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

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."

Sep 11, 2020
Santhera Announces Full Enrollment Of ReveraGen’s Pivotal VISION-DMD Study With Vamorolone In Duchenne Muscular Dystrophy
Clinical Trials and Research

Santhera Announces Full Enrollment of ReveraGen’s Pivotal VISION-DMD Study With Vamorolone in Duchenne Muscular Dystrophy

Santhera Pharmaceuticals announces that partner ReveraGen Biopharma Inc. has completed enrollment into the pivotal VISION-DMD study with vamorolone in patients with Duchenne muscular dystrophy (DMD). Subject to a positive study outcome, this could allow for a regulatory submission to the US FDA in the fourth quarter of 2021 with the potential to offer an alternative to current standard of care in young boys with DMD.

The 48-week Phase 2b VISION-DMD study (VBP15-004; clinicaltrials.gov: NCT03439670) is designed as a pivotal trial to demonstrate efficacy and safety of vamorolone administered orally at doses of 2.0 mg/kg/day and 6.0 mg/kg/day versus prednisone 0.75 mg/kg/day and placebo in ambulant boys ages 4 to <7 years with DMD. Efficacy outcome measures after the first 24-week treatment period are muscle strength and motor function outcomes with Time to Stand test (TTSTAND) as the primary study endpoint.

 

Sep 11, 2020

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