Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associate… (NCT03887533) | Clinical Trial Compass
TerminatedPhase 1/2
Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1
Stopped: Enrollment was poor due to COVID-19 pandemic
United States2 participantsStarted 2020-01-06
Plain-language summary
Background:
For people who have Niemann-Pick disease, type C1 (NPC1), cholesterol and other fats have trouble moving out of liver and other tissue cells. This makes the cells sick. Researchers want to find out if a drug called VTS-270 can help.
Objective:
To test if VTS-270 is safe and effective in treating chronic liver disease associated with NPC1.
Eligibility:
People ages 3-60 with NPC1
Design:
Participants may be screened by phone or under another protocol.
Participants will have visits once a month for 12 months. If they have intrathecal injections, the study may last 15 months or more. The first visit will last about 5 days. Others will last 2-3 days.
Participants will get VTS-270 injected into a vein at each visit. They can also choose to have intrathecal injections. These are like spinal taps.
Some visits will also include:
Physical exam
Urine tests
Blood tests. A small tube or needle will be inserted into the participants vein to collect blood. The small tube will also be used to give the VTS-270.
Hearing tests: For one test, participants will have electrodes taped to their head. These will record brain waves.
Breathing tests
Ultrasound of abdomen: Sounds waves will take pictures of the participant s body.
Chest x-ray: This is a picture of the lungs.
Who can participate
Age range
3 Years – 60 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
. Age greater than or equal to 3 and less than or equal to 60 years old at time of enrollment
. Diagnosis of NPC1 based upon one of the following:
. Evidence of NPC1-related liver disease as defined by one of the following:
. Intraparenchymal echogenic bands consistent with fibrosis
. Abnormal liver echogenicity with AST or ALT above the upper limit of normal.
. Hepatomegaly with AST or ALT above the upper limit of normal.
. Ability to travel to the NIH Clinical Center repeatedly for evaluation and follow-up.
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
1
Participants With Adverse Events by Grade
Timeframe: 18 months
2
Participants With Reduction in Plasma Cholestane-3β
Timeframe: Assessed at baseline and at 52 weeks
3
Participants With Reduction in Plasma Bile Acid B (5α)
Timeframe: Assessed at baseline and at 52 weeks
4
Participants With Reduction in C-Triol (6β-triol)
Timeframe: Assessed at baseline and at 52 weeks
Trial details
NCT IDNCT03887533
SponsorEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
. Willingness to discontinue all non-prescription supplements, except for an age-appropriate multivitamin/mineral supplement.
Exclusion criteria
. Unstable frequency, type or duration of seizures. Quantified by a seizure log over one month prior to enrollment.
. Subject requiring antiepileptic medication changes (other than dose adjustments for weight) in the month prior to enrollment.
. Neurologically asymptomatic. Determination made by the investigators based on history, neurological exam and consultant input.
. Suspected infection of the central nervous system
. Spinal deformity that would impact the ability to perform a lumbar puncture
. Skin infection in the lumbar region
. Prior use of anticoagulants or a bleeding disorder with increased risk of clinical bleeding.
. Patients unable to complete a behavioral audiological evaluation including pure-tone threshold assessment (500 Hz to 8000 Hz). In consultation with the medical monitor and audiologists, a sedated ABR may be utilized to monitor ototoxicity if the participant is being sedated to receive IT VTS-270.