An Open-Label Extension Study of Palovarotene to Prevent Heterotopic Ossification in People With Fibrodysplasia Ossificans Progressiva (FOP) in France

• Status: Active, not recruiting
• Trial end date: 2022-07-01
• Target enrollment: 0
• Participant gender: All

Summary

Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by heterotopic ossification (HO), i.e., abnormal bone formation, often associated with painful, recurrent episodes of soft tissue swelling (flare-ups). Lesions begin in early childhood and lead to progressive ankyloses of major joints with resultant loss of movement.

In this study, the ability of different palovarotene dosing regimens to prevent the formation of new HO will be evaluated in adult and pediatric participants with FOP in France.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Clementia Pharmaceuticals Inc.

Criteria

Inclusion Criteria:

• Prior participation in Amendment 1 of the current study (PVO-1A-204).

• Written, signed, and dated informed consent and, for participants who are minors, age-appropriate participant assent (performed according to local regulations).

• Accessible for treatment with palovarotene and follow-up (able and willing to travel to a site for the initial and all follow-up clinic visits).

• Able to undergo low-dose, WBCT scan, excluding head.

• Females of child-bearing potential (FOCBP) must have a negative blood or urine pregnancy test (with sensitivity of at least 50 mIU/mL) prior to administration of palovarotene.

• Male and FOCBP participants must agree to remain abstinent from heterosexual sex during treatment and for 1 month after treatment or, if sexually active, to use two effective methods of birth control during and for 1 month after treatment. Additionally, sexually active FOCBP participants must already be using two effective methods of birth control 1 month before treatment is to start. Specific risk of the use of retinoids during pregnancy, and the agreement to remain abstinent or use two effective methods of birth control will be clearly defined in the informed consent and the participant or legally authorized representatives.

Exclusion Criteria:

• Any reason that, in the opinion of the Investigator, would lead to the inability of the participant and/or family to comply with the protocol.

• Amylase or lipase >2x above the upper limit of normal or with a history of pancreatitis.

• Elevated aspartate aminotransferase or alanine aminotransferase >2.5x the upper limit of normal.

• Fasting triglycerides >400 mg/dL with or without therapy.

• If currently using vitamin A or beta carotene, multivitamins containing vitamin A or beta carotene, herbal preparations containing vitamin A or beta carotene, or fish oil, and unable or unwilling to discontinue use of these products during palovarotene treatment.

• Participants experiencing suicidal ideation (type 4 or 5) or any suicidal behavior within the past month as defined by the Columbia Suicide Severity Rating Scale (C-SSRS).