Phase 1 Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of H3B-6527 in Participants With Advanced Hepatocellular Carcinoma

The purpose of this study is to determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of H3B-6527, and to assess the safety, tolerability and pharmacokinetics of H3B-6527.

Inclusion Criteria

• Participants with hepatocellular carcinoma.
• Must have had at least one prior standard-of-care therapy, unless contraindicated.
• Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 or 1.
• Must be willing to undergo a biopsy up to 8 weeks before administration of H3B-6527 on Cycle 1 Day 1 for part 2 (dose expansion).
• Adequate bone marrow and organ function.

Exclusion Criteria

• Uncontrolled significant active infections, except hepatitis B virus (HBV) or hepatitis C virus (HCV).
• Known human immunodeficiency virus infection.
• Presence of gastric or esophageal varices requiring active treatment.
• Previous treatment with a selective FGF19-FGFR4 targeted therapy.
• Females of childbearing potential, or males who have not had a successful vasectomy, who are unable or unwilling to follow adequate contraceptive measures.
• Hereditary problems of galactose intolerance, the Lapp lactase deficiency, or glucose-galactose malabsorption.

Trial Phase Phase I

Trial Type Treatment

Lead Organization
H3 Biomedicine Inc.

• Primary ID H3B-6527-G000-101
• Secondary IDs NCI-2016-01306, 2016-001915-19
• Clinicaltrials.gov ID NCT02834780