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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Due to different study designs, the sponsor separated Part C into a separate registration (NCT04958642), leaving Parts A/B here in NCT02534844. This study is to find out how safe and effective VTS-270 is for patients with Niemann-Pick Type C1 (NPC1) disease who have neurologic symptoms (listed under Keywords). In Parts A/B, two out of every three patients will receive the study drug. The third patient will receive 1 to 2 small needle pricks at the location where the LP and IT injection is normally made (sham control). In Part C, all participants will receive study drug, as described in the Part C registration record. Start date for this record is the first day a participant was enrolled in Parts A/B. The trial is actually continuing until the last primary outcome measure of safety data are collected from Part C participants. The last primary outcome measure of safety, along with final adverse events results will be posted in the separate Part C registration record.
Study identification
- NCT ID
- NCT02534844
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2, Phase 3
- Enrollment
- 56 participants
Conditions and interventions
Conditions
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
- Oct 2015 (Actual)
- Primary completion
- Mar 28, 2018 (Actual)
- Completion
- Mar 28, 2018 (Actual)
- Last update posted
- Feb 22, 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 22, 2023 · Synced Oct 22, 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 public registry record, listed eligibility criteria, and available contact information for NCT02534844 live on ClinicalTrials.gov.