Therapy Areas
Cyclo Therapeutics Commences Patient Enrollment in TransportNPC, a Pivotal Phase 3 Study Evaluating Trappsol Cyclo in Niemann-Pick Type C1
17 June 2021 - - US-based biotechnology company Cyclo Therapeutics, Inc. (NASDAQ: CYTH) has begun its pivotal Phase 3 study evaluating Trappsol Cyclo, a proprietary formulation of hydroxypropyl beta cyclodextrin, delivered intravenously, for the treatment of Niemann-Pick Disease Type C1, the company said.

The TransportNPC study has the regulatory and IRB approval required to commence patient enrollment, and site activation is underway.

NPC is a rare genetic disease affecting 1 in 100,000 live births globally. Approximately 95% of individuals with NPC have mutations in the NPC1 gene and 5% have mutations in the NPC2 gene.

NPC affects nearly every cell in the body due to a deficiency in either the NPC1 or NPC2 protein, which are required for the transport and processing of cholesterol within the cell.

As cholesterol accumulates within cells, NPC causes symptoms that affect the brain, liver, spleen, lung and other organs and often leads to premature death.

The TransportNPC study is 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 compared to placebo administered intravenously and SOC in patients with NPC1.

The TransportNPC study intends to enroll at least 93 pediatric (age three to less than 18 years) and adult patients with NPC1 in at least 23 study centers in nine countries.

Eligible patients will be randomized 2: 1 to receive Trappsol Cyclo 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. The company expects to report topline results from the interim analysis in the first half of 2023.

This study has dual primary objectives based on regulatory preferences. For the US and countries following FDA guidance, the primary objective is to evaluate the mean change from baseline to 48 weeks or 96 weeks as measured by improvement in Trappsol Cyclo versus placebo using a 4-Domain modified Niemann-Pick Disease Type C Severity Scale (4D-NPC-SS [Ambulation, Fine Motor, Speech and Swallow]) composite score.

For the EU and countries following EMA guidance, the primary objective is to evaluate the mean change from baseline to 48 weeks or 96 weeks as measured by improvement in 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).

As previously announced, the company received a positive opinion from the Paediatric Committee of the EMA and agreement on its Paediatric Investigation Plan for Trappsol Cyclo.

The PIP opinion from PDCO endorsed the clinical program to evaluate the safety, tolerability and efficacy of Trappsol Cyclo in pediatric patients of all ages.

The Phase 3 study will consist of a main study cohort that includes pediatric patients with NPC1 from age three to less than 18 years, and in addition, a single-arm sub-study of patients from birth to less than three years of age with NPC1 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 be conducted in the EU, and countries following EMA guidelines, and others as approved by local regulatory authorities.

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 is one of the chief requirements for sponsors to receive a Priority Review Voucher in the US upon marketing authorization.

Cyclo Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families suffering from disease.

The company's Trappsol Cyclo, an orphan drug designated product in the United States and Europe, is the subject of three ongoing formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease.

The company is planning an early phase clinical trial using Trappsol Cyclo intravenously in Alzheimer's Disease based on encouraging data from an Expanded Access program for late-onset Alzheimer's Disease (NCT03624842).