Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
Public ClinicalTrials.gov record NCT03471143. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Phase 1/2a Study of 2-Hydroxypropyl-Beta-Cyclodextrin Therapy for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
Study identification
- NCT ID
- NCT03471143
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- Washington University School of Medicine
- Other
- Enrollment
- 4 participants
Conditions and interventions
Conditions
Interventions
- VTS-270 Drug
Drug
Eligibility (public fields only)
- Age range
- Up to 6 Months
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Feb 21, 2019
- Primary completion
- Oct 3, 2024
- Completion
- Oct 3, 2024
- Last update posted
- Jan 4, 2026
2019 – 2024
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| St. Louis Children's Hospital | St Louis | Missouri | 63110 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03471143, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jan 4, 2026 · Synced May 18, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03471143 live on ClinicalTrials.gov.