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

NCT03678480 | Withdrawn Phase 2 DMC FDA Drug

• 1 other identifier: HTD1801.PCT006
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Primary Sclerosing Cholangitis; Cholangitis; Cholangitis, Sclerosing; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Adolescent

Outcome Measures

Primary Outcomes (1)

• change in gamma-glutamyl transferase (GGT)

  18 weeks

Secondary Outcomes (7)

• 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

Study Arms (2)

HTD1801 500 mg BID (twice daily), or 1000 mg/day

EXPERIMENTAL

HTD1801 tablets in double-blind capsules, 250 mg

Drug: HTD1801

Ursodeoxycholic Acid (UDCA) 250 mg BID, or 500 mg/day

ACTIVE COMPARATOR

UDCA tablets in double-blind capsules, 250 mg

Drug: Ursodeoxycholic Acid

Interventions

HTD1801 DRUG

HTD1801 capsules, 250mg

HTD1801 500 mg BID (twice daily), or 1000 mg/day

Ursodeoxycholic Acid DRUG

UDCA capsules, 125mg

Ursodeoxycholic Acid (UDCA) 250 mg BID, or 500 mg/day

Eligibility Criteria

• Age: 12 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• HighTide Biopharma Pty Ltd: lead

MeSH Terms

Conditions

Cholangitis, Sclerosing; Cholangitis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases

Interventions

Ursodeoxycholic Acid

Intervention Hierarchy (Ancestors)

Deoxycholic Acid; Cholic Acids; Bile Acids and Salts; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Cholanes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 18, 2018
• First Posted: September 19, 2018
• Study Start: March 1, 2021
• Primary Completion: October 4, 2021
• Study Completion: November 4, 2021
• Last Updated: February 15, 2021

Record last verified: 2020-07

Data Sharing

• IPD Sharing: Will not share