Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
Public ClinicalTrials.gov record NCT04958642. 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 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 this separate registration (NCT04958642), leaving Parts A/B in NCT02534844. The trial's final results for the primary outcome measure of Adverse Events (AE) will be reported here. This study is to evaluate how safe and effective adrabetadex is for participants with Niemann-Pick Type C1 (NPC1) disease who experience neurologic symptoms (listed under Keywords). In Parts A/B (NCT02534844), two out of every 3 participants will receive the study drug. The third participant will receive 1 to 2 small needle pricks at the location where the IT injection is normally made (sham control). In Part C, all participants will receive study drug.
Study identification
- NCT ID
- NCT04958642
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 2, Phase 3
- Enrollment
- 66 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
- Dec 23, 2015 (Actual)
- Primary completion
- Apr 11, 2022 (Actual)
- Completion
- Apr 11, 2022 (Actual)
- Last update posted
- Aug 29, 2023
2015 – 2022
United States locations
- U.S. sites
- 15
- U.S. states
- 13
- U.S. cities
- 15
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35233 | — |
| Children's Hospital of Orange County: CHOC Children's | Orange | California | 92867 | — |
| University of California San Francisco | San Francisco | California | 94143-0780 | — |
| University of Colorado Denver | Aurora | Colorado | 80045 | — |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | — |
| 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 NCT04958642, 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 Aug 29, 2023 · Synced Oct 22, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04958642 live on ClinicalTrials.gov.