Our lead product candidate, palovarotene, is an oral small molecule that has shown potent activity in preventing abnormal new bone formation as well as fibrosis in a variety of tissues. We are developing palovarotene for the treatment of fibrodysplasia ossificans progressiva (FOP), multiple osteochondromas (MO) and dry eye disease.
We have completed enrollment in our Phase 3 MOVE Trial for palovarotene in fibrodysplasia ossificans progressiva (FOP) and we are currently enrolling patients in our Phase 2 MO-Ped Trial for palovarotene in multiple osteochondromas (MO).
In addition, palovarotene has been shown to exert multiple effects in various tissues including in ocular tissues, where RARγ agonists generally demonstrate anti-fibrotic properties. In October 2018, we initiated and enrolled the first subject in a Phase 1 trial that will evaluate the safety, tolerability, and pharmacokinetic profile of single and multiple ascending doses of palovarotene ophthalmic solution in healthy volunteers.
For complete information on our palovarotene studies, please visit clinicaltrials.gov and search for palovarotene.
Phase 3 MOVE Trial for FOP
Trial ID Number: PVO-1A-301
Clinicaltrial.gov Identifier: NCT03312634
- Be clinically diagnosed with FOP and between the ages of 4 and 65 years
- Reside in one of the countries where a clinical site is located, due to regulatory requirements (please see clinicaltrials.gov for an updated list of sites)
- Have been flare-up free the 4 weeks before enrolling
- Can undergo all study procedures, including low-dose whole-body CT (WBCT) scans
- Meet all other enrollment criteria (please see clinicaltrials.gov)
Enrollment is completed in August 2018. All participants will take palovarotene daily for 24 months, with increased dosing in the event of a flare up. Participants are required to visit the clinical site at enrollment, and at months 6, 12, 18 and 24 during which they will undergo WBCT scans and other assessments. Safety assessments will be performed at months 3, 9, 15 and 21 at the participant’s home.
- Chronic treatment: All participants will receive 5 mg of palovarotene once daily for 24 months.
- Flare-up treatment: In the event of a flare-up, participants will receive 20 mg palovarotene once daily for 4 weeks, followed 10 mg palovarotene once daily for 8 weeks, (which may be extended by the investigator until the flare-up has resolved).
- All dosing will be weight-adjusted for those under the age of 18 years who have not reached skeletal maturity.
- No placebo will be administered.
To contact Clementia for more information on the MOVE Trial, please click here.
MOVE Trial FAQ’s
Phase 2 MO-Ped Trial for MO
Trial ID Number: PVO-2A-201
Clinicaltrial.gov Identifier: NCT03442985
- Be between the ages of between 2-14 years
- Have symptomatic MO
- Have confirmed EXT1 or EXT2 mutations
- Reside in one of the countries where a clinical site is located, due to regulatory requirements
- We intend to enroll 80 participants per dosing arm (240 participants in total).
- Treatment regimen: Oral palovarotene at multiple doses (5 mg and 2.5 mg) vs. placebo
- Treatment Period: 24-month treatment with interim analysis at 12 months
To contact Clementia for more information on the MO-Ped Trial, please click here.
MO-Ped Trial FAQ’s
At Clementia, our mission is to bring innovative and effective treatments to individuals who currently have none. With this goal in mind, we are developing palovarotene, an investigational drug for fibrodysplasia ossificans progressiva (FOP) and multiple osteochondromas (MO). We support expanded access to eligible investigational drugs for people with a serious or life-threatening disease or condition who meet specific requirements. Click here to read our early access policy.
Clementia Early Access Program for FOP
Clementia is committed to providing access to eligible fibrodysplasia ossificans progressiva (FOP) patients who are unable to participate in a clinical trial and have urgent medical needs, through our Early Access Program (EAP), where applicable laws permit and under defined conditions. All physician requests will be carefully evaluated against the parameters of our EAP policy.
If you have questions or are interested in receiving more information, please have your FOP physician send an email to: GlobalMedInfo@Ipsen.com