In April, clinical research teams activated the phase 3 CAREFENDR study led locally by Alexandra Phan, MD, FACP, Professor of Hematology and Oncology. The global trial evaluates whether a once-daily oral therapy can better control symptoms of carcinoid syndrome in patients with well-differentiated neuroendocrine tumors (NETs).

NETs are a rare group of cancers that often grow slowly and can develop in the gastrointestinal tract, pancreas, or lungs. In some patients, these tumors release excess hormones into the bloodstream, leading to carcinoid syndrome, a condition that can cause frequent diarrhea, flushing, and other symptoms that significantly impact daily life. While people may live with these tumors for many years, managing symptoms over time can be challenging.

Current treatments are designed to suppress hormone release, but they are typically given as injections every few weeks. CAREFENDR is testing whether paltusotine, a once-daily pill, can reduce hormone production in a more consistent and convenient way, potentially allowing patients to manage symptoms from home. The study also evaluates how well paltusotine controls symptoms and its safety over time, helping researchers understand its potential as a long-term treatment option.

If successful, this approach could shift how carcinoid syndrome is managed, moving from periodic injections to a daily oral therapy that offers more consistent control, fewer clinic visits, and greater flexibility in patients’ lives.

By bringing studies like CAREFENDR to patients locally, the MCW Cancer Center plays an important role in advancing new treatment options for rare cancers and ensuring patients have access to promising therapies close to home.

The study’s sponsor, Crinetics Pharmaceuticals Inc., aims to enroll approximately 140 participants worldwide.

Learn about the trial.