Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)
Status:
Completed
Trial end date:
2020-02-10
Target enrollment:
Participant gender:
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 2 different dose
levels and what effects it has on people who have this condition. NPC-1 is caused by a defect
in the 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 cells in people who have NPC-1. Approximately 12 patients will be asked to take part in
this research study for up to 20 weeks (w) in total (including screening. treatment and
follow-up). Recruitment is expected to take 6- 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. A sample(s) of cerebrospinal fluid (CSF) will be taken by lumbar puncture
during the first treatment dose and may be collected during subsequent doses. Liver and skin
biopsy specimens will be taken to assess filipin staining. Cholesterol metabolism will be
investigated in liver samples and splenic and hepatic elasticity will be assessed by
ultrasound. 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.This study is being sponsored and funded by CTD holdings Inc. It is planned to be
run in the USA,.