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.|
|ClinicalTrials.gov Identifier: NCT04316637|
Expanded Access Status : Available
First Posted : March 20, 2020
Last Update Posted : June 18, 2020
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.
|Condition or disease||Intervention/treatment|
|Niemann-Pick Disease, Type C||Drug: Arimoclomol|
|Study Type :||Expanded Access|
|Expanded Access Type :||Intermediate-size Population|
|Official Title:||Early Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US|
- Drug: Arimoclomol
Participants receive prescribed arimoclomol by oral administration
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04316637
|Contact: Orphazyme Inc Medical Information||+1 email@example.com|
|Contact: Clinigen Customer service||+1 firstname.lastname@example.org|
|United States, California|
|Children's Hospital of Orange County (CHOC)||Available|
|Orange, California, United States, 92868|
|Contact: Nina Movsesyan, PhD 714-509-3008 email@example.com|
|Principal Investigator: Raymond Wang, MD|
|United States, Massachusetts|
|Boston Childrens Hospital||Available|
|Boston, Massachusetts, United States, 02215|
|Contact: Eorna Maguire-Lobos 617-919-1399 Eorna.Maguire@childrens.harvard.edu|
|Contact: Olaf Bodamer, MD Olaf.Bodamer@childrens.harvard.edu|