A Rollover Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged ≥14 Years With Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed the Relevant Parent Studies.
NCT ID: NCT05027802
Last Updated: 2025-06-22
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
61 participants
INTERVENTIONAL
2022-03-14
2024-11-30
Brief Summary
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The aim of the study is also to ensure treatment continuity to participants who have completed one of the parent studies (Study PVO-1A-301, Study PVO-1A-202 and Study PVO-1A-204) and who, in the investigator's judgement, may benefit from palovarotene therapy.
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Palovarotene Chronic/Flare-Up Regimen
Chronic treatment: participants will receive 5 mg palovarotene or the dose received during participation in the parent study at the time of transition to Study CLIN-60120-452 or prior to interrupting/stopping palovarotene treatment.
Flare-up treatment: at the time of a flare-up (or substantial high-risk traumatic event likely to lead to a flare-up) participants will receive 20 mg palovarotene for 28 days, followed by 10 mg palovarotene for 56 days.
Palovarotene
Palovarotene will be taken orally once daily at approximately the same time each day.
Interventions
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Palovarotene
Palovarotene will be taken orally once daily at approximately the same time each day.
Eligibility Criteria
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Inclusion Criteria
* Participant must be ≥14 years of age (aligned with the age of treated participants in the ongoing parent studies PVO-1A-301 and PVO-1A-202/PVO-1A-204) and qualify as 100% skeletally mature (if \<18 years, based on assessments carried out at parent EOS Visit; if ≥18 years, automatically considered 100% skeletally mature) or have reached final adult height based on investigator's assessment, at the time the Study CLIN- 60120-452 informed consent is signed.
Exclusion Criteria
* Uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal, endocrine, metabolic, ophthalmologic, immunologic, psychiatric, or other significant disease.
* Symptomatic vertebral fracture.
* Intercurrent known or suspected non-healed fracture at any location;
* Any other medical condition/clinically significant abnormalities that would expose the participant to undue risk or interfere with study assessments.
* Amylase or lipase \>2× above the upper limit of normal (ULN) or with a history of chronic pancreatitis.
* Elevated aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \>2.5× ULN.
* Fasting triglycerides \>400 mg/dL with or without therapy.
* Suicidal ideation (type 4 or 5) or any suicidal behaviour at the Inclusion Visit as defined by the Columbia-Suicide Severity Rating Scale (C-SSRS).
* Current use of vitamin A or beta carotene, multivitamins containing vitamin A or beta carotene, or herbal preparations, fish oil, and unable or unwilling to discontinue use of these products during palovarotene treatment.
* Exposure to synthetic oral retinoids other than palovarotene within 4 weeks of the Inclusion Visit.
* Concurrent treatment with tetracycline or any tetracycline derivatives due to the potential increased risk of pseudotumor cerebri.
* Use of concomitant medications that are strong inhibitors or inducers of cytochrome P450 (CYP450) 3A4 activity; or kinase inhibitors such as imatinib.
* Palovarotene is reimbursed in the country where the study is being conducted.
* 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.
14 Years
ALL
No
Sponsors
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Ipsen
INDUSTRY
Responsible Party
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Principal Investigators
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Ipsen Medical Director
Role: STUDY_DIRECTOR
Ipsen
Locations
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University of California San Francisco (UCSF)
San Francisco, California, United States
Mayo Clinic
Rochester, Minnesota, United States
Children's Hospital of Philidelphia
Philadelphia, Pennsylvania, United States
The Perelman School of Medicine - The University of Pennsylvania
Philadelphia, Pennsylvania, United States
Hospital Italiano de Buenos Aires
Buenos Aires, , Argentina
Royal North Shore Hospital
Saint Leonards, New South Wales, Australia
Hospital Israelita Albert Einstein
Morumbi, São Paulo, Brazil
Toronto General Hospital
Toronto, , Canada
Groupe Hospitalier Necker Enfants Malades
Paris, , France
Istituto Giannina Gaslini
Genoa, , Italy
Hospital Universitario Ramon y Cajal
Colmenar Viejo, , Spain
Norrlands Universitetssjukhus
Umeå, , Sweden
Royal National Orthopaedic Hospital
London, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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Other Identifiers
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2021-002244-70
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
CLIN-60120-452
Identifier Type: -
Identifier Source: org_study_id
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