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 range3 Years – 60 Years
SexALL
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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.
✓. 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.
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)
. 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.