Clinical trial results are in!

Posted by Karen - October 16th, 2016

On Friday, Clementia Pharmaceuticals sent out an exciting press release. This was MUCH anticipated in the fibrodysplasia ossificans progressiva community. The subject of the release was, TA DA, the results of the first ever post-gene-discovery-era clinical test of a drug for FOP!

So here’s the scoop… In 2014, Clementia  started a Phase 2 clinical test of palovarotene, which I will call “PV” for short (they were able to skip over Phase 1 – testing for toxicity using unaffected patients – because another pharma company had done Phase 1 while considering PV for a different disease). In simplest terms, PV has been shown in pre-clinical tests to interfere with cartilage formation, and if you can stop cartilage from forming, then you don’t get that nasty, unwanted FOP bone. Anyway, to qualify for the test, a person had to be in the first few days of a new FOP flare-up on a major joint or limb, and have an adult skeleton (i.e., largely finished childhood skeletal growth). Subjects were divided randomly and anonymously into 3 groups – the first group did 2 weeks at 10 mg of daily PV and then 4 weeks at 5 mg daily PV (the “10/5 group”), the second group did 2 weeks at 5 mg of daily PV and then 4 weeks at 2.5 mg daily PV (the “5/2.5 group”), and a placebo group (they took pills, but the pills contained no PV).  This test carried on into 2015, and later in 2015, was expanded to include children age 8 and up. [NOTE – my daughter didn’t take part in the test. At age 10 in 2015, she was in the right age group following the expansion, but didn’t develop any flare-ups in the necessary body parts to be eligible.] The final subject of the trial was enrolled in early 2016, and once that person’s test regime was finished, Clementia began assessing the results. Friday’s press release set out the nature of their findings.

I’m going to now take a stab at explaining what was revealed. Please keep in mind that I’m not a scientist, so I might mess something up… In any event, one of the findings was that the placebo group had a 2.6X higher risk of forming any new FOP bone (“heterotopic ossification”, or “HO” for short) as compared to the 10/5 group. Also, among all test subjects who DID develop new HO, both the 10/5 group and the 5/2.5 group developed less HO than the placebo group. Another result was that subjects in the 10/5 group reported less pain, and a reduction in duration of flare-up symptoms as compared to the other 2 groups.

Dr. Fred Kaplan, principal investigator for the test, described the results as encouraging. At the present time, Clementia is continuing with an extension study of PV. In the extension study, which is for subjects of the original Phase 2 test, subjects are guaranteed to get PV and do not get a placebo. Clementia is increasing the dose and extending the number of days of dosing. The extension study also includes a new regime for adult patients and some newly enrolled adult subjects. As I understand it, these patients start during a flare-up free phase, and they take a low daily dose of PV. The daily dose continues until such time as a flare-up may begin, and at that point, the dose is ramped up higher for a period of time.

Did these test results show a slam dunk, home run, Stanley Cup victory (to cram in as many sports analogies as I can, ha ha – this is for my 14 year old son Owen ;-) )? No. But frankly, it would have been astonishing if the first ever Phase 2 drug test for FOP had produced a perfect result. What this test did reveal was that PV appears to have promise in treating FOP, and that with further tinkering as to dosing and duration of treatment, PV could very possibly turn out to be a viable treatment for FOP. Clementia is continuing it’s work with PV and planning to conduct further studies.

If you are interested in reading the full press release, click on this link:

This is happy, happy news for the FOP community! My biggest and most heartfelt thanks for all the people with FOP who took part, and continue to do so, in testing of PV. It wasn’t easy – it involved multiple trips to study sites in various cities, x-rays, blood tests, CT-scans, toleration of side effects (dry skin, so I’m told), and more. These folks are real soldiers. Profound thanks also to the Pennsylvania research group and its associates, who made the initial scientific discoveries which lead to use of PV, to the scientists administering the testing, and of course to Clementia itself.

Onward and upward! I’m looking forward to more PV test results, and to tests of other potential FOP medications by other pharmaceutical companies (Regneron has a Phase 1 test underway, and other companies have drugs on the horizon for possible testing). The future is looking bright.

Hoping to bring this to an end…

Skeleton of Harry Eastlack, a man who had FOP.

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