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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03312634




Registration number
NCT03312634
Ethics application status
Date submitted
9/10/2017
Date registered
18/10/2017

Titles & IDs
Public title
An Efficacy and Safety Study of Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva.
Scientific title
A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)
Secondary ID [1] 0 0
2017-002541-29
Secondary ID [2] 0 0
PVO-1A-301
Universal Trial Number (UTN)
Trial acronym
MOVE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fibrodysplasia Ossificans Progressiva 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Injuries and Accidents 0 0 0 0
Other injuries and accidents

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Palovarotene

Experimental: Palovarotene Chronic/Flare-Up Regimen - Participants received 5 mg palovarotene once daily for up to 48 months; and 20 mg palovarotene once daily for 28 days, followed by 10 mg for 56 days for flareups. (Dosing was adjusted for weight in skeletally immature subjects.)


Treatment: Drugs: Palovarotene
Palovarotene was taken orally once daily at approximately the same time each day following a meal.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annualized New Heterotopic Ossification (HO)
Timepoint [1] 0 0
Baseline (within one month of screening/Day 1) and up to 24 months
Secondary outcome [1] 0 0
Percentage of Participants With Any New HO
Timepoint [1] 0 0
From Baseline (Day 1) up to end of 4-year follow-up period (approximately 57 months)
Secondary outcome [2] 0 0
Number of Body Regions With New HO
Timepoint [2] 0 0
From Baseline (Day 1) up to end of 4-year follow-up period (approximately 57 months)
Secondary outcome [3] 0 0
Percentage of Participants With Flare-Ups
Timepoint [3] 0 0
Month 12
Secondary outcome [4] 0 0
Ratio of Flare-Up Per Participant-Month of Exposure
Timepoint [4] 0 0
From Baseline (Day 1) up to end of 4-year follow-up period (approximately 57 months)

Eligibility
Key inclusion criteria
Key

* Written, signed, and dated informed subject/parent consent; and for subjects who are minors, age-appropriate assent (performed according to local regulations).
* Males or females at least 4 years of age.
* No flare-up symptoms within the past 4 weeks, including at the time of enrollment.
* Abstinent or using two highly effective forms of birth control.
* Accessible for treatment and follow-up; able to undergo all study procedures including low-dose WBCT (excluding head) without sedation.

Key
Minimum age
4 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Weight <10 kg.
* Concomitant medications that are strong inhibitors or inducers of cytochrome P450 (CYP450) 3A4 activity; or kinase inhibitors such as imatinib.
* Amylase or lipase >2x above the upper limit of normal (ULN) or with a history of chronic pancreatitis.
* Elevated aspartate aminotransferase or alanine aminotransferase >2.5x ULN.
* Fasting triglycerides >400 mg/dL with or without therapy.
* Female subjects who are breastfeeding.
* Subjects with uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal, endocrine, metabolic, ophthalmologic, immunologic, psychiatric, or other significant disease.
* Simultaneous participation in another clinical research study (other than palovarotene studies) within 4 weeks prior to Screening; or within five half-lives of the investigational agent, whichever is longer.
* Any reason that, in the opinion of the Investigator, would lead to the inability of the subject and/or family to comply with the protocol.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/11/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
7/09/2022
Sample size
Target
Accrual to date
Final
107
Recruitment in Australia
Recruitment state(s)
NSW,QLD
Recruitment hospital [1] 0 0
Royal North Shore Hospital - Saint Leonards
Recruitment hospital [2] 0 0
Queensland University of Technology - Woolloongabba
Recruitment postcode(s) [1] 0 0
2065 - Saint Leonards
Recruitment postcode(s) [2] 0 0
4102 - Woolloongabba
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Minnesota
Country [3] 0 0
United States of America
State/province [3] 0 0
Pennsylvania
Country [4] 0 0
Argentina
State/province [4] 0 0
Buenos Aires
Country [5] 0 0
Brazil
State/province [5] 0 0
SP
Country [6] 0 0
Canada
State/province [6] 0 0
Ontario
Country [7] 0 0
France
State/province [7] 0 0
Paris
Country [8] 0 0
Italy
State/province [8] 0 0
Liguria
Country [9] 0 0
Japan
State/province [9] 0 0
Bunkyo-ku
Country [10] 0 0
Spain
State/province [10] 0 0
Avinguda De Fernando Abril Martorell, Nº 106
Country [11] 0 0
Sweden
State/province [11] 0 0
Umeå
Country [12] 0 0
United Kingdom
State/province [12] 0 0
Stanmore

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Clementia Pharmaceuticals Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ipsen Medical Director
Address 0 0
Ipsen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03312634