Palovarotene is an investigational medicinal product that Clementia is developing as an orally administered treatment for FOP. Palovarotene, a retinoic acid receptor gamma agonist (RARγ), is thought to prevent and/or minimize heterotopic ossification (HO) in FOP by disruption the cell’s signaling system.
Palovarotene has completed a Phase 2 program which evaluated four different dosing regimens. Data from the Phase 2 program supported the U.S. Food & Drug Administration (FDA) designation of palovarotene as Breakthrough Therapy, which is granted when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies.
The Phase 3 trial is a global, multi-center, open-label (no placebo) trial. Approximately 80 adults and children over 4 years of age with the classic FOP mutation (R206H mutation) will be needed to complete the Phase 3 clinical trial. The trial is designed to evaluate whether a daily dosing regimen of palovarotene, with higher doses during times of flare-ups, will reduce the formation of new HO.
Those that enroll in the Clementia trial will receive treatment with palovarotene for two years. The data from the trial may be used to support a drug application to the regulatory authorities for marketing authorization.
The trial will be conducted internationally in the following countries:
- South Africa
- United Kingdom
- United States
Frequently Asked Questions
Who can participate in the palovarotene trial?
The palovarotene Phase 3 study will enroll male or female individuals at least 4 years old (weighing at least 10 kg) and who have a clinical diagnosis of FOP, due to the classic mutation (R206H mutation). Due to local regulations, the participant needs to live in a country where a clinical study site is located. Additionally, four weeks must have elapsed since the end of the last flare-up before enrollment. Additional exclusion criteria regarding medical history, medication use, allergies, and laboratory values are available on clinicaltrials.gov.
In what countries will the study be conducted?
Clementia anticipates conducting the Phase 3 trial in approximately 20 sites across 14 different countries. It should be noted that the application and approval process will vary greatly from country to country. So, some sites may be enrolling while others sites are still going through the approval process. As soon as a site is ready to enroll, Clementia will update the list on clinicaltrials.gov.
If I’m currently enrolled in Clementia’s Natural History Study, can I participate in the palovarotene Phase 3 trial?
Individuals participating in Clementia’s Natural History Study who meet the enrollment criteria can enroll in the MOVE study.
If I participated in the palovarotene Phase 2 program, can I enroll in the Phase 3 trial?
No, the enrollment into the Phase 3 trial is limited to individuals who have not previously participated in a palovarotene study. The ongoing Phase 2 open-label study is being amended so that all participants (including children) can receive the chronic/flare-up dosing regimen and will no longer need to travel to the clinical site at the time of a flare-up.
How long will the palovarotene Phase 3 trial last?
The palovarotene trial is a two-year treatment trial.
How many people can participate in the trial?
The palovarotene trial will enroll approximately 80 adults and children with FOP.
How many people will be on placebo vs actual study drug?
There will be no placebo group. Instead, all trial participants will receive palovarotene over the course of the study. The results for these 80 individuals will be compared to the data obtained in Clementia’s Natural History Study.
How will palovarotene be dosed in the Phase 3 trial?
Palovarotene will be given chronically at 5 mg once daily. During flare-up activity, the dose will be increased to 20 mg once daily for 4 weeks followed by 10 mg once daily for 8 weeks. The dose will be adjusted for weight in children who are still growing.
What are the travel commitments and requirements to participate in the study?
Clementia has hired a travel services agent that is specialized in assisting with clinical studies. All reasonable costs associated with participating in this study will be paid for by Clementia, including travel and accommodations for you and a caregiver. Travel to the investigational site is expected once every 6 months for examinations and assessments, with home assessments performed between clinic visits.
Who do I contact if I’m interested in learning more about Clementia’s trial?
Have your doctor contact Clementia clinical trial resource line or email address:
You can also refer to the MOVE Trial Frequently Asked Questions document available in English and eight other languages.