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.

Study Purpose

The main objective of this study is to further evaluate the safety and efficacy of palovarotene in adult and paediatric participants with FOP. 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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 14 Years and Over
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - Participant has completed the EOS or End of Treatment Visit of Study PVO-1A-301 or PVO-1A-202 (PVO-1A-202 Parts C and D correspond to Study PVO-1A-204 in France) and did not previously withdraw consent from any of the parent studies to be eligible for Study CLIN-60120-452.
  • - 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:

  • - History of allergy or hypersensitivity to retinoids, gelatin, lactose (note that lactose intolerance is not exclusionary) or palovarotene, or unresponsiveness to prior treatment with palovarotene.
  • - 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.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT05027802
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 3
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Ipsen
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Ipsen Medical Director
Principal Investigator Affiliation Ipsen
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Recruiting
Countries Argentina, Australia, Canada, France, Italy, Sweden, United Kingdom, United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Fibrodysplasia Ossificans Progressiva (FOP)
Arms & Interventions

Arms

Experimental: 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.

Interventions

Drug: - Palovarotene

Palovarotene will be taken orally once daily at approximately the same time each day.

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

San Francisco, California

Status

Recruiting

Address

University of California San Francisco (UCSF)

San Francisco, California, 94143

Mayo Clinic, Rochester, Minnesota

Status

Not yet recruiting

Address

Mayo Clinic

Rochester, Minnesota, 55905

Children's Hospital of Philidelphia, Philadelphia, Pennsylvania

Status

Active, not recruiting

Address

Children's Hospital of Philidelphia

Philadelphia, Pennsylvania, 19104

Philadelphia, Pennsylvania

Status

Active, not recruiting

Address

The Perelman School of Medicine - The University of Pennsylvania

Philadelphia, Pennsylvania, 19104

International Sites

Hospital Italiano de Buenos Aires, Buenos Aires, Argentina

Status

Not yet recruiting

Address

Hospital Italiano de Buenos Aires

Buenos Aires, , C1181ACH

Royal North Shore Hospital, Saint Leonards, New South Wales, Australia

Status

Recruiting

Address

Royal North Shore Hospital

Saint Leonards, New South Wales, 2065

Toronto General Hospital, Toronto, Canada

Status

Active, not recruiting

Address

Toronto General Hospital

Toronto, , M5T 2S8

Paris, France

Status

Active, not recruiting

Address

Groupe Hospitalier Necker Enfants Malades

Paris, , 75015

Istituto Giannina Gaslini, Genoa, Italy

Status

Active, not recruiting

Address

Istituto Giannina Gaslini

Genoa, , 16147

Norrlands Universitetssjukhus, Umeå, Sweden

Status

Active, not recruiting

Address

Norrlands Universitetssjukhus

Umeå, , 90737

Royal National Orthopaedic Hospital, London, United Kingdom

Status

Active, not recruiting

Address

Royal National Orthopaedic Hospital

London, , HA7 4LP