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
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
Longitudinal Study of Urea Cycle Disorders
Urea cycle disorders (UCD) are a group of rare inherited metabolism disorders. Infants and
children with UCD commonly experience episodes of vomiting, lethargy, and coma. The purpose
of this study is to perform a long-term analysis of a large group of individuals wi...
Age: Birth - 66+
Gender: All