnaires will be completed by the participant on paper. All other data collected in the study will be recorded in an electronic case report form (except for qualitative participant and caregiver interview data).
A maximum of 87 participants from studies PVO-1A-301, PVO-1A-202/PVO-1A-204 are eligible to be enrolled and will receive palovarotene once daily at the dose received during their participation in the parent study at the time of transition to Study CLIN-60120-452 or prior to interrupting/stopping palovarotene treatment. Participants will continue to receive palovarotene until it is commercially available in the country where the study is being conducted, or another access programme becomes available, or until the study end date of November 2024 is reached, whichever occurs first.
REC name
South Central - Hampshire A Research Ethics Committee
REC reference
21/SC/0410
Date of REC Opinion
1 Feb 2022
REC opinion
Further Information Favourable Opinion