Inborn amino acid metabolism disorder

Search Trials
A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
Status:
Recruiting
Last Changed:
Aug 7, 2019
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Withdrawn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
Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia
Status:
Not yet recruiting
Last Changed:
Jan 21, 2019
First Changed:
Jan 21, 2019
Disease(s):
Methylmalonic Acidemia (MMA)
Intervention(s):
mRNA-3704
Second-line Therapy of Unresectable Cholangiocarcinoma by RADIOEMBOLIZATION
Status:
Terminated
Last Changed:
Aug 7, 2013
First Changed:
Jun 28, 2011
Disease(s):
Cholestasis, Progressive Familial Intrahepatic 3
Intervention(s):
Yttrium microsphere injection
Phase 2 Clinical Trial of SGS-742 Therapy in Succinic Semialdehyde Dehydrogenase Deficiency
Status:
Active, not recruiting
Last Changed:
Oct 26, 2018
First Changed:
Dec 24, 2013
Disease(s):
Metabolic Disease
Intervention(s):
SGS-742Placebo

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