Clinical Studies & Trials

Clinical Studies & Trials

About Clinical Trials

Clinical trials are an important part of drug development and must take place before a medicine can be approved for use as a drug prescribed by doctors. Read below to learn more about the phases of clinical trials and how new therapies become available to people who need them. To see definitions for terms used, visit the Clinical Trial Glossary page.

Clinical trials are voluntary studies carried out in people to answer specific research questions. Clinical trials:

  • Study the safety and effectiveness of new drugs or procedures
  • Assess new ways of using existing treatments
  • Can be designed to learn more about a disease (e.g. a
    natural history study

    Natural History Studies: Type of observational study designed to look at the course of a disease without any type of treatment or intervention (other than standard of care). Natural history studies help researchers better understand the course of disease over time and what demographic, genetic or environmental factors are associated with the disease progression.


It’s important to know that:

  1. Clinical trials are not treatments; they are research studies. Participating in a clinical trial doesn’t mean that a drug will make your symptoms better or will stop the spread of a disease. You may even get a

    Placebo (control): An inactive product that resembles the test medicine or procedure, but without a treatment value. A placebo can be a pill, a powder, or a solution that resembles the test drug, but has no physical effect.

    in a clinical trial which contains no active ingredients at all.
  2. Investigational drugs may have unwanted side effects that can range from mild to very serious or life-threatening. However, by participating in a clinical trial, you are helping researchers evaluate the safety and effectiveness of new drugs for FOP and answer important scientific questions.
  3. An important part of many clinical trials is a placebo, which contains no active ingredients and acts as a control to compare any possible effect with the research drug. Participants who receive a placebo will still often receive the research drug after the placebo part of the trial is over.

The decision to participate in a clinical trial is yours. Participation is always voluntary, and you have the right to withdraw from a clinical trial at any time.

The FOP community is fortunate that there are multiple clinical trials happening for this ultra-rare disease. You may fit the eligibility criteria for more than one trial. This guide, created by PRA Health Sciences (now ICON), can be helpful to you in your decision-making process.

Learn more about Clinical Trial Phases.

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