We have an exciting announcement! The first two clinical study sites have been selected
for the MO-Ped Drug Trial. They are Shriners Hospital, Philadelphia, PA and the MHE Center at the Paley Institute,
West Palm Beach, FL. The FAQS sheet provided by Clementia gives detailed explanations of the stages and
procedures involved in the trial. After reviewing this information, if you have questions or
if you are interested in having your child participate, do not contact the study site; contact
Clementia directly at http://clementiapharma.com/clinical-trials/#mo-ped-trial
Click here for a printable PDF of the MO-Ped Trial Faqs.
MO‐Ped Trial FAQs
1. Is MO, HMO, HME, and MHE all the same disease? Yes. Multiple Osteochondromas (MO) has many names, including Hereditary Multiple Osteochondromas (HMO), Multiple Hereditary (or hereditary multiple) Exostoses (MHE or HME), or familial exostoses. The different names come from the use of “osteochondromas” or “exostoses” to describe the bone growths that occur with MO and the hereditary nature of the disease. The term osteochondromas is preferred since it is more accurate medical description of the formation of
cartilage growths that later ossify (or turn to bone), and we will use the term MO to refer to the condition throughout this document.
2. What is the MO‐Ped Trial?
The MO Ped Trial (Multiple Osteochondromas in Pediatrics) is a global Phase 2 clinical study designed to evaluate the efficacy
and safety of two dosages of palovarotene versus placebo (i.e. sugar pill), in pediatric subjects. Each subject will receive 2 years of study drug, and the study ends when all phases of the study including the last visit or any scheduled follow‐up procedures
have been completed. All subjects who complete the MO‐Ped Trial may be eligible to enroll in a follow up study. In this follow up study, all enrolled subjects will receive palovarotene. If the therapeutic efficacy of palovarotene is unexpectedly large, the trial may
end early at a 1‐year interim analysis.
3. Who can participate in the MO‐Ped Trial?
The MOPed Trial will enroll both male and female pediatric subjects from 2 to 14 years of age to assess palovarotene’s effect on
osteochondroma formation and other related events that impact a subject’s physical function and quality of life. We are testing if
palovarotene is effective in preventing new osteochondroma formation and growth, which are thought to occur only while the
growth plate is active. The 14‐year old age and bone age limit would permit at least 2 years of observation while the growth plate is active. All individuals must weigh at least 10 kg, and must have a genetic diagnosis of Multiple Osteochondromas
(MO) with exostosin 1 or 2 (Ext1 or Ext2) mutations confirmed by a central laboratory. Both individuals with a spontaneous
mutation or an inherited mutation are eligible for this trial.
Please note that an individual interested in participating in the MOPed Trial does not have to be genetically tested before the trial
starts, as the gene test will take place during the screening visit.