Cure SMA Publishes Article on SMA Clinical Trial Readiness Program in the U.S.



Cure SMA is proud to share with the SMA community that it has published a paper in the Orphanet Journal of Rare Diseases, titled ‘The SMA Clinical Trial Readiness Program: Creation and Evaluation of a Program to Enhance SMA Trial Readiness in the United States.’

The Cure SMA Clinical Trial Readiness Program is an initiative developed under the Cure SMA Industry Collaboration in an effort to better understand the capacity of SMA Centers in the U.S. to continue carrying out effective clinical trials in an ever-changing SMA landscape. A program pilot successfully launched in 2018 with nine volunteer research centers in the U.S.

Before creating this program, Cure SMA assessed 21 of the top SMA Centers in the U.S. to project future trial recruitment needs, learn about other clinical trial network and readiness programs, and gain insight from established SMA trial sites on their capacity and factors affecting readiness. This publication describes the process Cure SMA underwent to create this program, including our outreach to sites and evaluation process; approach to developing best-practices and supportive resources; and methods to receive input on the program from all site stakeholders (i.e., principal investigators, clinical research coordinators, and physical therapists/evaluators).

In a napshot, the Cure SMA Clinical Trial Readiness Program engages prospective trial sites to optimize SMA trial readiness nationally, and was created with input from trial sponsors, research hospitals, and the patient community, as represented by Cure SMA. We now have the opportunity to engage sites who see SMA patients, are familiar with SMA motor function outcome measures, have prior experience conducting clinical trials, and who wish to become involved in SMA research.

Since 2018, the program has expanded with additional sites (currently 15) and Cure SMA has offered new best practices toolkits, webinars, and workshops for Physical Therapists and Clinical Research Coordinators. We have also continued to create ways to support research staff during the COVID-19 pandemic, including a new, soon-to-be-announced scholarship program.

In late 2019, Cure SMA also launched a new webpage with program information and free digital copies of all our available toolkits. This has allowed us to evaluate uptake and value of these resources, which are estimated to have already reached more than 200 Primary Investigators, Physical Therapists/Clinical Evaluators, and Clinical Research Coordinators in the U.S. To date, feedback has been very positive.

If you are interested in learning more about the Cure SMA Clinical Trial Readiness Program, or becoming a ‘Readiness Site,” please contact [email protected].

Funding for this work was provided by grants from the Cure SMA Industry Collaboration (IC). The members of the SMA IC at the time the work was completed were AveXis, Inc, Biogen, Genentech/Roche Pharmaceuticals, Novartis, Pharmaceuticals, Astellas Pharmaceuticals, Cytokinetics Inc, Ionis Pharmaceuticals, Inc., and Scholar Rock.

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