Clinical Trials

Clinical Trials

New medical treatments must be proven safe and effective before they can be offered to a large number of patients. New treatments are tested through clinical trials, a series of research studies using a limited number of patients. Any new type of treatment can be tested in a clinical trial.

If you or a loved one has ALD or AMN, you know the heartbreaking frustration that there are not yet treatments that can stop or significantly slow 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. 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.

Find Your Clinical Trial

CenterWatch was the first Internet site to publish detailed information about active clinical trials that could be accessed by patients and their advocates. Today, they have one of the largest online clinical trial databases actively seeking patients. Visit their website to learn more.

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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 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.

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?

*Information from ALD Connect

Whole Genome Sequencing in Leukodystrophy

childrens_hospital_logoThe purpose of this study is to investigate the diagnostic efficacy, clinical utility, and cost effectiveness of next-generation sequencing (NGS) for leukodystrophies by determining if NGS improves diagnostic effectiveness in the leukodystrophies relative to current diagnostic approaches and whether NGS results in changes to healthcare-related costs. Learn more…

bluebird Bio’s Gene Therapy Study for Childhood Cerebral adrenoleukodystrophy (ALD)

BlueBirdBio_logoAn international clinical research study, called the Starbeam Study, is now enrolling boys, aged 17 and younger, who have been diagnosed with Childhood Cerebral Adrenoleukodystrophy (CCALD). CCALD symptoms usually occur in early childhood and progress rapidly, if untreated, ultimately leading to death. The Starbeam Study will assess the effectiveness and safety of an investigational gene therapy approach, known as gene transfer. The study involves transferring a new copy of the ABCD-1 gene into the patient’s own blood stem cells. The goal of the Starbeam Study is to determine if the one-time investigational gene therapy treatment can stop the progression of CCALD and if it is safe and well-tolerated. TO LEARN MORE CLICK HERE.

MD1003 for adult patients suffering from adrenomyeloneuropathy (AMN)

ELA_logo_2014A new clinical trial completely funded by ELA for 800,000 Euros to evaluate the effects of a new molecule on patients suffering from adrenomyeloneuropathy (AMN). Dr Frederic Sedel’s research work to which ELA contributed led to the identification of the MD1003 molecule as a promising treatment for progressive multiple sclerosis. Certain preliminary results on adrenomyeloneuropathy (AMN) paved the way for a new clinical trial testing the effects of MD1003 on AMN which will soon be starting. TO LEARN MORE CLICK HERE.