A Phase 2b Study of Zagociguat in Patients with MELAS
PRIZM is a Phase 2b randomized, double-blind, placebo-controlled, 3-treatment, 2-period,
crossover study evaluating the efficacy and safety of oral zagociguat 15 and 30 mg vs.
placebo when administered daily for 12 weeks in participants with genetically and
phenotypi...
Age: 18 - 75 years
Gender: All
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an
inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for
detoxification of ammonia. Individuals with OTC deficiency can build-up excess levels of
...
Age: 24 hours - 7 months
Gender: Male
TReatment for ImmUne Mediated PathopHysiology
TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three arm,
randomized, controlled trial of immunosuppressive therapy for children with acute liver
failure. The study will determine if suppressing inflammatory responses with either
corticost...
Age: 1 year - 18 years
Gender: All
TReatment for ImmUne Mediated PathopHysiology
TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three arm, randomized, controlled trial of immunosuppressive therapy for children with acute liver failure. The study will determine if suppressing inflammatory responses with either corticostero...
Age: Birth - 18 years
Gender: All