CTD-TCNPC-201 Phase I/II study of Intravenous Trappsol Cyclo in NPC-1
Research type
Research Study
Full title
A Phase I/II study to evaluate the safety and pharmacokinetics of intravenous Trappsol Cyclo (HP-β-CD) in patients with Niemann-Pick disease type C (NPC-1) and the pharmacodynamic effects of treatment upon markers of cholesterol metabolism and clinical outcomes
IRAS ID
201668
Contact name
Reena Sharma
Contact email
Sponsor organisation
Cyclo Therapeutics
Eudract number
2015-005761-23
Duration of Study in the UK
1 years, 9 months, 4 days
Research summary
This research study is being conducted to find out whether Trappsol® Cyclo™, an experimental treatment for people with Niemann Pick disease Type C (NPC-1) is safe at 3 different dose levels and what effects it has on people who have this condition. NPC-1 is caused by the lack of a protein which is important for the transport of fatty substances like cholesterol out of cells. Without this protein, fats build up in the cells ultimately leading to organ damage. The way in which this experimental treatment works is not fully understood but laboratory experiments have shown that it can potentially remove cholesterol build up from the bodies cells in people who have NPC-1. Approximately 12 patients will be asked to take part in this research study for up to 56 weeks in total. Recruitment is expected to take 9 months. Patients who take part will receive treatment by an intravenous infusion every two weeks. The study will look at what the body does to the drug as well as what the drug does to the body by taking and examining blood and urine samples. Samples of Cerebrospinal fluid (CSF) are also taken by lumbar puncture during and following the first treatment dose. Patients will also have their hearing tested, be asked questions by their doctor as well completing questionnaires to help assess any changes in their condition during treatment. Optional assessments patients can choose to take part in include liver biopsies and additional lumbar punctures for CSF to see if the drug is affecting these. This study is being sponsored and funded by a company called CTD holdings INC. It is planned to be run in the UK, Italy, and Sweden.
REC name
HSC REC A
REC reference
16/NI/0174
Date of REC Opinion
26 Sep 2016
REC opinion
Further Information Favourable Opinion