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..

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?