Springbok Analytics Begins Phase 1 Trial for Clenbuterol in FSHD Treatment

Springbok Analytics, a muscle analytics firm, announced its involvement in a Phase 1 Clinical Trial aimed at evaluating the safe dosage of clenbuterol for treating Facioscapulohumeral muscular dystrophy (FSHD). The company's advanced MRI-based muscle analysis technology provides detailed data on individual muscle volume and fat content in trial participants, aiding researchers in understanding the progression of FSHD. Besides focusing on the safety and tolerance of clenbuterol, Springbok aims to lay a solid groundwork for future FSHD clinical trials.

The trial is part of a broader NIH Wellstone grant led by Drs. Jeff Chamberlain and Stephen Tapscott. The research activities are coordinated by Drs. Jeffrey M. Statland and Rabi Tawil from the University of Kansas and the University of Rochester, respectively. Dr. Seth Friedman at Seattle Children's oversees the imaging procedures. Dr. Friedman expressed enthusiasm about expanding their work with Springbok, highlighting the potential benefits of the technology in providing new insights into the modulation of FSHD by drug therapy. The shorter exam times also improve patient comfort, and the detailed muscle analytics contribute to creating personalized progression models.

FSHD is among the most common muscular dystrophies, affecting around 20,000 people in the United States. Currently, there are no FDA-approved treatments for FSHD, highlighting a significant unmet medical need. Clenbuterol has shown promise in decreasing DUX4-related targets in patient-derived FSHD cell assays, with small studies in other neuromuscular disorders indicating improved muscle strength and function.

Springbok's advancements in artificial intelligence have enhanced both imaging accuracy and processing speed. A recent study on the disease burden among FSHD clinical trial participants revealed a strong preference for imaging over invasive muscle biopsies, which often involve removing healthy muscle tissue. About 85 percent of surveyed FSHD patients expressed willingness to participate in MRI procedures, compared to just 44 percent for open muscle biopsies. However, only 30 percent of those willing to undergo an MRI were prepared to stay in the machine for two hours or more. Springbok's comprehensive body analysis can be completed in under 40 minutes, with scalable protocols designed to meet the needs of global multi-site studies.

Dr. Jeffrey M. Statland from the University of Kansas Medical Center explained the rationale behind the trial: if clenbuterol proves to be safe, well-tolerated, increases muscle mass, and reduces DUX4 levels, it could potentially halt disease progression and restore motor function. He emphasized the importance of Springbok's precision in studying FSHD, which affects individuals and their muscles in varied ways.

Springbok also provides patient-facing reports with 3D muscle visualizations, empowering individuals to better manage their muscle health and track disease progression. Scott Magargee, Springbok's Co-Founder and CEO, noted the importance of the patient experience in clinical trials, particularly concerning scanner time and the availability of reporting. Springbok's technology helps researchers and pharmaceutical companies conduct more effective clinical trials by generating precise and personalized insights, aiding those at the forefront of FSHD research in their quest for better treatments and, ultimately, a cure.

Springbok's imaging protocol is compatible with all major scanner platforms and has been implemented at numerous imaging centers worldwide. The company is also developing standardized guidelines for using muscle MRI as a screening tool for drug trial enrollment. The data from this trial will support a Phase 3 study to confirm clenbuterol's efficacy in treating FSHD.