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.
Frequently Asked Questions on 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. Below you will find answers to questions you may have about the program.
If you have additional questions or are interested in receiving more information, please have your FOP physician send an email to: EarlyAccess@clementiapharma.com
1. In which countries do you plan to implement such a program?
Clementia’s goal is to provide access to investigational palovarotene to eligible FOP patients regardless of geographic location, subject to a patient’s eligibility, compliance with local regulatory requirements, and availability of investigational palovarotene supplies.
2. How are eligible patients requesting access to investigational palovarotene prioritized?
All FOP patients age of 4 or above, and following a request from their physician, will be considered. To determine eligibility, each case requires an individual assessment confirming that program conditions are met. There are many factors that determine eligibility, such as; meeting the medical eligibility criteria, a determination by the treating physician that the potential benefits of investigational palovarotene outweigh the potential risks, local regulations, and available supplies of investigational palovarotene.
3. I am actively participating in the MOVE Trial. Can I leave the clinical trial and access investigational palovarotene in the EAP?
No. The primary focus of the EAP is to provide access to investigational palovarotene to FOP patients who were unable to participate in the MOVE Trial, and for whom there is no comparable or satisfactory alternative therapy, with their treating physicians’
support. In order to successfully achieve our goal and make available an approved treatment option to as many FOP patients as possible, the successful completion of the MOVE Trial is a critical step. Patient withdrawals from the study may compromise the ability of the clinical trial to effectively evaluate the safety and efficacy of investigational palovarotene, interfere with the regulatory review and approval process, and otherwise affect the potential development of investigational palovarotene.
4. Are patients with Multiple Osteochondromas (MO) able to access investigational palovarotene through Clementia’s Early Access Program (EAP)?
No, Clementia’s EAP is designed for eligible FOP patients who meet the program criteria and who are unable to participate in the Phase 3 MOVE Trial. Clinical data generated to date in the Phase 2 study in FOP patients suggests that the potential benefits of treatment for this specific patient population outweigh potential or known risks of investigational palovarotene. Investigational palovarotene is at an earlier stage of development for patients with MO and is currently being evaluated in a Phase 2 PVO-2A-201 trial, MO-Ped. Our focus in patients with MO is to generate data supporting potential efficacy and safety in this patient population, prior to considering an EAP program, as this is the best path forward to help the whole MO population as soon as possible. We are currently recruiting children aged 2-14 years old with a confirmed genetic diagnosis for the Ext1 or Ext2 mutations for participation in the MO-Ped Trial. Further information can be found via this link: https://clinicaltrials.gov/ct2/show/NCT03442985?cond=osteochondroma&draw=2&rank=4. MO patients should discuss with their treating physicians and clinical trial investigators if they are eligible for MO-Ped.
Once again, for more information please have your treating FOP physician send an email to: EarlyAccess@clementiapharma.com.