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Wed Jun 3, 2015, 05:10 PM
Clinical trials are research studies that use volunteer patients to study ways to understand, prevent, diagnose, and treat diseases.
Why participate?
Patients may consider participating in a trial for a few reasons:
To have access to new treatments not yet available to the public.
Because of the expert care provided by treatment centers running clinical trials.
To contribute to medical research that can help all future patents. Most of the drugs you read about in these forums were studied in clinical trials before they became widely available.
There are downsides to clinical trials as well:
Patients in most clinical trials receive treatments about which less is known than standard treatments, so outcomes are less predictable.
The treatment may be unsuccessful or have adverse effects.
You may need to undergo more tests than with standard treatment, because researchers want as much data as they can get. Your successful treatment is the primary goal, but not the only goal.
You may need to travel to a treatment center that's running the trial.
Learning about clinical trials
The primary source of information about clinical trials in the United States is the ClinicalTrials.gov (https://clinicaltrials.gov) website.
The following are excellent summaries from the Aplastic Anemia & MDS International Foundation (http://www.aamds.org/) (AA&MDSIF).
Clinical Trial Basics (http://www.aamds.org/node/86)
More about Clinical Trials (http://www.aamds.org/about/pnh/clinical-trial-more)
Finding the right clinical trial
Each clinical trial has specific eligibility requirements. Researchers may be looking for patients with specific diseases, certain treatment histories, and a certain current status. It takes effort to identify clinical trials that might be suitable for a given patient.
There are a few ways to find clinical trials for a bone marrow failure disease:
Ask your doctor.
Talk to a Patient Educator (http://www.aamds.org/node/123) at the AA&MDSIF, or email questions to clinicaltrials@aamds.org.
Watch the Clinical Trial Announcements (http://www.mds-foundation.org/clinical-trial-announcements/) page at the Myelodysplastic Syndromes Foundation (http://www.mds-foundation.org/) website.
Search Clinicaltrials.Gov (https://clinicaltrials.gov).
Use the following links to search Clinicaltrials.Gov by disease (source: AA&MDSIF (http://www.aamds.org/about/pnh/clinical-trial-finding)):
Aplastic anemia clinical trials: U.S. (http://www.clinicaltrials.gov/ct2/results?term=aplastic+anemia&recr=Open&cntry1=NA%3AUS) or International (http://www.clinicaltrials.gov/ct2/results?term=aplastic+anemia&recr=Open)
MDS clinical trials: U.S. (http://www.clinicaltrials.gov/ct2/results?term=myelodysplastic+syndromes&recr=Open&cntry1=NA%3AUS) or International (http://www.clinicaltrials.gov/ct2/results?term=myelodysplastic+syndromes&recr=Open)
PNH clinical trials: U.S. (http://www.clinicaltrials.gov/ct2/results?term=paroxysmal+nocturnal+hemoglobinuria&recr=Open&cntry1=NA%3AUS) or International (http://www.clinicaltrials.gov/ct2/results?term=paroxysmal+nocturnal+hemoglobinuria&recr=Open)
Why participate?
Patients may consider participating in a trial for a few reasons:
To have access to new treatments not yet available to the public.
Because of the expert care provided by treatment centers running clinical trials.
To contribute to medical research that can help all future patents. Most of the drugs you read about in these forums were studied in clinical trials before they became widely available.
There are downsides to clinical trials as well:
Patients in most clinical trials receive treatments about which less is known than standard treatments, so outcomes are less predictable.
The treatment may be unsuccessful or have adverse effects.
You may need to undergo more tests than with standard treatment, because researchers want as much data as they can get. Your successful treatment is the primary goal, but not the only goal.
You may need to travel to a treatment center that's running the trial.
Learning about clinical trials
The primary source of information about clinical trials in the United States is the ClinicalTrials.gov (https://clinicaltrials.gov) website.
The following are excellent summaries from the Aplastic Anemia & MDS International Foundation (http://www.aamds.org/) (AA&MDSIF).
Clinical Trial Basics (http://www.aamds.org/node/86)
More about Clinical Trials (http://www.aamds.org/about/pnh/clinical-trial-more)
Finding the right clinical trial
Each clinical trial has specific eligibility requirements. Researchers may be looking for patients with specific diseases, certain treatment histories, and a certain current status. It takes effort to identify clinical trials that might be suitable for a given patient.
There are a few ways to find clinical trials for a bone marrow failure disease:
Ask your doctor.
Talk to a Patient Educator (http://www.aamds.org/node/123) at the AA&MDSIF, or email questions to clinicaltrials@aamds.org.
Watch the Clinical Trial Announcements (http://www.mds-foundation.org/clinical-trial-announcements/) page at the Myelodysplastic Syndromes Foundation (http://www.mds-foundation.org/) website.
Search Clinicaltrials.Gov (https://clinicaltrials.gov).
Use the following links to search Clinicaltrials.Gov by disease (source: AA&MDSIF (http://www.aamds.org/about/pnh/clinical-trial-finding)):
Aplastic anemia clinical trials: U.S. (http://www.clinicaltrials.gov/ct2/results?term=aplastic+anemia&recr=Open&cntry1=NA%3AUS) or International (http://www.clinicaltrials.gov/ct2/results?term=aplastic+anemia&recr=Open)
MDS clinical trials: U.S. (http://www.clinicaltrials.gov/ct2/results?term=myelodysplastic+syndromes&recr=Open&cntry1=NA%3AUS) or International (http://www.clinicaltrials.gov/ct2/results?term=myelodysplastic+syndromes&recr=Open)
PNH clinical trials: U.S. (http://www.clinicaltrials.gov/ct2/results?term=paroxysmal+nocturnal+hemoglobinuria&recr=Open&cntry1=NA%3AUS) or International (http://www.clinicaltrials.gov/ct2/results?term=paroxysmal+nocturnal+hemoglobinuria&recr=Open)