Trials / Available
AvailableNCT04316637
Early Access Program With Arimoclomol in US Patients With NPC
Early Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US
- Status
- Available
- Phase
- —
- Study type
- Expanded Access
- Enrollment
- —
- Sponsor
- ZevraDenmark · Industry
- Sex
- All
- Age
- 2 Years
- Healthy volunteers
- —
Summary
NPC is a rare, relentlessly progressive, neurological disease and associated with serious morbidity and shortened life expectancy. The purpose of this Expanded Access Program is to provide early access to arimoclomol for patients with Niemann-Pick Type C disease who, in the opinion and the clinical judgement of the treating physician, may benefit from treatment with arimoclomol. Participants will receive treatment with arimoclomol until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Arimoclomol | Participants receive prescribed arimoclomol by oral administration |
Timeline
- First posted
- 2020-03-20
- Last updated
- 2024-08-16
Locations
13 sites across 1 country: United States
Source: ClinicalTrials.gov record NCT04316637. Inclusion in this directory is not an endorsement.