Trial record 1 of 8 for:
orphazyme
Early Access Program With Arimoclomol in US Patients With NPC
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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. |
| ClinicalTrials.gov Identifier: NCT04316637 |
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Expanded Access Status :
Available
First Posted : March 20, 2020
Last Update Posted : July 27, 2021
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Sponsor:
Orphazyme
Information provided by (Responsible Party):
Orphazyme
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| Tracking Information | |||||||||
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| 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. |
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| Detailed Description | Not Provided | ||||||||
| Study Type | Expanded Access | ||||||||
| Expanded Access Type | Intermediate-size Population | ||||||||
| Intervention | Drug: Arimoclomol
Participants receive prescribed arimoclomol by oral administration
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| Publications * | Not Provided | ||||||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||||||
| Expanded Access Status | Available | ||||||||
| Contacts |
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| 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 | ||||||||