Phase 1 Study to Evaluate Safety and Antiviral Activity of PBGENE-HBV in Adult Patients With Chronic Hepatitis B

NCT06680232 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: PBGENE-HBV-01
• Study Type: interventional
• Target: 45
• Locations: 4 countries
• Sites: 4

Brief Summary

This is a Phase 1, open-label, dose escalation and dose expansion study to evaluate the safety, tolerability, PK, and antiviral activity of PBGENE-HBV in adult participants with chronic hepatitis B.

Conditions

HEPATITIS B CHRONIC

Keywords

HEPATITIS B CHRONIC; Gene Therapy; Gene Editing; PBGENE-HBV

Outcome Measures

Primary Outcomes (1)

• Safety to Assess Treatment-emergent Adverse Events (TEAEs)

  Frequency of TEAEs

  4 weeks after final dose

Secondary Outcomes (7)

• Additional Safety

  48 weeks

• Pharmacokinetics of AUC

  4 weeks

• Pharmacokinetics of Cmax

  4 weeks

• Pharmacokinetics of Cmin

  4 weeks

• Pharmacokinetics of half life (t1/2)

  4 weeks

Study Arms (1)

Participants in both Part 1 and 2 will receive a finite course of PBGENE-HBV.

EXPERIMENTAL

All participants will receive a finite course of multiple IV dose administrations of PBGENE-HBV. In Part 1, this will be done in a dose escalation manner which may be evaluated further in a Part 2 expansion cohort.

Biological: PBGENE-HBV

Interventions

PBGENE-HBV BIOLOGICAL

PBGENE-HBV is an in vivo gene editing intervention based on a novel proprietary ARCUS® platform designed to potentially cure chronic hepatitis B virus (HBV) by eliminating cccDNA, the key source of replicating hepatitis B virus, while also inactivating integrated HBV DNA in hepatocytes.

Participants in both Part 1 and 2 will receive a finite course of PBGENE-HBV.

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or women of non-child bearing potential
• BMI 18.0 to 35.0
• Good overall health deemed by the study Investigator
• CHB infection documented at least 12 months prior to screening
• HBeAg-negative CHB
• Must be virologically suppressed on current NA treatment

You may not qualify if:

• No history of cirrhosis of the liver
• No current infections of Hepatitis A, D, and E, human immunodeficiency virus (type 1 and 2), and no history of or current hepatitis C. In addition, no other active infections deemed clinically relevant.
• No signs of hepatocellular carcinoma
• Not received an organ transplant
• No malignancy within 5 years of screening, except for specific cancers that are cured by surgical resection (e.g., basal cell skin cancer)
• No investigational agent received within 6 months of screening

Sponsors & Collaborators

• Precision BioSciences, Inc.: lead

Study Sites (4)

Massachusetts General Hospital/Harvard University

Boston, Massachusetts, 02114, United States

RECRUITING

Queen Mary Hospital, The University of Hong Kong

Hong Kong, Hong Kong, Hong Kong

RECRUITING

ICS ARENSIA Exploratory Medicine SRL

Chisinau, 2025, Moldova

RECRUITING

New Zealand Clinical Research

Auckland, 1010, New Zealand

RECRUITING

MeSH Terms

Conditions

Hepatitis B, Chronic

Condition Hierarchy (Ancestors)

Hepatitis B; Blood-Borne Infections; Communicable Diseases; Infections; Hepadnaviridae Infections; DNA Virus Infections; Virus Diseases; Hepatitis, Viral, Human; Hepatitis, Chronic; Hepatitis; Liver Diseases; Digestive System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Stanley Frankel, MD Medical Monitor

  Precision BioSciences, Inc.

  STUDY DIRECTOR

Central Study Contacts

Precision Trial Manager

CONTACT

800-371-8953; ELIMINATE-B@precisionbiosciences.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Part 1 is to identify a safe and well tolerated dose regimen of PBGENE-HBV Part 2 is an expansion cohort to aid in selecting a dosing regimen.

Study Record Dates

• First Submitted: October 24, 2024
• First Posted: November 8, 2024
• Study Start: November 14, 2024
• Primary Completion: December 1, 2025
• Study Completion (Estimated): December 1, 2026
• Last Updated: September 26, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

MO (1); US (1); NZ (1); HK (1)