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

Who is this study for? Patients with Fibrodysplasia Ossificans Progressiva
What treatments are being studied? Palovarotene
Status: Completed
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Maximum Age: 65
Healthy Volunteers: No
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• 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.

Locations
Other Locations
France
Hôpital Necker-Enfants Malades, Department of Genetics
Paris
Time Frame
Start Date: November 28, 2016
Completion Date: June 28, 2022
Participants
Target number of participants: 9
Treatments
Experimental: Palovarotene
All participants will receive non-flare-up based treatment of 5 mg palovarotene once daily for 24 months.~Participants who continued non-flare-up based treatment under Amendment 1 will receive non-flare-up based treatment for up to an additional 48 months (therefore 72 months over the entire study).~Participants who start non-flare-up based treatment under Amendment 1 will receive non-flare-up based treatment for up to 48 months.~Participants that are skeletally immature will receive weight-adjusted doses.~In the event of an eligible flare-up all participants will receive 20 mg palovarotene for 28 days, followed by 10 mg palovarotene for 56 days. Participants under the age of 18 years will receive weight-adjusted doses.
Sponsors
Leads: Clementia Pharmaceuticals Inc.

This content was sourced from clinicaltrials.gov

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