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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03442985
Registration number
NCT03442985
Ethics application status
Date submitted
11/10/2017
Date registered
22/02/2018
Titles & IDs
Public title
An Efficacy and Safety Study of Palovarotene for the Treatment of MO
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Scientific title
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Palovarotene in Subjects With Multiple Osteochondromas
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Secondary ID [1]
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2017-002751-28
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Secondary ID [2]
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PVO-2A-201
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Universal Trial Number (UTN)
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Trial acronym
MO-Ped
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Exostoses, Multiple Hereditary
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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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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Palovarotene 2.5 mg
Treatment: Drugs - Palovarotene 5.0 mg
Other interventions - Placebo
Experimental: Palovarotene 2.5 mg daily regimen -
Experimental: Palovarotene 5.0 mg daily regimen -
Placebo comparator: Placebo regimen -
Treatment: Drugs: Palovarotene 2.5 mg
Subjects received a weight-adjusted dose equivalent of 2.5 mg palovarotene, once daily, for up to 24 months.
Treatment: Drugs: Palovarotene 5.0 mg
Subjects received a weight-adjusted dose equivalent of 5.0 mg palovarotene, once daily, for up to 24 months.
Other interventions: Placebo
Subjects received placebo, once daily, for up to 24 months.
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Intervention code [1]
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Treatment: Drugs
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Intervention code [2]
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Other interventions
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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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Annualized Rate of New Osteochondromas (OCs)
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Assessment method [1]
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The annualized rate of new OCs was assessed by whole-body magnetic resonance imaging (MRI) (that is, the total number of new OCs divided by the time in years between the baseline and latest post-baseline MRI).
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Timepoint [1]
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Month 12
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Secondary outcome [1]
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Mean Change From Baseline in the Total Volume of New OCs at Month 12
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Assessment method [1]
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The change from baseline in the total volume of OCs was assessed by whole-body MRI. Baseline was defined as the last available value prior to first administration of study drug.
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Timepoint [1]
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Baseline (Day 1) and Month 12
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Secondary outcome [2]
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Percentage of Participants With No New OCs
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Assessment method [2]
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The percentage of participants with no new OCs as assessed by whole-body MRI. Participants with new OCs not identified by MRI due to surgical resection during the treatment period were categorized as having new OCs for this analysis.
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Timepoint [2]
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Month 12
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Secondary outcome [3]
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Annualized Rate of New or Worsening Deformities
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Assessment method [3]
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The annualized rate of new or worsening deformities as assessed by radiographic imaging of both upper and lower limbs.
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Timepoint [3]
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Month 12
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Secondary outcome [4]
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Annualized Rate of MO-Related Surgeries
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Assessment method [4]
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The MO-related surgeries included any procedure indicated for the treatment of MO, such as an excision of a symptomatic OC or correction of a limb deformity.
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Timepoint [4]
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Month 12
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Secondary outcome [5]
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Maximum Observed Plasma Drug Concentrations at Steady State (Cmax,ss) of Palovarotene
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Assessment method [5]
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The Cmax,ss of palovarotene was evaluated. The pharmacokinetic (PK) sampling was performed at Month 1. If samples could not be obtained at Month 1, then one additional attempt was made at a subsequent visit.
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Timepoint [5]
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Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose
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Secondary outcome [6]
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Minimum Observed Plasma Drug Concentrations at Steady State (Cmin,ss) of Palovarotene
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Assessment method [6]
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The Cmin,ss of palovarotene was evaluated. The PK sampling was performed at Month 1. If samples could not be obtained at Month 1, then one additional attempt was made at a subsequent visit.
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Timepoint [6]
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Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose
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Secondary outcome [7]
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Time to Maximum Observed Drug Concentration at Steady State (Tmax,ss) of Palovarotene
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Assessment method [7]
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The Tmax,ss of palovarotene was evaluated. The PK sampling was performed at Month 1. If samples could not be obtained at Month 1, then one additional attempt was made at a subsequent visit.
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Timepoint [7]
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Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose
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Secondary outcome [8]
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Area Under the Plasma Concentration-Time Curve at Steady State From Time 0 to 24 Hours After Dosing (AUC0-24,ss) of Palovarotene
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Assessment method [8]
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The AUC0-24,ss of palovarotene was evaluated. The PK sampling was performed at Month 1. If samples could not be obtained at Month 1, then one additional attempt was made at a subsequent visit.
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Timepoint [8]
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Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose
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Secondary outcome [9]
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Number of Participants With Palatability of Sprinkled Palovarotene and Placebo
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Assessment method [9]
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Palatability of palovarotene and placebo when sprinkled on specific foods as assessed with a 5-point hedonic face scale at the first dose (Day 1) and at Month 1 in all participants (including \<4 years old) who sprinkled the palovarotene or placebo onto a spoonful of specific foods. The hedonic face scale ranges from 1 to 5 where, 1= dislike very much, 2= dislike slightly, 3= neither like nor dislike, 4= like slightly, 5= like very much. Higher scores indicate positive outcome.
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Timepoint [9]
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Day 1 and Month 1
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Eligibility
Key inclusion criteria
Key
* Written, signed, and dated informed subject/parent consent and age-appropriate assent (performed according to local regulations).
* A clinical diagnosis of MO with disease-causing exostosin 1 or 2 gene mutations.
* Male or female from 2 to 14 years of age.
* Female subjects must be premenarchal at screening.
* A bone age at screening of 14 years or less.
* Symptomatic MO, defined as five or more clinically evident osteochondromas and a new or enlarged osteochondroma that occurred in the preceding 12 months, five or more clinically evident osteochondromas and the presence of a painful osteochondroma, a skeletal deformity, a joint limitation, or prior surgery for a MO-related complication.
* The ability to undergo whole body MRI with or without sedation/general anesthesia.
* Use of two effective methods of birth control during treatment, and for 1 month after treatment discontinuation, unless committed to true abstinence from heterosexual sex. Sexually active females of child-bearing potential must also agree to start effective methods of birth control at screening.
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Minimum age
2
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Maximum age
14
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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
* Weight under 10 kg.
* Other syndromic conditions such as Langer-Giedion or Potocki-Shaffer.
* Any subject with neurologic signs suggestive of spinal cord impingement.
* Concomitant medications that are strong inhibitors or inducers of cytochrome P450 3A4 activity.
* Amylase or lipase >2 times the above the upper limit of normal (>2×ULN) or with a history of chronic pancreatitis.
* Elevated aspartate aminotransferase or alanine aminotransferase above 2.5×ULN.
* Any surgical implant that is contraindicated for MRI.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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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
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Stopped early
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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
22/03/2018
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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
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Actual
30/10/2020
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Sample size
Target
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Accrual to date
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Final
193
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
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Westmead Children's Hospital - Westmead
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Recruitment postcode(s) [1]
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2145 - Westmead
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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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United States of America
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State/province [2]
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District of Columbia
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United States of America
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Florida
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United States of America
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State/province [4]
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Illinois
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United States of America
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State/province [5]
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Maryland
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United States of America
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State/province [6]
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Massachusetts
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Country [7]
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United States of America
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State/province [7]
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Minnesota
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United States of America
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Oregon
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Country [9]
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United States of America
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Pennsylvania
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United States of America
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Texas
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Belgium
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Antwerp
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Canada
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Ontario
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Canada
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Quebec
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France
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Paris
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France
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Toulouse
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Italy
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Emilia-Romagna
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Japan
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Aiti
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Japan
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Osaka
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Netherlands
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Noord-Holland
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Portugal
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Coimbra
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Spain
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Madrid
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Turkey
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Izmir
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Turkey
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Istanbul
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United Kingdom
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London
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United Kingdom
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Manchester
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United Kingdom
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State/province [26]
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Stanmore
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Clementia Pharmaceuticals Inc.
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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
This is a randomized, double-blind, placebo-controlled study comparing the safety and efficacy of 2 dosage regimens of palovarotene versus placebo in preventing disease progression in pediatric subjects with multiple osteochondromas (MO).
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Trial website
https://clinicaltrials.gov/study/NCT03442985
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Trial related presentations / publications
Inubushi T, Lemire I, Irie F, Yamaguchi Y. Palovarotene Inhibits Osteochondroma Formation in a Mouse Model of Multiple Hereditary Exostoses. J Bone Miner Res. 2018 Apr;33(4):658-666. doi: 10.1002/jbmr.3341. Epub 2017 Nov 30.
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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
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
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What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/85/NCT03442985/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/85/NCT03442985/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT03442985