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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 11 June 2019
Main ID:  NCT03442985
Date of registration: 11/10/2017
Prospective Registration: Yes
Primary sponsor: Clementia Pharmaceuticals Inc.
Public title: An Efficacy and Safety Study of Palovarotene for the Treatment of MO MO-Ped
Scientific title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Palovarotene in Subjects With Multiple Osteochondromas
Date of first enrolment: April 20, 2018
Target sample size: 240
Recruitment status: Recruiting
URL:  https://clinicaltrials.gov/show/NCT03442985
Study type:  Interventional
Study design:  Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).  
Phase:  Phase 2
Countries of recruitment
Australia Canada France Japan Netherlands Portugal Spain Turkey
United Kingdom United States
Contacts
Name:     Clementia Call Center (US/Canada Only)
Address: 
Telephone: 1-800-750-8710
Email: ClinicalTrials@clementiapharma.com
Affiliation: 
Key inclusion & exclusion criteria

Key Inclusion Criteria:

- 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 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.



Age minimum: 2 Years
Age maximum: 14 Years
Gender: All
Health Condition(s) or Problem(s) studied
Exostoses, Multiple Hereditary
Intervention(s)
Drug: Palovarotene 2.5 mg
Drug: Palovarotene 5.0 mg
Other: Placebo
Primary Outcome(s)
Annualized rate of new osteochondromas [Time Frame: Month 24]
Secondary Outcome(s)
Annualized rate of MO-related surgeries [Time Frame: Months 12 and 24/EOT]
Annualized rate of new or worsening deformities [Time Frame: Months 12 and 24/EOT]
Palatability [Time Frame: Day 1 and Month 1]
Palovarotene area under the concentration-time curve (AUC) [Time Frame: Month 1]
Total volume of osteochondromas [Time Frame: Months 12 and 24/end-of-treatment (EOT)]
Secondary ID(s)
PVO-2A-201
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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