This is an exciting time for the FOP community with a number of clinical trials investigating effective treatments for FOP happening globally.
Whilst FOP is a considered an ultra-rare condition, there are many research institutions, pharmaceutical companies, and other organisations activity looking into an effective treatment and cure for FOP.
As of January 2020, there are 16 organisations looking into a range of drugs that may provide that treatment and cure that we all so desperately want and need. Whilst not all of these drugs will become a treatment, the learning will inform researchers’ work and hopefully, will ultimately help to find the drug/s that will make FOP a manageable condition.
FOP Friends actively funds the FOP research team at the University of Oxford, led by Professor Alex Bullock. Without the financial support from us, they would not be able to continue the focused work they are doing to find a treatment for FOP.
FOP Friends also works in partnership with the IFOPA and other organisations to support the Accelerating Cures and Treatments (ACT) for FOP. The programme encourages innovative research into finding a way to treat FOP.
See all the drugs for FOP, currently in development here: IFOPA: FOP Drugs in Development Jan 2020
Active Trials – Recruiting
FOP Connection Registry
The FOP Registry is a global, voluntary database that collects demographic and disease information directly from individuals with FOP through a secure website. An App has also just been released. It is sponsored by the IFOPA.
The FOP Registry is open to all individuals with FOP and is available in seven languages: English, French, German, Italian, Portuguese, Russian and Spanish.
Every person living with FOP has unique information to share. The Registry collects this information to help further research to find treatments and, one day, a cure for FOP.
Data from the Registry will be used to help researchers, clinicians and drug developers to better understand FOP. Completing the Registry is one way people affected by FOP can help to make a difference.
In recognition of the time it can take to complete a survey (around 1 hour, every six months), the IFOPA has implemented a Gift Card scheme. Every time a participant completes a survey, they will receive a $25 (or equivalent) gift card as a way of saying thank you. The gift-card reward program is open to new and existing FOP Registry participants. Learn more here: Registry Rewards
Visit the FOP Registry website to register and make a difference.
Active Trials – Not Recruiting
Following on from the promising studies in mice, US biotechnology company Regeneron, introduced their first clinical trial of anti-Activin A antibody (REGN2477) in humans in 2016. After the success of highlighting that the drug is well-tolerated amongst a cohort of healthy volunteers, Regeneron announced their Phase 2 LUMINA-1 trial, recruiting participants living with FOP. The potential drug is administered monthly via injection (IV) and eligibility criteria included age requirements of 18 – 60-years.
US biotechnology company, Clementia (now Ipsen), announced in 2016 that their Phase 2 study of their potential drug had shown positive trends reducing bone formation, pain and severity of flare-ups amongst individuals living with FOP. Although these trends were not found to be statistically significant, this is still positive news, as showing that something is statistically significant is a challenge for all trials of rare diseases, where the number of participants (40 in this trial) makes it harder to prove significant trends.
After receiving Breakthrough Therapy status in the US, the company launched their Phase 3 MOVE Trial for palovarotene. The potential drug is administered daily via oral tablets and eligibility criteria included age requirements of 4-years and older. In 2019, Clementia (now Ipsen) announced they were in the process of filing the first-ever application seeking regulatory approval for their FOP drug, palovarotene.
Update January 2020
IPSEN have just announced that they have decided to pause dosing in patients taking palovarotene.
The IDMC’s (Independent Data Monitoring Committee’s) key recommendation is that the trial is paused “Based on encouraging therapeutic activity signals” for review.
That a “pause” will allow for further analysis can be carried out to try to achieve the best outcome for patients.
In layman’s terms, they need to ensure “success” criteria are appropriate to have the best chance for ultimate approval and have to work with the groups and regulatory bodies that set these standards to ensure they approve the next steps and future protocol for the trial.
At present we do not know the timescales involved but we will be in regular communication with the IPSEN team for updates and will share updates as they arrive.
** For patients on the trial. This does not mean treatment and the trial has been discontinued, it is a pause in the dosing and you need to speak with your Principle Investigators regarding ongoing assessments which will continue.
This has not changed IPSEN’s commitment to the FOP community and the continuation of FOP programmes.
If you have any questions please feel free to send them via direct message and we can collate and aim to provide answers.
Download IPSEN’s letter to the community here: IPSEN-LETTER
Update: December 2019
In December 2019, Ipsen issued a partial clinical hold following recent safety reports submitted by the company to the FDA of cases of early growth plate closure in paediatric patients with FOP treated with palovarotene. There has been no changes for adult patients on the trial. No new patients will be enrolled while the partial clinical hold is in effect.
Ipsen said: “…the safety of patients is always a top priority, and the company is committed to ensuring the safe and effective use of its medicines. Ipsen is committed to researching and developing therapies for children and adults living with FOP and MO, two rare and devastating bone diseases with no current therapeutic treatment options. Ipsen is committed to working diligently with the FDA to provide all requested information with the goal of resolving the partial clinical hold. Ipsen continues to prepare the FDA New Drug Application (NDA) filing for palovarotene in acute/flare-up FOP”.
Read the full statement here: Ipsen Press Release.
Clementia Pharmaceuticals has been acquired by the French pharmaceutical company Ipsen: www.ipsen.com/press-releases/ipsen-completes-acquisition-of-clementia-pharmaceuticals/
Things to consider before committing to a Clinical Trial
Taking part in a clinical trial is a big decision, and one that shouldn’t be taken lightly. There are risks associated with participating in trials so it is important that you read all information available; understand the potential risks and benefits; and make an informed decision regarding your or your child’s involvement in a study.
The IFOPA has produced an excellent list of questions for you to consider, if you are thinking of enrolling yourself or a child onto a clinical trial: Questions to ask.
The NHS has general advice regarding clinical trials which you can read here: Information about Participating in a Clinical Trial NHS
You can also read about other people’s experiences of taking part in a trial here: National Institute of Health Research UK
The IFOPA has produced a webinar, presented by their Director or Research Development & Partnerships, Adam Sherman. This gives an overview of the drug development and approval process in the US and the role that clinical studies and trials play in this process. Clinical Studies and Trials: Your Choice, Your Role, Your Responsibility.
If you have any questions regarding involvement in clinical trials, please do not hesitate to get in touch with us at firstname.lastname@example.org.