ECE2022 Eposter Presentations Calcium and Bone (114 abstracts)
Palovarotene for the treatment of fibrodysplasia ossificans progressiva: methodology of the phase III open-label PIVOINE rollover trial
Alexander Artyomenko 1 , Aude Houchard 2 & Azzeddine Zemam 2
1Ipsen, Slough, UK; 2Ipsen, Boulogne-Billancourt, France.
Objectives: Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder characterized by heterotopic ossification (HO) and progressive restriction of mobility. To date, no approved disease-modifying treatments for FOP exist, but interim phase III trial (NCT03312634) results suggest marked efficacy for palovarotene (PVO).1 Here, we describe methodology of the PIVOINE trial (NCT05027802) designed to allow treatment continuity and further evaluation of PVO safety and efficacy.
Methods: Patients will receive 5 mg PVO daily, or the parent study completion dose, for a maximum of 3 years; during flare-ups, patients will receive 20 mg daily for 4 weeks, then 10 mg daily for 8 weeks. Enrollment criteria: completion of a parent study (end of study/treatment visit of NCT03312634 or NCT02279095/NCT02979769), ≥14 years old, full skeletal maturity if aged <18 years or deemed to be final adult height. PIVOINE aims to enroll 61 patients; recruitment has not begun. Outcomes are presented in Table.
Summary: Results from PIVOINE, estimated to end in November 2024, will allow further evaluation of PVO in FOP.
References: 1. Pignolo R et al. ASBMR 2020;35(Suppl 1):16–17.
Funding: Sponsored by Ipsen.
| Primary Incidence of treatment-emergent adverse eventsa Secondaryb Cumulative Analogue Joint Involvement Scale (CAJIS) total scorec Use of aids, assistive devices and adaptationsc FOP-Physical Function Questionnaire percentage of worst score (total score; upper extremities/mobility sub-scores)c Frequency of healthcare utilization Observed/percentage predicted:c Forced vital capacity (FVC) Forced expiratory volume in 1 second (FEV1) Diffusion capacity of the lung for carbon monoxide (DLCO) Absolute/percentage predicted:c FEV1/FVC ratio Patient Reported Outcomes Measurement Information System (PROMIS) Global Health Scale physical and mental function scoresc Number of investigator-reported flare-ups, outcomes and durationc Percentage of patients with new bone growth |
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| aCollected continuously over trial period; bCollected every 6 months over trial period; cRaw values and change from inclusion visit. | ||
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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