Early Access Program With Arimoclomol in US Patients With NPC
Public ClinicalTrials.gov record NCT04316637. 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
Early Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US
Study identification
- NCT ID
- NCT04316637
- Recruitment status
- Available
- Study type
- Expanded access
- Phase
- Not listed
- Lead sponsor
- ZevraDenmark
- Industry
- Enrollment
- Not listed
Conditions and interventions
Conditions
Interventions
- Arimoclomol Drug
Drug
Eligibility (public fields only)
- Age range
- 2 Years and older
- Sex
- All
- Healthy volunteers
- Not listed
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Not listed
- Primary completion
- Not listed
- Completion
- Not listed
- Last update posted
- Aug 15, 2024
United States locations
- U.S. sites
- 13
- U.S. states
- 10
- U.S. cities
- 13
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Alabama Birmingham | Birmingham | Alabama | 35233 | Available |
| UCSF Benioff Children's Hospital and Research Center/ UCSF | Oakland | California | 94609 | Available |
| Children's Hospital of Orange County (CHOC) | Orange | California | 92868 | Available |
| Nicklaus Children's Hospital | Miami | Florida | 33155 | Available |
| Rush University Medical Center | Chicago | Illinois | 60612 | Available |
| Boston Childrens Hospital | Boston | Massachusetts | 02215 | Available |
| Mayo Clinic Children's Center | Rochester | Minnesota | 55905 | Available |
| New York University School of Medicine | New York | New York | 10017 | Available |
| Cincinnati Children's Hospital Medical Center (CCH) | Cincinnati | Ohio | 45229 | Available |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | Available |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | Available |
| Dell Children's Medical Center | Austin | Texas | 78723 | Available |
| UT Health / McGovern Medical School; Division of Medical Genetics | Houston | Texas | 77030 | Available |
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 NCT04316637, 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 15, 2024 · Synced May 11, 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 NCT04316637 live on ClinicalTrials.gov.