MDA Launches Clinical Neuromuscular Disease Registry

To answer these questions — and more — the MDA clinical neuromuscular registry will document the care provided to thousands of people with neuromuscular diseases across the country.

Aiding clinical trials

A neuromuscular registry also has the potential to expedite clinical trials and to make trials more efficient.

For the first time, a national registry will be able to inform the researchers and biopharmaceutical companies interested in neuromuscular research by validating the number of individuals who have a specific genetic mutation.

A registry also makes it possible to quickly disseminate information about clinical trials to potential participants. Easing the burden of clinical trial recruitment makes it easier for companies and researchers to invest in discovering treatments for these rare conditions.

The registry also can help researchers determine if a treatment is having a positive or negative effect. When trial participants have had similar health care and a similar disease course, researchers can more confidently attribute any effects to the drug being tested.

What will a registry do?

MDA clinicians will collect clinical and health-outcome data from individuals with neuromuscular disease, such as information about the types of medical care they’re receiving and their disease progression. (Participation in the registry is completely voluntary for individuals seen in MDA clinics.)

The MDA registry seeks to:

• gain a better understanding of the course of illness for specific neuromuscular diseases;
• collect data about genotype-phenotype correlations (the relationship between genetic mutations and symptoms/disease course) to allow for better prediction of disease progression based on genetic information;
• identify long-term outcomes that highlight best clinical practices;
• provide data that can be used to develop a quality-improvement program for MDA clinics; and
• expedite research by establishing a database of individuals eligible for clinical trials in neuromuscular disorders.

MDA’s registry plans

MDA’s clinical advisory committee, clinical research networks, registry advisory board, and family members and/or people affected by neuromuscular disease have been instrumental in the development of the registry.

The registry will be rolled out in stages, starting with collecting information from individuals with amyotrophic lateral sclerosis (ALS), DMD and spinal muscular atrophy (SMA).

These diseases were chosen to start because there currently are a number of experimental therapies in development that address them, and because government funded working groups have identified and standardized the information that is important to collect in clinical trials for these three diseases. In addition, there are formal “standards of care” for individuals with these diseases, and the registry can help determine to what extent these standards are being implemented and how they affect outcomes.

Expect the registry to officially launch up to 25 MDA clinics by the end of 2012. It will be expanded each year to include additional diseases and MDA clinics.

When your MDA clinic and your disease become a part of the MDA registry, your clinic team will explain the process to you and gain your consent to enter your protected health information into the Web-based registry. No registry information will be collected without your consent.

As development of the registry progresses, MDA will host webinars and other educational initiatives, so stay tuned — there’s much more to come about this exciting new MDA program!

Jodi Wolff is MDA’s director of clinical services and summer camp.