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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05027802
Registration number
NCT05027802
Ethics application status
Date submitted
25/08/2021
Date registered
30/08/2021
Date last updated
31/05/2024
Titles & IDs
Public title
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.
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Scientific title
Rollover Study; Multicentre, Phase III, Open-label 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 Study PVO-1A-301 or PVO-1A-202/PVO-1A-204 and May Benefit From Palovarotene Therapy.
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Secondary ID [1]
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2021-002244-70
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Secondary ID [2]
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CLIN-60120-452
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Universal Trial Number (UTN)
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Trial acronym
PIVOINE
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Fibrodysplasia Ossificans Progressiva (FOP)
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Condition category
Condition code
Musculoskeletal
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Other muscular and skeletal disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Injuries and Accidents
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Other injuries and accidents
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Palovarotene
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.
Treatment: Drugs: Palovarotene
Palovarotene will be taken orally once daily at approximately the same time each day.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incidence and description of all serious and non-serious treatment-emergent adverse events (TEAEs) whether or not they are considered as related to the study intervention;
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Assessment method [1]
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Adverse events will be coded using the Medical Dictionary for Regulatory Activities (MedDRA) and will be classified by PT (Preferred Term) and SOC (System Organ Class)
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Timepoint [1]
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Three years.
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Secondary outcome [1]
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Raw values and change from the Inclusion Visit in CAJIS (Cumulative Analogue Joint Involvement Scale) total score
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Assessment method [1]
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Timepoint [1]
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Every six months up to three years
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Secondary outcome [2]
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Raw values and shift from the Inclusion Visit in the use of assistive devices and adaptations for daily living
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Assessment method [2]
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The use of assistive devices and adaptations for daily living will be collected using the FOP assistive devices assessment.
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Timepoint [2]
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Every six months up to three years
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Secondary outcome [3]
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Raw values and change from the Inclusion Visit in % of worst score for total score, upper extremities subscore and mobility subscore
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Assessment method [3]
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Using the adult form of the FOP-PFQ (FOP-Physical Function Questionnaire) for all participants
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Timepoint [3]
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Every six months up to three years
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Secondary outcome [4]
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Frequency of healthcare services utilization
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Assessment method [4]
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Timepoint [4]
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Three years
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Secondary outcome [5]
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Raw values and change from the Inclusion Visit in observed and % predicted FVC (Forced Vital Capacity)
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Assessment method [5]
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Timepoint [5]
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Every six months up to three years
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Secondary outcome [6]
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Raw values and change from the Inclusion Visit in observed and % predicted FEV1 (Forced Expiratory Volume in One Second)
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Assessment method [6]
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Timepoint [6]
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Every six months up to three years
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Secondary outcome [7]
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Raw values and change from the Inclusion Visit in absolute and % predicted FEV1/FVC ratio
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Assessment method [7]
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Timepoint [7]
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Every six months up to three years
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Secondary outcome [8]
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Raw values and change from the Inclusion Visit in observed and % predicted DLCO (Diffusion Capacity of the Lung for Carbon Monoxide)
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Assessment method [8]
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Timepoint [8]
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Every six months up to three years
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Secondary outcome [9]
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Raw values and change from the Inclusion Visit in physical and mental function (mean global physical and mental health score converted into T-scores)
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Assessment method [9]
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Using the adult form of the PROMIS (Patient Reported Outcomes Measurement Information System) Global Health Scale for all participants
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Timepoint [9]
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Every six months up to three years
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Secondary outcome [10]
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Raw values and change from the Inclusion Visit in number of investigator-reported flareups, flare-up outcomes (new bone growth, restricted movement) and flare-up duration by body location and overall;
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Assessment method [10]
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Timepoint [10]
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Every six months up to three years
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Secondary outcome [11]
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Percentage of participants with new bone growth overall and by flare-up status
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Assessment method [11]
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Timepoint [11]
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Every six months up to three years
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Eligibility
Key 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.
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Minimum age
14
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
14/03/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
3/01/2025
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Actual
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Sample size
Target
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Accrual to date
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Final
61
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
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Royal North Shore Hospital - Saint Leonards
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Recruitment postcode(s) [1]
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2065 - Saint Leonards
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Minnesota
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Country [3]
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United States of America
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State/province [3]
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Pennsylvania
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Country [4]
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Argentina
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State/province [4]
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Buenos Aires
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Country [5]
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Brazil
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State/province [5]
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Sao-Paulo
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Country [6]
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Canada
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State/province [6]
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Toronto
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Country [7]
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France
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State/province [7]
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Paris
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Country [8]
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Italy
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State/province [8]
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Genoa
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Country [9]
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Spain
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State/province [9]
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Colmenar Viejo
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Country [10]
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Sweden
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State/province [10]
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Umeå
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Country [11]
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United Kingdom
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State/province [11]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Ipsen
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05027802
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Ipsen Medical Director
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Address
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Ipsen
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05027802
Download to PDF