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Early Access Program With Arimoclomol in US Patients With NPC

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04316637
Expanded Access Status : Available
First Posted : March 20, 2020
Last Update Posted : July 27, 2021
Information provided by (Responsible Party):

Tracking Information
First Submitted Date March 18, 2020
First Posted Date March 20, 2020
Last Update Posted Date July 27, 2021
Descriptive Information
Brief Title Early Access Program With Arimoclomol in US Patients With NPC
Brief 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.

Detailed Description Not Provided
Study Type Expanded Access
Expanded Access Type Intermediate-size Population
Intervention Drug: Arimoclomol
Participants receive prescribed arimoclomol by oral administration
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Expanded Access Status Available
Contact: Orphazyme Inc Medical Information +1-866-696-3346
Contact: Clinigen Customer service +1 877-768-4303
Listed Location Countries United States
Removed Location Countries  
Administrative Information
NCT Number NCT04316637
Responsible Party Orphazyme
Study Sponsor Orphazyme
Collaborators Not Provided
Investigators Not Provided
PRS Account Orphazyme
Verification Date July 2021