MO-Ped
Research type
Research Study
Full title
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Palovarotene in Subjects with Multiple Osteochondromas
IRAS ID
236247
Contact name
Mars Skae
Contact email
Sponsor organisation
Clementia Pharmaceuticals Inc.
Eudract number
2017-002751-28
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
2 years, 3 months, 1 days
Research summary
Study PVO-2A-201 is a multicentre, randomised, double-blind, placebo-controlled, 25-month study of disease progression in children with a rare bone disease called Multiple Osteochondromas (MO).
This study compares the effect of two doses of a trial medication with placebo on the rates of formation of new bone lesions (osteochondromas, OC's), which interfere with movement or circulation, causing substantial pain and limb functional limitations, and the frequency of MO-related surgeries during the study.
To ensure consistency in assessments of OCs and deformities, imaging by whole body MRI and x-rays of the upper and lower extremities will be interpreted by a central laboratory using standard procedures and blinded to the treatment groups. The study will also compare the changes from baseline in the size of OCs as assessed by Magnetic Resonance Imaging (MRI), as well as pain due to OCs, and quality of life.
Participants will receive a thorough examination by whole body MRI and upper/lower limb x-rays to determine the number, size, and location of OCs, joint deformities, and other skeletal abnormalities. However, prior to enrolment, any participant under 7 years old, or deemed by the Investigator to require procedural sedation, will be assessed by paediatric sedation team to determine the participant’s tolerance for MRI and the level of sedation required for the MRI.
240 participants will be randomised to one of two active treatments or placebo on Study Day 1 with a 1:1:1 allocation. Alternating on-site and remote visits (at home or at a local medical facility) will occur every 3 months unless the Investigator deems a site visit necessary. Urine pregnancy tests will be performed monthly for females of childbearing potential.Participants will undergo all procedures specified in the study protocol.
REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
18/NW/0742
Date of REC Opinion
21 Dec 2018
REC opinion
Further Information Favourable Opinion