NCT02939547

Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)

Completed Phase 1 Last updated 21 February 2021

What this trial tests

Phase 1 trial testing Hydroxypropyl-beta-cyclodextrin in Niemann-Pick Disease, Type C1 in 13 participants. Completed in 10 February 2020.

Timeline

11 October 2017

Primary endpoint
10 February 2020

10 February 2020

Quick facts

Lead sponsor	Cyclo Therapeutics, Inc.
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	basic science
Enrollment	13
Start date	11 October 2017
Primary completion	10 February 2020
Estimated completion	10 February 2020
Sites	2 locations across United States

Drugs / interventions tested

• Hydroxypropyl-beta-cyclodextrin — full drug profile →

Conditions studied

• Niemann-Pick Disease, Type C1 — all drugs for Niemann-Pick Disease, Type C1 →

Sponsor

Cyclo Therapeutics, Inc. — full company profile →

Who can join

18 and older, any sex, with Niemann-Pick Disease, Type C1. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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,.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial.
   Ory DS, Ottinger EA, Farhat NY, King KA, et al · · 2017 · cited 268× · PMID 28803710 · DOI 10.1016/s0140-6736(17)31465-4
2. The Extending Spectrum of NPC1-Related Human Disorders: From Niemann-Pick C1 Disease to Obesity.
   Lamri A, Pigeyre M, Garver WS, Meyre D. · · 2018 · cited 41× · PMID 29325023 · DOI 10.1210/er.2017-00176
3. Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine.
   Las Heras M, Szenfeld B, Ballout RA, Buratti E, et al · · 2023 · cited 40× · PMID 37567876 · DOI 10.1038/s41525-023-00365-w
4. Cyclodextrins: Only Pharmaceutical Excipients or Full-Fledged Drug Candidates?
   Kovacs T, Nagy P, Panyi G, Szente L, et al · · 2022 · cited 40× · PMID 36559052 · DOI 10.3390/pharmaceutics14122559
5. Intravenous 2-hydroxypropyl-β-cyclodextrin (Trappsol® Cyclo™) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial.
   Hastings C, Liu B, Hurst B, Cox GF, et al · · 2022 · cited 31× · PMID 36279795 · DOI 10.1016/j.ymgme.2022.10.004
6. 2-Hydroxypropyl-β-cyclodextrins and the Blood-Brain Barrier: Considerations for Niemann-Pick Disease Type C1.
   Calias P. · · 2017 · cited 31× · PMID 29065825 · DOI 10.2174/1381612823666171019164220
7. Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis.
   Hastings C, Vieira C, Liu B, Bascon C, et al · · 2019 · cited 28× · PMID 31639011 · DOI 10.1186/s13023-019-1207-1
8. Recent neuroimaging, neurophysiological, and neuropathological advances for the understanding of NPC.
   Benussi A, Cotelli MS, Padovani A, Borroni B. · · 2018 · cited 16× · PMID 29511534 · DOI 10.12688/f1000research.12361.1