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.

Study Overview

• Status: Withdrawn
• Conditions: Adolescent; Digestive System Diseases; Biliary Tract Diseases; Primary Sclerosing Cholangitis; Cholangitis; Cholangitis, Sclerosing; Bile Duct Diseases
• Intervention / Treatment: Drug: HTD1801; Drug: Ursodeoxycholic Acid

• Study Type: Interventional
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 10 years to 15 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: HTD1801 500 mg BID (twice daily), or 1000 mg/day; HTD1801 tablets in double-blind capsules, 250 mg	Drug: HTD1801; HTD1801 capsules, 250mg
Active Comparator: Ursodeoxycholic Acid (UDCA) 250 mg BID, or 500 mg/day; UDCA tablets in double-blind capsules, 250 mg	Drug: Ursodeoxycholic Acid; UDCA capsules, 125mg

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
change in gamma-glutamyl transferase (GGT)	18 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: HighTide Biopharma Pty Ltd

Study record dates

Study Major Dates

• Study Start (Anticipated): March 1, 2021
• Primary Completion (Anticipated): October 4, 2021
• Study Completion (Anticipated): November 4, 2021

Study Registration Dates

• First Submitted: September 18, 2018
• First Submitted That Met QC Criteria: September 18, 2018
• First Posted (Actual): September 19, 2018

Study Record Updates

• Last Update Posted (Actual): February 15, 2021
• Last Update Submitted That Met QC Criteria: February 10, 2021
• Last Verified: July 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Gastrointestinal Diseases; Digestive System Diseases; Cholangitis; Cholangitis, Sclerosing; Bile Duct Diseases; Biliary Tract Diseases; Gastrointestinal Agents; Cholagogues and Choleretics; Ursodeoxycholic Acid
• Other Study ID Numbers: HTD1801.PCT006

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No