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NCT02939547 · ClinicalTrials.gov registry record · Phase 1
Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)
A Phase 1 study of Niemann-Pick Disease, Type C1, sponsored by Cyclo Therapeutics.
- Completed
- Registry status
- Phase 1
- Development phase
- 13
- Enrollment target
- 2
- Study locations
NCT02939547: Completed Phase 1 study of Niemann-Pick Disease, Type C1, sponsored by Cyclo Therapeutics.
NCT02939547 is a Phase 1 study of Niemann-Pick Disease, Type C1 that has completed, run by Cyclo Therapeutics. The registered enrollment target is 13 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (78% lower). The trial reports 2 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02939547, a Phase 1 study of Niemann-Pick Disease, Type C1, has completed, sponsored by Cyclo Therapeutics.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 13 participants
- Enrollment target
- 2
- Study locations
Study 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,.
Primary Outcome
To compare the C max of Trappsol following 2 different dose levels of intravenous Trappsol in patients with NPC-1 disease following single and multiple doses
Conditions Studied
Interventions
- DRUG Hydroxypropyl-beta-cyclodextrin
Study Locations (2)
California
- UCSF Benioff Children's Hospital Oakland - Oakland
New Jersey
- Atlantic Health System/Morristown Medical Center - Morristown
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 13 participants |
| Start Date | 2017-10-11 |
| Est. Completion | 2020-02-10 |
| Phase | Phase 1 |
What the finished NCT02939547 record still lists
NCT02939547 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 13 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (78% lower).
The record links to 1 condition, with Niemann-Pick Disease, Type C1 appearing as the primary indexed condition, and to 1 intervention - of which Hydroxypropyl-beta-cyclodextrin is the first listed.
NCT02939547 reports a single indexed study location in California, New Jersey.
Frequently Asked Questions
What is clinical trial NCT02939547 about?
NCT02939547 is a clinical study titled "Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)". 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...
What is the current status of trial NCT02939547?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 13 participants. The study started on 2017-10-11. Estimated completion is 2020-02-10.
What conditions does trial NCT02939547 study?
This clinical trial studies the following conditions: Niemann-Pick Disease, Type C1.
What interventions are being tested in trial NCT02939547?
The interventions under investigation include: Hydroxypropyl-beta-cyclodextrin (DRUG).
Who is sponsoring clinical trial NCT02939547?
This trial is sponsored by Cyclo Therapeutics, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02939547 being conducted?
This trial has 2 study locations across California, New Jersey. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT02939547's enrollment target sits among peer trials
13 1847th of 2000 higher than 149 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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