Clinical Trials Factsheet

Understanding the Medical Research Process

There are different types of medical research:

• A case study is a report of a single case: of how one doctor treated one patient.
• An observational study is a report of the investigator’s observations about how a group of patients responded to a course of treatment. This kind of study can also be done to follow the natural history of a disorder like OI.
• Clinical trials compare the ways different groups of patients respond to different courses of treatment. A controlled clinical trial compares patients receiving a treatment with patients receiving no treatment (controls). It assesses how much of an effect the treatment has.

For a detailed discussion of the medical research process see the OI Foundation fact sheet Medical Research Clinical Trials.

Locating Studies

• The web site http://www.clinicaltrials.gov/ is a searchable web site with extensive information about clinical trials in general as well as links to the trials themselves.
• The OI Foundation periodically provides information about studies that are looking for participants who have OI. The Foundation’s Medical Advisory Council reviews all studies before they are advertised.
• The OI Registry announces studies that have been approved by its board to all eligible members and provides information about how to contact the investigator.

Points to Consider

• OI is a rare condition and there is still a lot to learn about its causes, its symptoms across a lifetime, and best treatments.
• Many people who participate in clinical trials experience a considerable sense of satisfaction in knowing that they are helping advance medical understanding and treatments for themselves and others.
• If you are thinking about participating in a clinical study it is important to get answers to all of your questions. Here are some sample questions.
  • What is the purpose of the study?
  • Who is going to be in the study?
  • Why do researchers believe the experimental treatment being tested may be effective? Has it been tested before?
  • What kinds of tests and experimental treatments are involved?
  • How do the possible risks, side effects, and benefits in the study compare with my current treatment? How might this trial affect my daily life?
  • How long will the trial last?
  • Will hospitalization be required?
  • Who will pay for the experimental treatment?
  • Will I be reimbursed for other expenses?
  • What type of long-term follow-up care is part of this study?
  • How will I know that the experimental treatment is working?
  • Will results of the trials be provided to me?
  • Who will be in charge of my care?