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cTAP Study Shows Real-World, Natural History Data Comparable To Placebo Data From Clinical Trials In Duchenne Muscular Dystrophy


CAMBRIDGE, Mass., Sept. 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, multi-collaboration study in DMD indicating that both real world data (RWD) and natural history data (NHD) are highly comparable to data from patients treated with placebo in multiple recent clinical trials. Results demonstrate that use of RWD or NHD could supplement or possibly even replace use of a placebo arm in future DMD clinical research, potentially helping to streamline efforts to develop new treatments. 

“This study found a striking level of consistency in the six-minute walk distance assessment in DMD patients from six clinical trial placebo arms and patients from five different real world and natural history studies,” said Craig McDonald, a co-author of the study and Professor and Chair of the Department of Physical Medicine and Rehabilitation at the University of California Davis. “This rigorous study establishes a strong foundation for using natural history data as a substitute for placebo control in clinical trials and as a comparator to determine the effectiveness of prescribed drug treatments versus standard of care treatment.”

CTAP Study Shows Real-World, Natural History Data Comparable To Placebo Data From Clinical Trials In Duchenne Muscular Dystrophy