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Clinical Trials 101

Learn more about what it means to be in a clinical trial.

What is a Clinical Trial, and Why are They Necessary for ALD/AMN?

If you or a loved one has ALD/AMN, you know the heartbreaking frustration that there are not many treatments that can stop or significantly slow the all forms of the disease. You may not know that there are hundreds of scientists and doctors working hard to discover those treatments. That discovery depends, in large part, on the willing spirit of people with ALD/AMN who volunteer to be a part of a clinical trial.

Clinical Trials 101: Placebo-Controlled Trials

Most ALD/AMN patients and caregivers want to do everything they can to help find new treatments for the disease. The idea of trying an experimental therapy is often regarded positively, despite knowing that risks may be present.  However, some people have misgivings when they learn that some participants in a trial may receive a placebo rather than the drug being tested.

Clinical Trial and Other Important Definitions

Familarizing yourself with terms related to clinical trials can improve your understanding of the clinical trial process and can better inform your decision to enroll in a clinical trial. A collection of terms from this website is provided here.

Drawn to Science: Clinical Trials Video

Produced by Roche.

Getting Involved in a Clinical Trial

People with ALD/AMN who enroll in a clinical trial are contributing to improved health care for everyone with the disease. Even when the results of a trial are negative, we learn that much more about the disease, and how to look for more promising new treatments.

What should people consider before participating in a trial?

People should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it, the care expected while in a trial, and the cost of the trial. The following questions might be helpful for the participant to discuss with the health care team. Some of the answers to these questions are found in the informed consent document.

  • What is the purpose of the study?
  • Who is going to be in the study?
  • Why do researchers believe the new treatment being tested may be effective? Has it been tested before?
  • What kinds of tests and 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 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 treatment is working? Will results of the trials be provided to me?
  • Who will be in charge of my care?

We recommend visiting the Center for Information and Study on Clinical Research Participation (CISCRP) webpage for additional educational material on clinical trials.  CISCRP is an independent non-profit organization dedicated to educating and informing the public and patients about clinical research. CISCRP also provides information and resources to help research and health professionals better serve their patients and study volunteers.  CISCRP has excellent educational videos on their website which illustrate some best practices for clinical trial participants.  Click here to see the videos.

Use to Identify Clinical Trials for ALD/AMN is a central database of clinical trials for medical disorders of all kinds, including ALD/AMN. It is an important resource for finding a clinical trial that is right for you. The site is maintained by the National Library of Medicine, a branch of the US government.
Click here to see all of the ALD trials.
Click here to see all of the AMN trials. 
Click here to search the databse using your own search terms. 

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