HEPATITIS B CHRONIC
Conditions
Keywords
HEPATITIS B CHRONIC, Gene Therapy, Gene Editing, PBGENE-HBV
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.
Detailed description
Refer to key Inclusion and Exclusion criteria.
Interventions
BIOLOGICALPBGENE-HBV
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.
Sponsors
Precision BioSciences, Inc.
Study design
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
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.
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * 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 Key
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety to Assess Treatment-emergent Adverse Events (TEAEs) | 4 weeks after final dose | Frequency of TEAEs |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Additional Safety | 48 weeks | Frequency and severity of adverse events and changes in physical examinations, vital signs, and safety labs (hematology, chemistry, and urinalysis) |
| Pharmacokinetics of AUC | 4 weeks | Total PBGENE-HBV exposure over time |
| Pharmacokinetics of Cmax | 4 weeks | Time at which Cmax (maximum peak concentration of PBGENE-HBV) is observed |
| Pharmacokinetics of Cmin | 4 weeks | Minimum (or trough) concentration of PBGENE-HBV |
| Pharmacokinetics of half life (t1/2) | 4 weeks | Terminal half life |
| Antiviral Activity of HBsAg and Anti-HBs | 48 weeks | Changes from baseline in hepatitis B surface antigen (HBsAg) and anti-HBs levels |
| Antiviral Activity of HBV DNA | 48 weeks | Changes from baseline of HBV DNA |
Countries
France, Hong Kong, Moldova, New Zealand, Romania, United States
Contacts
CONTACTPrecision Trial Manager
STUDY_DIRECTOROby Sogbetun Medical Monitor, MD
Precision BioSciences, Inc.
Outcome results
None listed