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

Phase 2

Withdrawn

• Conditions: Primary Sclerosing Cholangitis; Cholangitis; Cholangitis, Sclerosing; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Adolescent
• Interventions: Drug: HTD1801; Drug: Ursodeoxycholic Acid

• Registration Number: NCT03678480
• Lead Sponsor: HighTide Biopharma Pty Ltd

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-09-18
• Study First Submitted QC: 2018-09-18
• Study First Posted: 2018-09-19
• Status Verified: 2020-07
• Last Update Submitted: 2021-02-10
• Last Update Posted: 2021-02-15
• Study Start: 2021-03-01
• Primary Completion: 2021-10-04
• Study Completion: 2021-11-04

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
HTD1801 500 mg BID (twice daily), or 1000 mg/day	HTD1801	HTD1801 tablets in double-blind capsules, 250 mg
Ursodeoxycholic Acid (UDCA) 250 mg BID, or 500 mg/day	Ursodeoxycholic Acid	UDCA tablets in double-blind capsules, 250 mg

Primary Outcome Measures

Name	Time	Method
change in gamma-glutamyl transferase (GGT)	18 weeks

Secondary Outcome Measures

Name	Time	Method
percentage of patients whose GGT normalizes to <50 units/liter	18 weeks
change in aspartate aminotransferase (AST)	18 weeks
change in alanine aminotransferase (ALT)	18 weeks
change in alkaline phosphatase (ALP)	18 weeks
change in total bilirubin	18 weeks
change in C-reactive protein (CRP)	18 weeks
incidence of adverse events	18 weeks