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Early Access Program With Arimoclomol in US Patients With NPC

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ClinicalTrials.gov Identifier: NCT04316637
Expanded Access Status : Available
First Posted : March 20, 2020
Last Update Posted : October 9, 2020
Sponsor:
Information provided by (Responsible Party):
Orphazyme

Tracking Information
First Submitted Date March 18, 2020
First Posted Date March 20, 2020
Last Update Posted Date October 9, 2020
 
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 ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Expanded Access Status Available
Contacts
Contact: Orphazyme Inc Medical Information +1 773-770-6888 usmedicalaffairs@orphazyme.com
Contact: Clinigen Customer service +1 877-768-4303 usmapoperations@clinigengroup.com
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 September 2020