ALS PATIENTs DEMAND: The ALS Patient-Driven Electronic-based Multidrug Adaptive Network Design Clinical Trial

Amyotrophic lateral sclerosis (ALS) is a rare neurodegenerative disease which is uniformly fatal. The only FDA approved therapy for ALS, riluzole, increases survival by a few months. In patient focus groups there is overwhelming interest in using a drug 'cocktail' approach to ALS therapy, with drugs targeting different pathological pathways. We have designed a patient-driven clinical trial to assess which of 3 drug cocktails are the most effective in slowing disease progression in Amyotrophic Lateral Sclerosis. From the point of view of the researcher planning a clinical trial we are typically exclusionary, narrowing inclusion criteria to try and reduce variability between individuals. From the point of view of the patient with ALS, they often feel left out, or abandoned by the research community once the disease progresses beyond the earliest stages. By utilizing existing major initiatives to connect electronic medical records (EMRs) of large academic ALS centers, simplify the regulatory process, and rolling out common data elements both through the EMR and via CTSA funded REDCap databases, we can build a large national ALS clinical trial network. This gives us the bandwidth to consider a study with large numbers of ALS patients, and more importantly, broaden the inclusion criteria to include ALS patients often excluded from clinical trials. The innovations in infrastructure, coordinated from 2 CTSA Regional Coordinating Centers and comprising a network of 20 sites (17 CTSA sites, and 3 other centers), will serve as proof of principle that existing CTSA infrastructure can be leveraged to run meaningful therapeutic research. We are using tools developed through both the CTSA network and the Patient Centered Outcomes Research Network (PCORnet, Greater Plains Collaborative in the Midwest and Texas and pSCANNER in California).

Other Information:

• Principal Investigator (also GPC Principal Investigator): Richard Barohn, MD; University of Kansas Medical Center
• Participating GPC Sites:
  • University of Kansas Medical Center
• Status: Not funded

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