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NCT03471143 · ClinicalTrials.gov registry record · Phase 1
Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
A Phase 1 study, sponsored by Washington University School of Medicine.
- Completed
- Registry status
- Phase 1
- Development phase
- 4
- Enrollment target
NCT03471143 is a Phase 1 study that has completed, run by Washington University School of Medicine. The registered enrollment target is 4 participants.
The verdict
NCT03471143, a Phase 1 study, has completed, sponsored by Washington University School of Medicine.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 4 participants
- Enrollment target
Study Summary
Niemann-Pick disease, type C (NPC) is a lethal, autosomal recessive, lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence. NPC results from mutation of either the Niemann-Pick C1 disease (NPC1) (\~95% of cases) or NPC2 genes. NPC is characterized by the endolysosomal storage of unesterified cholesterol and lipids in both the central nervous system and peripheral tissues such as the liver. Individuals with NPC demonstrate progressive central nervous system decline including inability to coordinate balance, gait, extremity and eye movements. Acute liver disease in the newborn/infant period is frequently observed, but subsequently resolves. However, chronic, sub-clinical liver disease persists. Intrathecal 2-Hydroxypropyl-β-Cyclodextrin (HP-β-CD, VTS-270), also known as adrabetadex, has proven effective in reducing the signs and prolonging life in animal models and Phase 1/2a data support efficacy in NPC1 patients. Adrabetadex (VTS-270) also has been shown to be effective in treating liver disease in the NPC1 cat. This Phase 1/2a, open-label, multiple ascending dose trial will evaluate whether adrabetadex (VTS-270) administered intravenously is effective in treating acute liver disease in NPC1 infants.
Interventions
- DRUG VTS-270
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2019-02-22 |
| Est. Completion | 2024-10-04 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03471143
The ClinicalTrials.gov registry entry for NCT03471143 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 4 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Washington University School of Medicine, which has 1,502 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.
NCT03471143 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT03471143 about?
NCT03471143 is a clinical study titled "Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C". Niemann-Pick disease, type C (NPC) is a lethal, autosomal recessive, lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence. NPC results from mutation of either the Niemann-Pick C1 disease (NPC1) (\~95% of cases) or NPC2 genes. NPC is characterized ...
What is the current status of trial NCT03471143?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 4 participants. The study started on 2019-02-22. Estimated completion is 2024-10-04.
What interventions are being tested in trial NCT03471143?
The interventions under investigation include: VTS-270 (DRUG).
Who is sponsoring clinical trial NCT03471143?
This trial is sponsored by Washington University School of Medicine, which has 1,502 total clinical trials registered on ClinicalTrials.gov.
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