18. Are there any risks for my child to be exposed to study procedures such as MRI, x‐ rays, DXA, or blood sampling? Magnetic resonance imaging (MRI), X-rays, and blood sampling are important procedures needed to monitor the safety and
efficacy of palovarotene during the MO‐Ped Trial.
Risks related to these procedures are minimized when done by experienced personnel at study sites who are familiar with them.
There is no radiation exposure from the MRI scan. However, because children will have to lie still for up to 1 hour, it may be necessary to
sedate your child. The risks of sedation will be explained during the enrollment process.
The radiation exposure of the x‐rays and DXA (dual energy X – ray absorptiometry) represent minimal risk as they are well below
the annual background radiation of 3 mSv (millisieverts, a measure of radiation dosage) in the US, and belowthe 5 mSv per year
recommended by the US Food and Drug Administration (FDA).
19. Why is it necessary to live within a country that is hosting a MO‐Ped Trial site? As an investigational medicinal product, palovarotene can only be administered to human study subjects under special authorization by the national authorities within each country. Subjects must live in a country where the national authorities have approved the clinical study investigating palovarotene’s use for the treatment of MO. This authorization is necessary to import palovarotene into the country and
to perform home assessments within the country. Please look on (NCT03442985) for the most current information on clinical study sites and eligibility criteria.
20. How do I get to the clinical study site‐ Will my travel costs be reimbursed? Clementia has hired a travel services company that specializes in clinical studies. All reasonable costs associated with participating in the
MO‐Ped Trial will be paid for by Clementia, including air flight travel, ground travel and accommodations for study subjects
and a parent/caregiver. Travel to the study site is expected once every 6 months for examinations and assessments. Additional assessments are performed at home or if allowed, at a local medical facility between these study site visits.
21. Where are the targeted MO‐Ped Trial sites?
We anticipate up to 30 international study sites across approximately 12 countries to enroll approximately 240 patients in total. Each site must receive national and local approval to accept subjects. The time for these approvals may vary, so some sites would be open sooner than others. The following are countries where sites are planned: