The Study of an Investigational Drug, Palovarotene, in the Treatment of Preosseous Flare-ups in Subjects with Fibrodysplasia Ossificans Progressiva.

Phase 1

Conditions: Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) and abnormal heterotopic ossification (HO) in muscles, tendons, and ligaments. Lesions begin in early childhood and lead to progressive ankyloses of major joints with resultant loss of movement. Prognosis is poor and median life expectancy is 40 years.
MedDRA version: 18.0Level: PTClassification code 10068715Term: Fibrodysplasia ossificans progressivaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Registration Number: EUCTR2014-001453-17-GB

Lead Sponsor: Clementia Pharmaceuticals Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 40

Inclusion Criteria

•Male or female at least 6 years of age.
•Subjects clinically diagnosed with classic FOP.
•Symptomatic onset of a distinct flare-up within 7 days of Study Day 1 (start of study drug) and defined by the presence of at least two of six of the following symptoms: pain, soft tissue swelling, decreased ROM, stiffness, redness, and warmth. Symptoms must be reported by the subject during the pre-screening period, be consistent with their previous flare-ups, include a subject-reported onset date, and flare-up confirmed by the Investigator at the Screening visit.
•Flare-up is at an appendicular area (upper or lower extremity), abdomen, or chest; and subject has received, is receiving, or is willing to receive treatment per standard of care, which may or may not include prednisone (2 mg/kg PO [per os] to a maximum dose of 100 mg daily) for 4 days.
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

•Weight <20 kg.
•Intercurrent non-healed fracture at any location.
•Complete immobilization of joint at site of flare-up.
•The inability of the subject to undergo imaging assessments using plain radiographs.
•If currently using vitamin A or beta carotene, multivitamins containing vitamin A or beta carotene, herbal preparations, fish oil, and unable or unwilling to discontinue use of these products for the duration of the study.
•Exposure to synthetic oral retinoids in the past 30 days prior to Screening (signature of the informed consent).
•Concurrent treatment with tetracycline due to the potential increased risk of pseudotumor cerebri.
•History of allergy or hypersensitivity to retinoids or lactose.
•Concomitant medications that are inhibitors or inducers of CYP450 3A4 activity.