Evaluation of Trappsol Cyclo in Niemann-Pick Disease Type C1 Patients
Research type
Research Study
Full title
A Phase 3, Double-blind, Randomised,Placebo-controlled, Parallel-group, Multicentre Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo(Hydroxypropyl-ꞵ-cyclodextrin) and Standard of Care Compared to Placebo and Standard of Care in Patients with Niemann-Pick Disease Type C1
IRAS ID
301215
Contact name
Reena Sharma
Contact email
Sponsor organisation
Cyclo Therapeutics, Inc.
Eudract number
2020-003136-25
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
IND Number, 128390
Duration of Study in the UK
4 years, 3 months, 0 days
Research summary
This is a medical research study investigating the study drug Trappsol Cyclo, an experimental medication being studied to treat patients with Niemann-Pick Type C (NPC1)
Niemann-Pick Disease Type C (NPC) is a rare genetic (inherited) 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 early death.
The focus of this study is NPCI disease (accounts for the largest number of NPC cases).
Trappsol Cyclo is an experimental medication because its safety and effectiveness are still being researched. It has not yet been approved in any country as a treatment for any condition.
The purpose of this research is to test the effect of Trappsol Cyclo and standard of care (SOC) compared with placebo and standard of care, when given 2000 mg/kg by IV infusion (directly into the vein) over 6.5 hours every 2 weeks.
This study intends to enrol about 93 NPCI patients (newborns, children, young people and adults) and 12 patients in a substudy (aged newborn to under 3 years only).
The research will be carried out globally at about 24 study sites, 2 sites will be located in the UK.
Patients over 3 years old will be randomised 2:1 (i.e. by chance) to receive Trappsol Cyclo or placebo (looks like the study drug, but is not active).
Substudy patients (aged newborn to under 3 years) will receive Trappsol Cyclo
Study duration is 100 weeks double-blind treatment or 194 weeks open-label extension (optional)
Study participation is entirely voluntary and information describing the study will be provided.
Consent/assent will be obtained from each participant before any study procedures are performed.
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
21/EM/0218
Date of REC Opinion
14 Dec 2021
REC opinion
Further Information Favourable Opinion