Protara Therapeutics Announces Patient Dosing In Phase 3 Registrational THRIVE-3 Clinical Trial Evaluating IV Choline Chloride In Patients Receiving Long-Term Parenteral Support

Protara Therapeutics, Inc. (NASDAQ:TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced that patient dosing is now underway in its Phase 3 registrational THRIVE-3 clinical trial evaluating intravenous (IV) Choline Chloride in patients receiving long-term parenteral support (PS). IV Choline Chloride, the Company's investigational phospholipid substrate replacement therapy, was previously granted Fast Track designation by the U.S. Food and Drug Administration.

THRIVE-3 (NCT06910943) is a seamless Phase 2b/3 trial designed to assess the efficacy and safety of low and high dose IV Choline Chloride in adolescent and adult patients receiving long-term PS when oral or enteral nutrition is not possible, insufficient, or contraindicated. Following an 8-week Phase 2b open-label, dose-confirmation trial in 24 patients, approximately 105 additional patients will be enrolled in a 24-week Phase 3 double-blinded, randomized, placebo-controlled trial. The primary endpoint of the trial is the change in plasma choline concentration from baseline compared to placebo. In addition, patients will be eligible to advance to an open-label extension period following completion of each phase of the study.

Choline is an essential quaternary amine that is naturally available in some foods and is widely utilized throughout the human body as an important contributor to many steps of metabolism. It serves as a key methyl donor in multiple metabolic pathways and is abundantly stored as a phospholipid throughout the human body. Patients on PS are unable to synthesize choline from enteral nutrition sources, and there are currently no available PS formulations containing choline. IV choline is recommended for patients receiving PS by the American Society for Parenteral and Enteral Nutrition (ASPEN) in its Recommendations for Changes in Commercially Available Parenteral Multivitamin and Multi–Trace Element Products, as well as by the European Society for Clinical Nutrition and Metabolism (ESPEN) in its Guideline on Home Parenteral Nutrition.