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Summary

for people ages 6–65 (full criteria)
at San Francisco, California and other locations
study started
estimated completion:

Description

Summary

Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by heterotopic ossification (HO) often associated with painful, recurrent episodes of soft tissue swelling (flare-ups) that lead to ankyloses of major joints with cumulative and irreversible loss of movement and disability. In this study, the ability of palovarotene to prevent HO formation will be evaluated.

Official Title

A Phase 2, Open-Label Extension, Efficacy and Safety Study of a RARγ Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)

Details

The main objective of this Phase 2, multicenter, open-label study is to evaluate the safety and efficacy of different palovarotene dosing regimens in subjects with FOP.

In Part A, all subjects who completed Study PVO-1A-201 and enrolled into the current study received daily treatment with open-label palovarotene for an eligible flare-up at a dose of 10 mg for 14 days, followed by 5 mg for 28 days (or the weight-based equivalent). Part A is completed.

In Part B, subjects with at least 90% skeletal maturity were treated with 5 mg palovarotene on a daily basis (ie, chronically). In the event of an eligible flare-up, all subjects received 20 mg palovarotene daily for 28 days, followed by 10 mg for 56 days (dosing was weight-based in subjects who were skeletally immature). Dosing could be extended if the flare-up was not resolved by Flare-up Day 84 and continued until the flare-up resolved. Dose reduction, as directed by the Investigator, occurred in the event of intolerable side effects. The duration of Part B is up to 24 months.

In Part C, the dosing regimens implemented in Part B will continue except that subjects with less than 90% skeletal maturity will now receive chronic daily administration of palovarotene (5 mg, or the weight-based equivalent). The assessment of HO will occur every 12 months using low-dose, whole body computed tomography (WBCT), excluding head; other efficacy and safety outcomes will be evaluated remotely every 3 months, or monthly during flare-up based treatment. The duration of Part C is 36 months.

Keywords

Fibrodysplasia Ossificans Progressiva Open-label extension study Clinical trial Phase 2 Efficacy and safety Heterotopic ossification Flare-up Palovarotene Retinoic acid receptor agonist Retinoic acid receptor gamma agonist Clementia Myositis Ossificans Progressiva Munchmeyer's Disease FOP Myositis Ossificans

Eligibility

You can join if…

Open to people ages 6–65

  • Completion of Study PVO-1A-202/Part B.
  • Written, signed, and dated informed consent and, for subjects who are minors,age-appropriate subject 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.

You CAN'T join if...

  • Any reason that, in the opinion of the Investigator, would lead to the inability of the subject 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.

Locations

  • University of California San Francisco, Division of Endocrinology and Metabolism
    San Francisco, California, 94143, United States
  • Mayo Clinic, Department of Medicine
    Rochester, Minnesota, 55905, United States
  • University of Pennsylvania, Center for FOP & Related Bone Disorders
    Philadelphia, Pennsylvania, 19104, United States
  • The Royal National Orthopaedic Hospital, Brockley Hill
    Stanmore, Middlesex, HA7 4LP, United Kingdom
  • Hôpital Necker-Enfants Malades, Department of Genetics
    Paris, France
  • Hospital Italiano de Buenos Aires, Department of Pediatrics
    Buenos Aires, Argentina
  • Queensland University of Technology (QUT) Institute of Health and Biomedical Innovation (IHBI)
    Woolloongabba, Queensland, 4102, Australia

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Clementia Pharmaceuticals Inc.
Links
Website for the International FOP Association
Website for the French FOP Association
Click here for more information about this study: A Phase 2, Open-Label Extension, Efficacy and Safety Study of a RARγ-Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects with Fibrodysplasia Ossificans Progressiva (FOP)
ID
NCT02279095
Phase
Phase 2
Lead Scientist
Edward Hsiao
Study Type
Interventional
Last Updated
October 9, 2017