Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
Public ClinicalTrials.gov record NCT03643562. 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
Open-label Evaluation of Adrabetadex in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)
Study identification
- NCT ID
- NCT03643562
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- Mandos LLC
- Industry
- Enrollment
- 27 participants
Conditions and interventions
Conditions
Interventions
- Adrabetadex Drug
Drug
Eligibility (public fields only)
- Age range
- 4 Years and older
- 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
- Jun 17, 2018
- Primary completion
- Nov 1, 2021
- Completion
- Nov 1, 2021
- Last update posted
- Jan 16, 2024
2018 – 2021
United States locations
- U.S. sites
- 17
- U.S. states
- 14
- U.S. cities
- 16
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Arkansas System | Little Rock | Arkansas | 72205 | — |
| Loma Linda University Health System | Loma Linda | California | 92354 | — |
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | — |
| Children's Hospital of Orange County | Orange | California | 92868 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Children's National Hospital | Washington D.C. | District of Columbia | 20010 | — |
| Rare Disease Research, LLC | Atlanta | Georgia | 30318 | — |
| Rush University Medical Center | Chicago | Illinois | 60612 | — |
| Gillette Children's Specialty Healthcare | Saint Paul | Minnesota | 55101 | — |
| Children's Specialty Center of Nevada | Las Vegas | Nevada | 89109 | — |
| NYU Langone Medical Center | New York | New York | 10017 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| Oregon Health & Science University | Portland | Oregon | 97239 | — |
| The Children's Hospital at TriStar Centennial | Nashville | Tennessee | 37203 | — |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | — |
| Dell Children's Medical Center of Central Texas | Austin | Texas | 78723 | — |
| Carilion Medical Center, Carilion Clinic | Roanoke | Virginia | 24014 | — |
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 NCT03643562, 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 16, 2024 · Synced Jul 21, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03643562 live on ClinicalTrials.gov.