Alpers Syndrome

Search Trials
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
Status:
Not yet recruiting
Last Changed:
May 5, 2022
First Changed:
Feb 1, 2022
Disease(s):
Inherited Mitochondrial Disease
Intervention(s):
Vatiquinone
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
Status:
Recruiting
Last Changed:
Apr 12, 2022
First Changed:
May 7, 2020
Disease(s):
Mitochondrial Diseases
Intervention(s):
VatiquinonePlacebo
A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
Status:
Recruiting
Last Changed:
Dec 21, 2021
First Changed:
Apr 5, 2019
Disease(s):
Progressive Familial Intrahepatic Cholestasis (PFIC)
Intervention(s):
MaralixibatPlacebo
A Placebo-controlled Study of Maralixibat (SHP625) in Pediatric Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)
Status:
Withdrawn
Last Changed:
Mar 18, 2019
First Changed:
Nov 27, 2017
Disease(s):
Progressive Familial Intrahepatic Cholestasis (PFIC)
Intervention(s):
MaralixibatPlacebo
Alpers Huttenlocher Natural History Study
Status:
Unknown status
Last Changed:
Aug 5, 2019
First Changed:
Jan 27, 2017
Disease(s):
Alpers Huttenlocher Syndrome

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