The Implementation of Platform Trials for ALS Research: Melanie Quintana, PhD


The senior statistical scientist and director of consulting at Berry Consultants talked about innovative clinical trial design in ALS research at the 2023 MDA conference. [WATCH TIME: 5 minutes]

WATCH TIME: 5 minutes

“I would say [the greatest advancement we have so far], in terms of clinical trial design, is platform trials. I really feel that clinical trial innovation is going to help us find the next therapy and the next one after that, and really help us get to the point where potentially we're slowing completely stopping the progression of the disease.”

Innovative clinical trial design is critical for the advancement in effective therapeutic developments. In the field of amyotrophic lateral sclerosis (ALS), trials often observe the effects of modest treatments in a population of patients with heterogeneous disease progression. One way to enhance trial designs in this subspecialty is through statistical enrichment, which can reduce needed sample sizes and improve study's capabilities, as well as platform trials, which tests multiple drugs at the same time with statistical analyses.1

At the recent 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 19-22, in Dallas, Texas, Melanie Quintana, PhD, presented on innovative clinical trial design in ALS research as means to accelerate therapy development. The rest of the presentations in the session shared updates on research collaborations across the field, including new initiatives. Overall, the session explored translational science for ALS such as therapeutic strategies, biomarkers, gene therapy, and more.2

At the conference, Quintana, senior statistical scientist and director of consulting at Berry Consultants, sat down with NeurologyLive® in an interview to discuss more about her presentation, and specifically, the role industry plays in developing advanced therapies and improving efficiency of clinical trials. In addition, Quintana spoke on the important aspects of care for patients with ALS when participating in a clinical trial. She also mentioned the most recent advancements in this field and the challenges that still come up when conducting the research.

Click here for more coverage of MDA 2023.

1. Fournier CN. Considerations for Amyotrophic Lateral Sclerosis (ALS) Clinical Trial Design. Neurotherapeutics. 2022;19(4):1180-1192. Doi:10.1007/s13311-022-01271-2
2. Quintana M. Accelerating Drug Development Through an Innovative Clinical Trial Design – Through the Lens of a Statistical Scientist. Presented at: MDA Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX. Industry Updates in ALS session.
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