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

Fibrodysplasia Ossificans Progressiva Clinical Trial

Official title:
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)

Status Completed

Clinical Trial 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.

Clinical Trial Description

The main objective of this Phase 2, open-label study is to evaluate the safety and efficacy of different palovarotene dosing regimens in participants with FOP in France. Efficacy will be assessed based on the ability of palovarotene to prevent the formation of new heterotopic ossification (HO) as assessed by low-dose whole body computed tomography (WBCT) scan, excluding head. Participants who successfully completed study PVO-1A-201 as well as up to two new adult participants were followed for up to 24 months. Participants under Amendment 1 will be followed for up to an additional 48 months. No new participants will be enrolled. The Adult Cohort will include all participants with at least 90% skeletal maturity, regardless of age. The Pediatric Cohort will include all participants with less than 90% skeletal maturity. Any Pediatric Cohort participants who achieves ≥90% skeletal maturity during the study can be considered for enrollment into the Adult Cohort at the discretion of then Investigator. Participants that were skeletally immature at their last assessment will be invited back to participate in a safety follow-up (Part D) that includes yearly visits for up to a 2-year follow-up period following last dose. No dosing will occur in Part D. ;

Study Design

Related Conditions & MeSH terms

Fibrodysplasia Ossificans Progressiva

Clinical Trial Details

NCT number	NCT02979769
Study type	Interventional
Source	Ipsen
Contact
Status	Completed
Phase	Phase 2
Start date	November 28, 2016
Completion date	June 28, 2022

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT02279095` - An Open-Label Extension Study of Palovarotene Treatment in Fibrodysplasia Ossificans Progressiva (FOP)	Phase 2
Completed	`NCT03312634` - An Efficacy and Safety Study of Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva.	Phase 3
Recruiting	`NCT05394116` - A Study to Assess Safety, Tolerability and Efficacy of Garetosmab Versus Placebo Administered Intravenously (IV) in Adult Participants With Fibrodysplasia Ossificans Progressiva (FOP)	Phase 3
Completed	`NCT02066324` - Urine Sample Collection From FOP Patients	N/A
Completed	`NCT02190747` - An Efficacy and Safety Study of Palovarotene to Treat Preosseous Flare-ups in FOP Subjects	Phase 2
Completed	`NCT04829773` - Study Evaluating the Effect of Food on the Pharmacokinetics of Palovarotene and the Effect of Palovarotene on the Pharmacokinetics of the CYP3A4 Substrate Midazolam in Two Cohorts of Healthy Adult Subjects	Phase 1
Completed	`NCT04665323` - An International Cross-sectional Survey to Evaluate the Burden of Fibrodysplasia Ossificans Progressiva (FOP) on Patients and Their Families.
Completed	`NCT03188666` - A Study to Examine the Safety, Tolerability and Effects on Abnormal Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva	Phase 2
Terminated	`NCT02521792` - In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) Subjects	Phase 2
Completed	`NCT02322255` - A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP)
Recruiting	`NCT05039515` - A Study to Assess the Effectiveness and Safety of 2 Dosage Regimens of Oral Fidrisertib (IPN60130) for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP).	Phase 2
Recruiting	`NCT04307953` - Saracatinib Trial TO Prevent FOP	Phase 2
Not yet recruiting	`NCT06089616` - A Study to Document and to Further Describe Long-term Safety and Effectiveness of Palovarotene in Participants With Fibrodysplasia Ossificans Progressiva (FOP)
Completed	`NCT04818398` - Study of Single-Ascending Doses of DS-6016a in Healthy Japanese Subjects	Phase 1

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.