cTAP to Present Late-breaking Results Supporting Advancements in Clinical Trial Design for Duchenne Muscular Dystrophy at the World Muscle Society Congress

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- 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 23^rd 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.

Titled “Can natural history (NH) controls be used for functional outcomes in Duchenne muscular dystrophy (DMD) drug trials? Assessing the consistency of 48-week changes in six-minute walk distance (6MWD) between multiple real world data sources and clinical trial placebo arms,” the study was undertaken to address regulatory concerns that the risk of inconsistency between real world data and data collected in a clinical trial limits the extent to which drug developers may use real world data in DMD to supplement, or even substitute for, placebo-controlled trials.

Led by Professor Nathalie Goemans, University Hospitals Leuven, Child Neurology, Leuven, Belgium, the study also represents a notable example of successful collaboration across nearly 40 institutions: the Child Neurology Centre at the University Hospitals Leuven, Belgium, the 13-center Italian DMD Group, the U.S.-based 3-center imaging DMD registry, the 20-center Cooperative International Neuromuscular Research Group (CINRG) database, and the Biomarin-sponsored PRO-DMD-001 prospective observational study of disease progression (data provided by CureDuchenne).

Senior author Professor Mercuri, Department of Pediatric Neurology, Catholic University, Rome, Italy, said, “this study is an important milestone for drug development in Duchenne; it would not have been possible without cTAP.”

The poster will be available for viewing throughout the conference in the area designated for late breaking submissions.

Additional collaborative studies enabled by cTAP will be presented in poster sessions on Friday, October 5, including:

• P-300. Prognostic factors for changes in 4-stair climb ability in patients with Duchenne muscular dystrophy (DMD). Nathalie Goemans, Brenda Wong, James Signorovitch, Gautam Sajeev, Madeline Jenkins, Ibrahima Dieye, Zhiwen Yao, Intekhab Hossain, Susan J. Ward, and cTAP. Professors Nathalie Goemans and Eugenio Mercuri will present.
• P-299. Minimal detectable change in the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD). Francesco Muntoni, Adnan Manzur, Anna Mayhew, UK North Star Study Group, James Signorovitch, Gautam Sajeev, Zhiwen Yao3, Ibrahima Dieye, Madeline Jenkins, Susan J. Ward, and cTAP. Professor Francesco Muntoni will present.

About cTAP

The collaborative Trajectory Analysis Project (cTAP) is an international, multi-stakeholder collaboration that identifies novel opportunities to use patient data and analyses to generate insights and tools for more effective clinical trial design in Duchenne muscular dystrophy. Its dynamic alliance of academic, industry, and patient stakeholders work collaboratively to identify priority research questions and engage cTAP’s unique set of resources to translate questions into insights with near-term, real-world application. Core to the success of this project are cTAP’s partnership with the outcomes research experts at Analysis Group, Inc., as well as the large and continually growing database of real world and clinical trial data sources that have been shared with cTAP. While cTAP’s initiating focus is Duchenne muscular dystrophy, its approach offers potential application to any challenging disease state characterized by heterogeneous progression. http://ctap-duchenne.org/

About Analysis Group

Founded in 1981, Analysis Group is one of the largest economics consulting firms, with more than 900 professionals across 14 offices. Analysis Group’s health care experts apply analytical expertise to health economics and outcomes research, clinical research, market access and commercial strategy, and health care policy engagements, as well as drug-safety related engagements in epidemiology. Analysis Group’s internal experts, together with its network of affiliated experts from academia, industry, and government, provide our clients with exceptional breadth and depth of expertise and end-to-end consulting services globally. http://www.analysisgroup.com/

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a uniformly fatal, progressive muscle-wasting disease affecting about one in 3,500-6,000 male live births. Patients with Duchenne lack the ability to make dystrophin, a protein crucial to muscle function. As their muscles deteriorate, they progressively lose the ability to walk, feed themselves, turn over in bed, and ultimately to breathe. While there is no cure, the past decade has seen an explosion in research resulting in more than 15 new therapies entering clinical development, with some receiving conditional approval. Learn more about Duchenne at http://cureduchenne.org, http://parentprojectmd.org and http://charleysfund.org.

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