Cyclo Therapeutics, Inc., a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families suffering from diseases, announced that it has posted its protocol on www.ClinicalTrials.gov and is preparing to commence its pivotal Phase 3 study evaluating Trappsol® Cyclo™, a proprietary formulation of hydroxypropyl beta cyclodextrin, used intravenously, in the treatment of Niemann-Pick Disease type C1 (NPC1). Both the FDA and EMA are in agreement with the Company’s pivotal Phase 3 protocol and have given clearance to proceed with the study.
NPC 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. As cholesterol accumulates in cells, NPC causes symptoms in the brain, liver, spleen, lung and other organs and often leads to premature death. There are currently no approved drug therapies for NPC in the United States and only one approved therapy in Europe.
The pivotal Phase 3 study will be a randomized, double-blind, placebo-controlled, parallel group, multicenter study designed to evaluate the safety, tolerability, and efficacy of 2000 mg/kg of Trappsol® Cyclo™ administered intravenously and standard of care (SOC) compared to placebo administered intravenously and SOC in patients with NPC1. The Phase 3 study intends to enroll at least 93 pediatric (age 3 years and older) and adult patients with NPC1 in at least 23 study centers in 9 countries. Eligible patients will be randomized 2:1 to receive investigational drug or placebo. Randomization will not be constrained based on patient age, nor will patient enrollment be gated by patient age. The study duration is 96 weeks and includes an interim analysis at 48 weeks.
This study has dual primary objectives. For the US and countries following FDA guidance, the primary objective is to evaluate the efficacy of Trappsol® Cyclo™ versus placebo using a 4-Domain Niemann-Pick Disease Type C Severity Scale (4D-NPC-SS [Ambulation, Fine Motor, Speech and Swallow]) composite score at Weeks 48 and 96. For the EU and countries following EMA guidance, the primary objective is to evaluate the efficacy of Trappsol® Cyclo™ versus placebo using the 5-Domain Niemann-Pick Disease Type C Severity Scale (5D-NPC- SS) composite score (Ambulation, Fine Motor, Speech, Swallow, and Cognition) at Weeks 48 and 96.
As previously announced, the Company received a positive opinion from the Paediatric Committee (PDCO) of the EMA and agreement on its Paediatric Investigation Plan (PIP) for Trappsol® Cyclo™. The PIP opinion from PDCO endorsed the clinical program to evaluate the safety, tolerability and efficacy of Trappsol® Cyclo™ in patients from 3 to less than 18 years of age with NPC Type C1, and in addition, to include a single-arm sub-study of patients from birth to less than 3 years of age with NPC Type C1 irrespective of symptoms to evaluate safety and to obtain descriptive data on global disease severity and the response to Trappsol® Cyclo™. The substudy in patients from birth to less than 3 years of age will only be conducted in the EU and countries following EMA guidelines.
Cyclo Therapeutics has received Orphan Drug Designation for Trappsol® Cyclo™ to treat NPC in both the US and EU, and additionally, Fast Track and Rare Pediatric Disease Designations in the US. The Rare Pediatric Disease Designation enables sponsors to receive a Priority Review Voucher for another investigational drug upon marketing authorization of the Rare Pediatric Disease drug in the US.