FOP drug trial is on! What does that mean??

Posted by Karen - November 9th, 2014

It’s actually happening. Taking place. Occurring right now! Yep, a trial of a possible drug for fibrodysplasia ossificans progressiva is underway. For me, this is exciting beyond words. It’s something I’ve been waiting for since my daughter Miranda was diagnosed with FOP in April of 2007.

Many people who have donated to my family’s annual FOP fundraising drives may be wondering what the scoop is. Also, maybe some folks with FOP are wondering if they can get in on the trial, but they’re not sure if they can. What are the particulars? How is this being carried out? Who gets to participate? Well, I’m about to answer those questions. This is my blog post about “stuff you want to know about the current FOP drug trial”…

Drug cartoon

In no special order, here are some basic details:

– The potential drug being tested is called Palovarotene. It is supposed to work on a new FOP soft tissue flare-up (ie, not on existing FOP bone). If it works as expected/hoped, it will interrupt the flare-up process, diminish swelling, and “short circuit” the early steps leading to pre-bone cartilage formation. In the best case scenario, it will end the flare-up entirely, before any FOP bone can form. The clinical test ongoing right now is to find out if the drug seems to work as expected.

– The trial, which is called a “phase two” trial, is being run by Clementia Pharmaceuticals, which is a Montreal-based company, in connection with scientists in the USA (Pennsylvania and California) and France. Three out of four participants receive Palovarotene, while one out of four receives a placebo.

– The trial started in July of 2014, and at that time was set to take place over an 18 month period (ie, until roughly December of 2015). I’m not sure if that time period still applies, but as far as I know it does. Of course, any one individual’s involvement in testing will likely not cover anything close to 18 months (probably just a number of weeks).

– People eligible to participate in the trial are individuals age 15 and up who are having a newly active FOP flare-up in a shoulder, arm, hip, or leg (I think there may also be some requirements as to whether the limb was previously restricted by FOP bone). And here’s a really, REALLY important bit – to participate, do you have to have somehow signed up for this early on, or got your name on a list, or something like that ? NO! Nope you don’t! As I understand it, at ANY TIME during the time the drug trial is on, a person in the right age group who fits the right criteria can get in on the trial. It’s as simple as that. See, one of the big challenges with FOP is that you can’t just take any random group of people and start giving them the drug – you have to wait until FOP patients are actually having the right kind of flare-up. So, it’s vital and crucial for people in the right age group and with the right kind of FOP flare-up to step up and volunteer to take part. If these folks don’t come forward and offer themselves up, the test will fail for lack of participants.

– Another important point is that people who take part have to travel to one of three testing locations in Pennsylvania, California and France. I understand the testing protocol requires a few trips of a few days at a time to one of the three sites. Now, travel costs are covered for participants, so no worries there. But – the bigger question is whether the person is mobile enough to travel by land or air to one of the testing sites. Adults with FOP, if you’re sitting on the fence and debating whether to take part in this trial – please keep this point in mind and remember that not everyone who might qualify for the testing is physically able to travel (or may be too far away to participate). So, it’s really up to folks with the right amount of mobility to get in on it, as well as people not too far from the test sites (NOTE – Canadians who can travel to the USA can take part).

– Here’s one cool bit – if a person takes part in the testing, and then later on during the test period gets another flare-up, the person is eligible to receive Palovarotene as part of an “extension” study. While you only have a 75% chance of testing Palovarotene during the first part of testing, you definitely get the Palovarotene during the extension study.

– If this first round of testing on Palovarotene works as hoped, the researchers will move on to considering further “phase two” tests. Another type of phase two test could and probably would involve children, and could also involve FOP flare-ups on the torso or head. This testing would be easier to carry out, because the potential pool of participants is quite a bit bigger – children tend to have more frequent flare-ups than adults, meaning more likelihood of being able to take part, and also children tend to be more mobile than adults with FOP (ie, travel is easier). So, if the FOP researchers can just get over this first hump of getting enough adults to participate in the current test…

For more information about Palovarotene drug testing (including how to get in on it), I urge you to go to the Clementia website at .

I will finish here with a plea – if you are an older teen or adult with FOP and you think you may have a flare-up which would let you qualify for testing, please please PLEASE look into getting involved. The FOP community is just too small to rely on the idea that “other people” will do it. There are no other people.

Here's what FOP is doing to the skeletons of FOP patients.

Here’s what FOP is doing to the skeletons of FOP patients.

And to those with FOP who are already participating in the trial, THANK YOU from the bottom of my heart.



3 Responses to “FOP drug trial is on! What does that mean??”

  1. Blair says:

    Excellent news and very informative post! I will watch with avid excitement and fervent hope!!

  2. Paula Zamudio says:

    Thanks Karen, for sharing this great news! This puts butterflies in my stomach. :)

  3. Marilyn Hair says:

    Thanks for a great summary of the Clinical Trial. Note that when you have a flare-up that qualifies for the study, you have to get to the study center and start the treatment within 7 days of the start of the flareup.

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