Cyclo Therapeutics will now proceed in the US with its Phase 3 clinical trial of Trappsol® Cyclo™ for treatment of Niemann-Pick Disease Type C
Cyclo Therapeutics, Inc., a clinical stage biotechnology company developing a cyclodextrin platform for the treatment of Neurodegenerative Diseases, including their lead candidate (Trappsol® Cyclo™) in the treatment of Niemann-Pick Disease Type C (NPC) announced that the company received notification from the US FDA that enrollment can proceed for its Phase 3 global pivotal clinical trial, “A Phase 3, Double-blind, Randomized, Placebo-controlled, Parallel-group, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo™ (Hydroxypropyl-β-cyclodextrin) and Standard of Care Compared to Placebo and Standard of Care in Patients with Niemann-Pick Disease Type C1”.
Niemann-Pick Disease Type C is a rare genetic disease affecting 1 in 100,000 live births globally. NPC affects every cell in the body due to a defect in the NPC1 protein which is responsible for cholesterol processing in the cell. NPC causes symptoms in the brain, liver, spleen, lung and other organs and often leads to premature death. There are no approved drug therapies for NPC in the United States and only one approved therapy in Europe.