A recent study, conducted by the collaborative Trajectory Analysis Project (cTAP) and its international partners, has established some key criteria that could help drug developers identify groups of patients who may be able to participate in clinical trials.
A key objective in research to treat Duchenne muscular dystrophy (DMD) would be a therapy that helps not only patients early in their disease but also older patients with more advanced disease. Developing such a therapy is challenging, as is designing a clinical trial to test its efficacy in later-stage patients. Historically, drug developers have limited testing population criteria to a binary parameter: can a patient still walk? Recently, the collaborative Trajectory Analysis Project (cTAP) and international collaborators announced a study that offers a rigorous data case for using a more sophisticated set of criteria to select a testing population, broadening the population eligible for trials and effective therapies.
cTAP is a multinational, multistakeholder nonprofit collaboration dedicated to advancing clinical trial design, analysis, and data resources in rare neuromuscular diseases. Leveraging expansive natural history and placebo datasets, a highly engaged network of world-class experts, and a proven track record of success in collaborative problem-solving, cTAP develops evidence-based advancements with real-world impact. With an initial focus on Duchenne muscular dystrophy, cTAP’s rigorous, data-driven solutions hold promise for numerous rare, heterogeneous diseases and patient communities worldwide.
You can read the full press release here.
