C3 funds retrospective analysis of clinical outcome assessments

Coalition to Cure Calpain 3 (C3) is pleased to announce the funding of a new research award to Dr. Meredith James and Professor Jordi Díaz-Manera of the John Walter Muscular Dystrophy Research Centre (JWMDRC) at Newcastle University. The project, titled “Improving clinical trial readiness by investigating the suitability of clinical outcome assessments and documenting the natural progression of LGMD2A/R1 – a retrospective analysis,” is a collaboration between the JWMDRC and Dr. Linda Lowes and Dr. Lindsay Alfano of the Center for Gene Therapy at Nationwide Children’s Hospital.

What role do clinical outcome assessments plan in C3’s mission to drive a treatment or cure for LGMD2A/R1?

The past several years have brought exciting advances in the development of potential gene therapy and small molecule treatments for LGMD2A/R1. As clinical trials to test these interventions approach, a clear understanding of disease symptoms and progression is essential. It is important to prepare for clinical trials by developing robust clinical outcome assessments (COAs) that are sensitive to changes and represent aspects of the disease that are meaningful to patients. COAs can help researchers understand the trajectory of LGMD2A/R1 in the absence of treatments, and they can be used to determine if interventional treatments change that trajectory.

This project brings together world leaders in the field of COA development from two clinics that have cared for many individuals living with LGMD2A/R1. The investigators will examine the clinical records of approximately 100 patients who have already been evaluated at these centers. Analysis of these records will allow the investigators to identify COAs that will be suitable for use in clinical trials, as well as to document the rate of disease progression.

---

Dr. James says, “On behalf of Professor Díaz-Manera, Dr. Lowes, Dr. Alfano and myself, we are grateful to C3 for providing the funding to drive our understanding of what outcome measures will be most suitable for Calpainopathy. With the combined data from two expert centers for LGMD, we can understand how the standard physiotherapy assessments perform in the LGMD2A/R1 population, then can drive trial readiness forward with informed and data driven guidance for future clinical trial design.”

---

“In order for clinical trials to be successful, they need to include COAs that are relevant to LGMD2A/R1 symptoms as well as important to individuals living with this disease,” explains C3 Scientific Director Dr. Jennifer Levy. “The results of this study will inform future research and trial design.”