Clinical trials

Clinical trials are an essential part of developing new treatments and test whether a specific treatment is effective and safe. Trials start with a small number of volunteer test subjects, and can grow to include larger number of participants.

There are different kinds of clinical trials. The gold standard is the controlled, randomized, and double-blind studies. See this educational video which explains some of the most important expressions from clinical trials.

While clinical studies of treatments for OI and other rare diseases have some differences, their basic structure is the same. All enroll volunteers at each phase, following protocols set out at the beginning of the study.

The phases of a clinical trial

The phases of a clinical trial include:

  • Preclinical or Non-clinical testing: scientists give the treatment to animals (like mice or zebrafish) to see if it produces a response and is safe
  • Phase 1: a small group of human participants are examined; looking for safety, proper dosage amounts and to confirm it has some response in humans. Phase 1 may be done in healthy volunteers or in patients with the disease, or at-risk of the disease, under study.
  • Phase 2: further tests safety and if it stimulates response in different types of people
  • Phase 3: determine if treatment is effective. These studies should be large enough to find relatively rare side effects that might be missed in earlier studies.
  • Phase 4: sometimes used after a treatment has been approved, this phase is used to find any long term effects of the treatment

Individuals with OI play an especially important role in the outcome of clinical trials because they have a rare condition. Compared to more common conditions, rare disease based clinical trials usually have far fewer participants, making every volunteer even more valuable to the research process. Progress really depends on us being willing to participate.

A clinical trial can be sponsored (financed) by both noncommercial stakeholders (like hospitals, universities, non profits etc) and commercial companies (industry).

OIFE provides advice from the patient perspective to both commercial and non commercial stakeholders who are planning clinical trials in OI.

Where can I find ongoing trials?

The best way to find out about studies in Europe, is to subscribe to the OIFE Magazine, where we include announcement from trials that are recruiting. To find out about more information on specific clinical studies, you can also go to and search for “osteogenesis imperfecta” in the “Condition or Disease” field.

The best way to hear more about potential clinical studies, if you live in the USA, is by enrolling at the OI Registry. People from outside the USA can also join the OI Registry, but most of the studies announced are recruiting in North America. To find out about more information on specific clinical studies, you can also go to

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