The Cure SMA Clinical Trial Readiness Program is designed to equip clinical trial sites with resources that support their ability to manage spinal muscular atrophy (SMA) clinical trials in an effective and patient-centered way. The program includes voluntary educational evaluations to assess a site’s preparedness for SMA trials, as well as print resources and periodic events to help sites and teams learn about SMA and tips for conducting clinical trials in SMA.

Thank you for completing the survey! You may now download the Best Practices Toolkits using the links below each description.

Cure SMA has created a series of three complementary toolkits to help research teams and clinical sites prepare for and conduct SMA clinical trials.

Toolkit 1: The Basics of Spinal Muscular Atrophy and the Effective Conduct of Clinical Trials in SMA

In recent years, major advancements have taken place in our understanding and ability to treat SMA. Shifts include a better understanding of natural history, new approved and investigational drugs, and more widespread adoption of newborn screening. This toolkit focuses on SMA as a disease and the landscape for disease management and treatment options, providing research teams with foundational knowledge and vocabulary to aid them in their work. It also discusses steps involved in trial preparation and conduct, issues that may arise in SMA trials, and things to consider in your day-to-day work. Finally, the toolkit offers an extensive list of resources prepared by other organizations and individuals to assist with protocol adherence (e.g., tools and templates for tracking study related procedures; lists of essential documents for before, during, and after a visit; a glossary of commonly used terms; and much more!).

» Download Toolkit 1: Clinical Trial Readiness

» Provide Feedback on the Trial Readiness Toolkit

Toolkit 2: Best Practices for Clinical Research Coordinators in SMA

Clinical research coordinators (CRCs, also known as Study Coordinators) play a pivotal role in successful execution of an SMA clinical trial, in that their interactions with research participants and their families can have an outsized impact on these individuals’ experiences throughout the trial process, and determine how effective sites are at recruitment, enrollment, and retention. This toolkit, tailored specifically to the role of the CRC, includes a brief overview of the responsibilities of a CRC when coordinating any clinical trial, as well as information on steps to take when preparing for an SMA trial. The majority of the toolkit focuses on key stages and aspects of SMA clinical trials, with discussion of strategies and tactics to support trial management, and specific challenges that CRCs may encounter in the context of SMA clinical trials.

» Download Toolkit 2: Clinical Research Coordinators

» Provide Feedback on the Clinical Research Coordinators Toolkit

Toolkit 3: Best Practices for Physical Therapists and Clinical Evaluators in SMA

Physical therapists/clinical evaluators (PTs/CEs) play a fundamental role in the assessment of individuals with SMA, which may involve impairment-based measures, as well as capacity and/or performance-based measures. To support PTs/CEs involved in SMA trials, Cure SMA, in collaboration with two PTs with significant experience in SMA and SMA clinical trials, has developed recommendations to promote the most effective conduct in clinical trials. These best practices are intended to help CEs – especially those new to SMA clinical trials – understand challenges they may encounter and find productive ways to navigate these challenges. Also included in this document is a set of appendices with links to additional resources, including seminal papers in SMA, manuals, and articles that delve more deeply into SMA issues.

» Download Toolkit 3: Physical Therapists and Clinical Evaluators

» Provide Feedback on the Physical Therapists and Clinical Evaluators Toolkit

Additional Resources

Cure SMA periodically organizes events such as workshops and webinars for members of research teams who work – or would like to work – on SMA clinical trials. To receive information about future events, please email [email protected].