IFOPA's Support of Clinical Trials

A Message about the IFOPA's Support for Clinical Trials

From time to time, the IFOPA is asked whether it knows about and supports a drug trial for FOP. What does it mean for the IFOPA to know enough about a drug trial to be able to support the trial? What does IFOPA "support" mean?

Understanding a drug trial means that we know something about the compound, or drug, being tested. For example, how is the drug believed to work - by what mechanism is it believed to stop FOP? How much research has been conducted on it before it was tested in humans? Has it been tested in a laboratory? If so, in what types of experiments? Has it been given to animals? If so, what type of animals, and what effects were seen? Was it given to healthy individuals before those with FOP? If so, what effects, both intended and not intended, were seen? Has it been shown to be safe for adults? What about children?

Understanding a drug trial also means that we know various details about how the trial will be conducted, or what is called the "research protocol." The protocol includes criteria for who can participate, dosing regimens, how the drug is administered, when the drug is administered, whether a placebo (a substance having no effect) is being used to compare with the drug, how the effectiveness of the treatment will be measured, what kinds of blood tests or imaging techniques (such as X-Rays, CT scans, and MRIs) are being employed, the number of patients to be enrolled in the study, the clinical sites involved in the study, the principle investigator, and so on. Those are the elements that are found in a drug trial that is approved by governmental agencies in the United States, Europe and many other parts of the world.

Lastly, understanding a trial also means knowing that it has the approval of both an ethics committee and the relevant governmental agency that oversees medical research in that region. An ethics committee is an independent group whose responsibility is to protect the rights, safety and well being of human participants in a clinical trial. Government agencies ensure that drugs work correctly and that their health benefits outweigh their known risks. (To read more about clinical trials see clinicaltrials.gov.)

In summary, then, the IFOPA supports clinical trials only when we understand the drug being tested, the research protocol, and the regulatory and ethics approvals for the trial. At this point in time the IFOPA knows of two trials of experimental medicines for FOP that meet applicable standards, namely, the PVO-1A-202 palovarotene Phase 2 trial sponsored by Clementia Pharmaceuticals in subjects with FOP and the recently announced REGN2477 Phase 1 trial in healthy volunteers sponsored by Regeneron Pharmaceuticals. If and when we become aware of further drug trials which deserve the IFOPA’s support, we will update this statement.

What Does IFOPA "Support" Mean?
Our "support" of a clinical trial means simply that we share information on the trial with the FOP community, especially how and where to enroll. The decision to enroll in a drug trial is, of course, up to the patients and their families.

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Michelle Davis
Executive Director
International FOP Association

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