VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease
Public ClinicalTrials.gov record NCT02534844. 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
A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled 3-Part Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease
Study identification
- NCT ID
- NCT02534844
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2, Phase 3
- Lead sponsor
- Mandos LLC
- Industry
- Enrollment
- 56 participants
Conditions and interventions
Conditions
Interventions
- Parts A/B: Adrabetadex Drug
- Parts A/B: Sham Control Other
Drug · Other
Eligibility (public fields only)
- Age range
- 4 Years to 21 Years
- 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
- Sep 30, 2015
- Primary completion
- Mar 27, 2018
- Completion
- Mar 27, 2018
- Last update posted
- Feb 21, 2023
2015 – 2018
United States locations
- U.S. sites
- 14
- U.S. states
- 12
- U.S. cities
- 14
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35223 | — |
| Children's Hospital of Orange County | Orange | California | 92867 | — |
| University of California San Francisco | San Francisco | California | 94143-0780 | — |
| University of Colorado Denver | Aurora | Colorado | 80045 | — |
| Shands Children's Hospital | Gainesville | Florida | 32608 | — |
| Rush University Medical Center | Chicago | Illinois | 60612 | — |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development | Bethesda | Maryland | 20892-2425 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Montefiore Medical Center | The Bronx | New York | 10467 | — |
| University of North Carolina | Chapel Hill | North Carolina | 27514 | — |
| Lehigh Valley Health Network | Allentown | Pennsylvania | 18101 | — |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Dell Children's Medical Center of Central Texas | Austin | Texas | 78723 | — |
| Multicare Institute for Research and Innovation | Tacoma | Washington | 98405 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 15 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02534844, 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 Feb 21, 2023 · Synced Jul 21, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02534844 live on ClinicalTrials.gov.