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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
Scientific title
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Palovarotene in Subjects With Multiple Osteochondromas
Secondary ID [1] 0 0
2017-002751-28
Secondary ID [2] 0 0
PVO-2A-201
Universal Trial Number (UTN)
Trial acronym
MO-Ped
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Exostoses, Multiple Hereditary 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

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annualized Rate of New Osteochondromas (OCs)
Timepoint [1] 0 0
Month 12
Secondary outcome [1] 0 0
Mean Change From Baseline in the Total Volume of New OCs at Month 12
Timepoint [1] 0 0
Baseline (Day 1) and Month 12
Secondary outcome [2] 0 0
Percentage of Participants With No New OCs
Timepoint [2] 0 0
Month 12
Secondary outcome [3] 0 0
Annualized Rate of New or Worsening Deformities
Timepoint [3] 0 0
Month 12
Secondary outcome [4] 0 0
Annualized Rate of MO-Related Surgeries
Timepoint [4] 0 0
Month 12
Secondary outcome [5] 0 0
Maximum Observed Plasma Drug Concentrations at Steady State (Cmax,ss) of Palovarotene
Timepoint [5] 0 0
Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose
Secondary outcome [6] 0 0
Minimum Observed Plasma Drug Concentrations at Steady State (Cmin,ss) of Palovarotene
Timepoint [6] 0 0
Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose
Secondary outcome [7] 0 0
Time to Maximum Observed Drug Concentration at Steady State (Tmax,ss) of Palovarotene
Timepoint [7] 0 0
Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose
Secondary outcome [8] 0 0
Area Under the Plasma Concentration-Time Curve at Steady State From Time 0 to 24 Hours After Dosing (AUC0-24,ss) of Palovarotene
Timepoint [8] 0 0
Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose
Secondary outcome [9] 0 0
Number of Participants With Palatability of Sprinkled Palovarotene and Placebo
Timepoint [9] 0 0
Day 1 and Month 1

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.

Key
Minimum age
2 Years
Maximum age
14 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
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
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
22/03/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
30/10/2020
Sample size
Target
Accrual to date
Final
193
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Westmead Children's Hospital - Westmead
Recruitment postcode(s) [1] 0 0
2145 - Westmead
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
District of Columbia
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
Oregon
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
Texas
Country [11] 0 0
Belgium
State/province [11] 0 0
Antwerp
Country [12] 0 0
Canada
State/province [12] 0 0
Ontario
Country [13] 0 0
Canada
State/province [13] 0 0
Quebec
Country [14] 0 0
France
State/province [14] 0 0
Paris
Country [15] 0 0
France
State/province [15] 0 0
Toulouse
Country [16] 0 0
Italy
State/province [16] 0 0
Emilia-Romagna
Country [17] 0 0
Japan
State/province [17] 0 0
Aiti
Country [18] 0 0
Japan
State/province [18] 0 0
Osaka
Country [19] 0 0
Netherlands
State/province [19] 0 0
Noord-Holland
Country [20] 0 0
Portugal
State/province [20] 0 0
Coimbra
Country [21] 0 0
Spain
State/province [21] 0 0
Madrid
Country [22] 0 0
Turkey
State/province [22] 0 0
Izmir
Country [23] 0 0
Turkey
State/province [23] 0 0
Istanbul
Country [24] 0 0
United Kingdom
State/province [24] 0 0
London
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Manchester
Country [26] 0 0
United Kingdom
State/province [26] 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)?
No
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/NCT03442985