The descriptions at clinicaltrials.gov
use medical language that can't always be understood by us laypeople, but with a bit of practice you can learn to get enough information to know if a particular trial is of interest.
The first fact to notice is what "Phase" the trial is. Here's a good summary at Wikipedia
Phase 1 trials: Researchers test an experimental drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2 trials: The experimental treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
Phase 3 trials: The treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow it to be used safely.
Phase 4 trials: Postmarketing studies delineate additional information, including the treatment's risks, benefits, and optimal use.
There are slightly different definitions in the glossary at clinicaltrials.gov
. They make the claim that Phase 1 trials are conducted with health volunteers, which I don't find to be the case for most trials related to bone marrow failure disease. They also mention that Phase 2 trials often involve placebos.
A Phase 1 trial may be the first time a drug is given to humans. Or it may instead be the first test of a drug' use for a given condition when its use is already well-established for other illnesses. The first test of a brand new drug may not be as likely to benefit the patients involved as a Phase 3 or Phase 4 trial of a drug that's already been through earlier trials to establish an effective dosage and compare its outcomes with other treatments. But a Phase 1 trial can be the first chance to get a drug that appears promising.
Phase 2 trials often answer the question "Does this drug or treatment protocol work?". Phase 3 trials often answer the question "Is this drug or treatment protocol better than other choices?" by comparing different treatments against each other head to head.
What else to look for
You can check who is running the trial, where it's taking place, and how long it will run. Ideally a trial would be at your own treatment center. If not, you'll want to know where you'd have to be, how often and for how long. Some patients are more able or willing than others to travel for treatment in a trial. Many trials are held at the NIH
in Bethesda Maryland.
You should also read the Eligibility/Recruitment information to see if you qualify for the trial. For example, a study may be limited to patients who are newly diagnosed and haven't had other treatments, while other studies might be designed for patients who have failed to improve after other treatments. Even if you want to get into a trial, they may not want you if you don't match their research agenda.
Finally, to get a complete understanding of a trial and get past the medical terminology, you can talk to your own doctor and/or contact the person listed as the "Contact" (or the office of the Principal Investigator) to learn about what the trial involves and whether it might suit you, armed with your list of questions.
The bottom line questions to ask yourself are: What are the possible benefits? What are the risks? How do they compare with what I'll do if I'm not in the trial?
Read How to Read a Study Record
for tips on reading clinical trial descriptions.
You can also get help understanding clinical trials from the AA&MDSIF