A Study of HTD1801 in Adolescents With Primary Sclerosing Cholangitis (PSC)

A Phase 2, Randomized, Double-Blind Study of HTD1801 vs Ursodeoxycholic Acid (UDCA) in Adolescents With Primary Sclerosing Cholangitis (PSC)

Randomized, double-blind, active-controlled, parallel-group study of HTD1801 in adolescents.

Inclusion Criteria:

• Weight ≥ 35 kg
• Previous cholangiographic evidence of PSC by magnetic resonance cholangiopancreatography (MRCP) or direct cholangiography or liver biopsy findings
• Serum GGT ≥ 2 × upper limit of normal (ULN)
• On a stable UDCA treatment regimen for ≥ 8 weeks

Exclusion Criteria:

• Secondary sclerosing cholangitis
• Percutaneous or endoscopically-placed biliary drain or stent
• History of cholangiocarcinoma or clinical suspicion of new dominant stricture within 1 year
• Ascending cholangitis requiring intravenous antibiotic therapy within 60 days prior to Screening
• Concomitant overlap syndrome with primary biliary cholangitis (PBC)
• Significant hepatic decompensation
• Alternative causes of chronic liver disease
• Hospitalization for colitis within 30 days prior to Screening
• Serum creatinine > 1.2 x ULN
• Hemoglobin < 10 g/dL
• Glucose-6-phosphate dehydrogenase (G6PD) deficiency