UnknownPhase 1

Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

United States12 participantsStarted 2019-05-23

Plain-language summary

The purpose of this study is to provide continued access to treatment for NPC-1 after participation and completion of the Phase I trial CTD-TCNPC-101, when administered at doses of 1500 mg/kg and 2500 mg/kg by slow IV infusion over a period of 8 to 9 hours every two weeks.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Completion of study CTD-TCNPC-101 with no safety concerns at the final visit * Negative urine pregnancy test for females of child bearing potential * Patients must be legally resident in the USA with access to healthcare * Written, informed consent Exclusion Criteria: * Inability to comply with the proposed protocol assessments or any uncertainty about their ability to give meaningful, informed consent (legal guardian may give consent with subject assent) * Concurrent medical conditions representing a contraindication to any of the study medications * Grade 3 renal impairment or worse as indicated by estimated Glomerular filtration rate (eGFR) \< 60mL/min/1.73m2 * Clinical evidence of acute liver disease including symptoms of jaundice or right upper quadrant pain or International Normalised Ratio (INR) \>1.8 * Male patients and female patients of childbearing potential who are not willing to use appropriate birth control (i.e. double barrier birth control) from enrollment until the follow-up visit

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product

Timeframe: 1-104 weeks

Serious adverse events (SAEs): Frequency, severity, time to onset, duration and relatedness to study product

Timeframe: 1-104 weeks

Number of discontinuations due to AEs: Patient Discontinuation, Study Treatment Discontinuation, Study and Site Discontinuation

Timeframe: 1-104 weeks

Auditory capacity will be measured by behavioral auditory assessment

Timeframe: 1-104 weeks

Trial details

NCT IDNCT03893071

SponsorCyclo Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-03

View on ClinicalTrials.gov More Niemann-Pick Disease, Type C1 trials

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product

Timeframe: 1-104 weeks

Serious adverse events (SAEs): Frequency, severity, time to onset, duration and relatedness to study product

Timeframe: 1-104 weeks

Number of discontinuations due to AEs: Patient Discontinuation, Study Treatment Discontinuation, Study and Site Discontinuation

Timeframe: 1-104 weeks

Auditory capacity will be measured by behavioral auditory assessment

Timeframe: 1-104 weeks