Beyond Traditional Placebo Controls in Rare Disease Clinical Trials: New cTAP Evidence Supports Patient-Centric Alternatives

CAMBRIDGE, Mass., February 7, 2023 - The Collaborative Trajectory Analysis Project (cTAP), a global coalition in rare disease, has announced evidence to support patient-centric alternatives to the conventional use of genetically matched placebo controls in clinical trials for RNA-editing therapies in Duchenne Muscular Dystrophy (DMD). Published in Neurology, the large, multi-institution, multi-national study of more than 700 patients with DMD quantified the impact of genotype classes on observed disease. Results showed that less than 2...

cTAP Study Shows Real-World, Natural History Data Comparable to Placebo Data from Clinical Trials in Duchenne Muscular Dystrophy

Results published in peer-reviewed journal Neurology indicate that real-world data (RWD) and natural history data (NHD) could augment or replace placebo controls in DMD clinical trials. Use of RHD and NHD have potential to streamline and accelerate clinical programs targeting new treatments for DMD. CAMBRIDGE, Mass., September 8, 2020 /PRNewswire/ – The Collaborative Trajectory Analysis Project (cTAP), a multi-stakeholder, global research coalition in Duchenne muscular dystrophy (DMD), today announced results from the largest ever multi-national,…

cTAP Publishes Comprehensive Analysis of NSAA

The collaborative Trajectory Analysis Project (cTAP), a multi-stakeholder, global coalition in Duchenne muscular dystrophy (DMD), has published research in the journal PLOS ONE providing quantitative insights to assist the design and analysis of clinical trials in patients with DMD. READ FULL PRESS RELEASE ON BUSINESSWIRE: cTAP Publishes Comprehensive Analysis of the North Star Ambulatory Assessment (NSAA), a Newly Adopted Primary Outcome Measure in Clinical Trials of Patients with Duchenne Muscular Dystrophy

cTAP Partner Wins ISPOR Award on Duchenne Muscular Dystrophy

Published December 5, 2018 The collaborative Trajectory Analysis Project (cTAP), a multi-stakeholder, pre-competitive global coalition in Duchenne muscular dystrophy, today announced that Analysis Group Managing Principal James Signorovitch, cTAP’s partner, has been selected as recipient of ISPOR’s “Best General Research Podium Presentation” award at ISPOR Europe 2018, which recently took place in Barcelona, Spain. Dr. Signorovitch’s research presentation titled Ambulatory Function in Duchenne Muscular Dystrophy (DMD): The Characteristic Trajectory and Variation Across Individuals, will be...

cTAP to Present Late-breaking Results at the World Muscle Society Congress

Published October 1, 2018 The collaborative Trajectory Analysis Project (cTAP), a multi-stakeholder, pre-competitive global coalition in Duchenne muscular dystrophy, today announced the acceptance of a late-breaking abstract for presentation at the 23rd International Congress of the World Muscle Society. This is in addition to the two previously accepted submissions that cTAP collaborators will present at the Congress, which is being held October 2-6, 2018 at the Intercontinental Hotel in Mendoza, Argentina. READ FULL ARTICLE: cTAP...

cTAP to Present at Annual Biopharmaceutical Conference

Published June 25, 2018 The collaborative Trajectory Analysis Program (cTAP), a multi-stakeholder, pre-competitive global coalition in Duchenne muscular dystrophy today announced that Susan J. Ward, Ph.D., Executive Director of cTAP, will present at the 2018 ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop Annual Conference in Washington DC on Thursday, September 13, 2018 at 1:15 p.m. EST. Debra Miller, CEO of CureDuchenne, a patient foundation in Duchenne that provided seed funding for cTAP and is a founding...

Summit Therapeutics Joins cTAP to Support the Development of Potential DMD Treatments

Summit Therapeutics Joins cTAP to Support the Development of Potential DMD Treatments Published September 29, 2017 The newly founded Collaborative Trajectory Analysis Project (cTAP) coalition of stakeholders has invited Summit Therapeutics, a company specialized in the discovery and development of drugs targeting Duchenne muscular dystrophy (DMD), to support the development of new therapeutic products aiming to treat DMD, such as utrophin modulators. cTAP was created to overcome the challenges of developing drugs for diseases characterized…