Status:
COMPLETED
An Open-Label Extension Study of Palovarotene Treatment in Fibrodysplasia Ossificans Progressiva (FOP)
Lead Sponsor:
Clementia Pharmaceuticals Inc.
Conditions:
Fibrodysplasia Ossificans Progressiva
Eligibility:
All Genders
6-65 years
Phase:
PHASE2
Brief 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, recurren...
Detailed Description
The main objective of this Phase 2, multicenter, open-label study is to evaluate the safety and efficacy of different palovarotene dosing regimens in participants with FOP. Efficacy will be assessed b...
Eligibility Criteria
Inclusion
- Completion of Study PVO-1A-202/Part B.
- 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 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 females of childbearing potential (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
- 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.
- 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).
Key Trial Info
Start Date :
October 9 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 20 2022
Estimated Enrollment :
58 Patients enrolled
Trial Details
Trial ID
NCT02279095
Start Date
October 9 2014
End Date
September 20 2022
Last Update
February 19 2025
Active Locations (8)
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1
University of California San Francisco, Division of Endocrinology and Metabolism
San Francisco, California, United States, 94143
2
Mayo Clinic, Department of Medicine
Rochester, Minnesota, United States, 55905
3
University of Pennsylvania, Center for FOP & Related Bone Disorders
Philadelphia, Pennsylvania, United States, 19104
4
Hospital Italiano de Buenos Aires, Department of Pediatrics
Buenos Aires, Argentina