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NCT03887533 · ClinicalTrials.gov registry record · Phase 1
Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1
A Phase 1 study, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
- Terminated
- Registry status
- Phase 1
- Development phase
- 2
- Enrollment target
NCT03887533 is a Phase 1 study that was terminated before completion, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). The registered enrollment target is 2 participants.
The verdict
NCT03887533, a Phase 1 study, was terminated before completion, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
- TERMINATED
- Registry status
- Phase 1
- Development phase
- 2 participants
- Enrollment target
Study 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.
Interventions
- DRUG VTS-270
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 2 participants |
| Start Date | 2020-01-06 |
| Est. Completion | 2021-10-25 |
| Phase | Phase 1 |
Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)305 total trials
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03887533
The ClinicalTrials.gov registry entry for NCT03887533 describes a study currently listed as terminated, categorized as Phase 1. The registered enrollment target is 2 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), which has 305 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which VTS-270 is the first listed.
NCT03887533 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT03887533 about?
NCT03887533 is a clinical study titled "Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1". 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 ...
What is the current status of trial NCT03887533?
This trial is currently terminated. It is a Phase 1 study. The enrollment target is 2 participants. The study started on 2020-01-06. Estimated completion is 2021-10-25.
What interventions are being tested in trial NCT03887533?
The interventions under investigation include: VTS-270 (DRUG).
Who is sponsoring clinical trial NCT03887533?
This trial is sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), which has 305 total clinical trials registered on ClinicalTrials.gov.
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