Trial Outcomes & Findings for A Multi-dose Study of ARC-520 in Patients With Hepatitis B 'e' Antigen (HBeAg) Negative, Chronic Hepatitis B Virus (HBV) Infection (NCT NCT02604199)

NCT ID: NCT02604199

Last Updated: 2026-01-13

Results Overview

Change From Baseline in log qHBsAg at Day 113 in response to multiple doses of ARC-520 versus placebo, using a a mixed effect model for repeated measures (MMRM). Includes parameter baseline as a continuous covariate, and treatment and visit as fixed factors, interaction of treatment and visit, and interaction of parameter baseline and visit.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

58 participants

Primary outcome timeframe

Baseline, Day 113

Results posted on

2026-01-13

Participant Flow

Participant milestones

Participant milestones
Measure
PBO Low Dose
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
PBO High Dose
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 1 mg/kg
Intravenous ARC-520 at 1.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 2 mg/kg
Intravenous ARC-520 at 2.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
Overall Study
STARTED
9
11
17
21
Overall Study
COMPLETED
9
8
17
18
Overall Study
NOT COMPLETED
0
3
0
3

Reasons for withdrawal

Reasons for withdrawal
Measure
PBO Low Dose
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
PBO High Dose
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 1 mg/kg
Intravenous ARC-520 at 1.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 2 mg/kg
Intravenous ARC-520 at 2.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
Overall Study
Pregnancy
0
0
0
1
Overall Study
Study terminated by sponsor
0
2
0
2
Overall Study
Withdrawal by Subject
0
1
0
0

Baseline Characteristics

A Multi-dose Study of ARC-520 in Patients With Hepatitis B 'e' Antigen (HBeAg) Negative, Chronic Hepatitis B Virus (HBV) Infection

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
PBO Low Dose
n=9 Participants
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
PBO High Dose
n=11 Participants
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 1 mg/kg
n=17 Participants
Intravenous ARC-520 at 1.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 2 mg/kg
n=21 Participants
Intravenous ARC-520 at 2.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
Total
n=58 Participants
Total of all reporting groups
Age, Continuous
46.2 years
STANDARD_DEVIATION 12.10 • n=9 Participants
48.7 years
STANDARD_DEVIATION 9.50 • n=6 Participants
45.0 years
STANDARD_DEVIATION 10.48 • n=9 Participants
45.7 years
STANDARD_DEVIATION 10.48 • n=205 Participants
46.2 years
STANDARD_DEVIATION 10.29 • n=16 Participants
Sex: Female, Male
Female
2 Participants
n=9 Participants
1 Participants
n=6 Participants
7 Participants
n=9 Participants
9 Participants
n=205 Participants
19 Participants
n=16 Participants
Sex: Female, Male
Male
7 Participants
n=9 Participants
10 Participants
n=6 Participants
10 Participants
n=9 Participants
12 Participants
n=205 Participants
39 Participants
n=16 Participants

PRIMARY outcome

Timeframe: Baseline, Day 113

Population: Intent to treat population: all participants who received at least 1 dose of study drug and had valid qHBsAg values at baseline and at least one time point on or after the Day 15 visit.

Change From Baseline in log qHBsAg at Day 113 in response to multiple doses of ARC-520 versus placebo, using a a mixed effect model for repeated measures (MMRM). Includes parameter baseline as a continuous covariate, and treatment and visit as fixed factors, interaction of treatment and visit, and interaction of parameter baseline and visit.

Outcome measures

Outcome measures
Measure
PBO High Dose
n=14 Participants
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
PBO Low Dose
n=15 Participants
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 2 mg/kg
n=15 Participants
Intravenous ARC-520 at 2.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 2 mg/kg
Intravenous ARC-520 at 2.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
Change From Baseline in Quantitative Hepatitis B Surface Antigen (Log qHBsAg) at Day 113
-0.157 log IU/mL
Standard Error 0.036
-0.070 log IU/mL
Standard Error 0.033
-0.379 log IU/mL
Standard Error 0.033

SECONDARY outcome

Timeframe: Baseline, Day 15, 29, 43, 57, 71, 85, 99

Population: Intent to treat population: all participants who received at least 1 dose of study drug and had valid qHBsAg values at baseline and at least one time point on or after the Day 15 visit.

Change From Baseline in log qHBsAg at Day 113 in response to multiple doses of ARC-520 versus placebo, using a a mixed effect model for repeated measures (MMRM). Includes parameter baseline as a continuous covariate, and treatment and visit as fixed factors, interaction of treatment and visit, and interaction of parameter baseline and visit.

Outcome measures

Outcome measures
Measure
PBO High Dose
n=15 Participants
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
PBO Low Dose
n=18 Participants
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 2 mg/kg
n=19 Participants
Intravenous ARC-520 at 2.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 2 mg/kg
Intravenous ARC-520 at 2.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
Change From Baseline in Quantitative Hepatitis B Surface Antigen (Log qHBsAg) Over Time
Day 57
3.072 log IU/mL
Standard Error 1.061
3.291 log IU/mL
Standard Error 0.705
2.329 log IU/mL
Standard Error 0.971
Change From Baseline in Quantitative Hepatitis B Surface Antigen (Log qHBsAg) Over Time
Day 15
3.084 log IU/mL
Standard Error 1.004
3.297 log IU/mL
Standard Error 0.712
2.506 log IU/mL
Standard Error 0.844
Change From Baseline in Quantitative Hepatitis B Surface Antigen (Log qHBsAg) Over Time
Day 29
3.098 log IU/mL
Standard Error 0.986
3.291 log IU/mL
Standard Error 0.694
2.503 log IU/mL
Standard Error 0.846
Change From Baseline in Quantitative Hepatitis B Surface Antigen (Log qHBsAg) Over Time
Day 43
3.016 log IU/mL
Standard Error 1.124
3.188 log IU/mL
Standard Error 0.712
2.509 log IU/mL
Standard Error 0.836
Change From Baseline in Quantitative Hepatitis B Surface Antigen (Log qHBsAg) Over Time
Day 71
3.051 log IU/mL
Standard Error 1.029
3.206 log IU/mL
Standard Error 0.663
2.301 log IU/mL
Standard Error 0.969
Change From Baseline in Quantitative Hepatitis B Surface Antigen (Log qHBsAg) Over Time
Day 85
2.907 log IU/mL
Standard Error 0.963
3.309 log IU/mL
Standard Error 0.736
2.366 log IU/mL
Standard Error 0.862
Change From Baseline in Quantitative Hepatitis B Surface Antigen (Log qHBsAg) Over Time
Day 99
2.938 log IU/mL
Standard Error 1.055
3.302 log IU/mL
Standard Error 0.700
2.360 log IU/mL
Standard Error 0.950

SECONDARY outcome

Timeframe: Through Day 169

Population: Safety Population: all participants who received at least 1 dose of study treatment or placebo, and had at least 1 post-dose safety assessment.

An AE is any untoward medical occurrence which does not necessarily have a causal relationship with this treatment. An SAE is any AE that: results in death; is life-threatening; requires inpatient hospitalization or prolongation of an existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; or is a medically important event or reaction. A treatment emergent AE (TEAE) was defined as an AE that was not present prior to the first study medication administration and started at/after the time of initiation of administration of study medication, or an AE which was present prior to initiation of study medication administration, which increased in severity after study medication administration.

Outcome measures

Outcome measures
Measure
PBO High Dose
n=11 Participants
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
PBO Low Dose
n=9 Participants
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 2 mg/kg
n=17 Participants
Intravenous ARC-520 at 2.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 2 mg/kg
n=21 Participants
Intravenous ARC-520 at 2.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Deaths and Discontinuations Due to AEs
≥ 1 AE
5 Participants
4 Participants
6 Participants
12 Participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Deaths and Discontinuations Due to AEs
≥ 1 TEAE
4 Participants
4 Participants
6 Participants
12 Participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Deaths and Discontinuations Due to AEs
≥ 1 Serious TEAE
0 Participants
0 Participants
2 Participants
1 Participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Deaths and Discontinuations Due to AEs
Deaths
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Deaths and Discontinuations Due to AEs
≥ 1 TEAE Leading to Study Discontinuation
0 Participants
0 Participants
0 Participants
0 Participants
Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Deaths and Discontinuations Due to AEs
≥ 1 TEAE Leading to Treatment Discontinuation
0 Participants
0 Participants
0 Participants
0 Participants

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Day 1 and Day 85

Population: Due to the early suspension and termination of the study, all pharmacokinetic analyses were removed from the initial statistical analysis plan for this study. No collected samples were analyzed and all were destroyed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Day 1 and Day 85

Population: Due to the early suspension and termination of the study, all pharmacokinetic analyses were removed from the initial statistical analysis plan for this study. No collected samples were analyzed and all were destroyed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Day 1 and Day 85

Population: Due to the early suspension and termination of the study, all pharmacokinetic analyses were removed from the initial statistical analysis plan for this study. No collected samples were analyzed and all were destroyed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Day 1 and Day 85

Population: Due to the early suspension and termination of the study, all pharmacokinetic analyses were removed from the initial statistical analysis plan for this study. No collected samples were analyzed and all were destroyed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Day 1 and Day 85

Population: Due to the early suspension and termination of the study, all pharmacokinetic analyses were removed from the initial statistical analysis plan for this study. No collected samples were analyzed and all were destroyed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Day 1 and Day 85

Population: Due to the early suspension and termination of the study, all pharmacokinetic analyses were removed from the initial statistical analysis plan for this study. No collected samples were analyzed and all were destroyed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Day 1 and Day 85

Population: Due to the early suspension and termination of the study, all pharmacokinetic analyses were removed from the initial statistical analysis plan for this study. No collected samples were analyzed and all were destroyed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Day 1 and Day 85

Population: Due to the early suspension and termination of the study, all pharmacokinetic analyses were removed from the initial statistical analysis plan for this study. No collected samples were analyzed and all were destroyed.

Outcome measures

Outcome data not reported

Adverse Events

PBO Low Dose

Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths

PBO High Dose

Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths

ARC-520 Injection 1 mg/kg

Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths

ARC-520 Injection 2 mg/kg

Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
PBO Low Dose
n=9 participants at risk
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
PBO High Dose
n=11 participants at risk
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 1 mg/kg
n=17 participants at risk
Intravenous ARC-520 at 1.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 2 mg/kg
n=21 participants at risk
Intravenous ARC-520 at 2.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
General disorders
Pyrexia
0.00%
0/9 • Through Day 169
0.00%
0/11 • Through Day 169
5.9%
1/17 • Through Day 169
4.8%
1/21 • Through Day 169
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Cholangiocarcinoma
0.00%
0/9 • Through Day 169
0.00%
0/11 • Through Day 169
5.9%
1/17 • Through Day 169
0.00%
0/21 • Through Day 169

Other adverse events

Other adverse events
Measure
PBO Low Dose
n=9 participants at risk
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
PBO High Dose
n=11 participants at risk
0.9% normal saline, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 1 mg/kg
n=17 participants at risk
Intravenous ARC-520 at 1.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
ARC-520 Injection 2 mg/kg
n=21 participants at risk
Intravenous ARC-520 at 2.0 mg/kg, once every 4 weeks for 4 doses plus daily oral entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg) throughout the study period
General disorders
Fatigue
0.00%
0/9 • Through Day 169
0.00%
0/11 • Through Day 169
5.9%
1/17 • Through Day 169
9.5%
2/21 • Through Day 169
General disorders
Influenza like illness
0.00%
0/9 • Through Day 169
0.00%
0/11 • Through Day 169
5.9%
1/17 • Through Day 169
4.8%
1/21 • Through Day 169
General disorders
Malaise
11.1%
1/9 • Through Day 169
0.00%
0/11 • Through Day 169
0.00%
0/17 • Through Day 169
4.8%
1/21 • Through Day 169
General disorders
Pyrexia
0.00%
0/9 • Through Day 169
0.00%
0/11 • Through Day 169
5.9%
1/17 • Through Day 169
9.5%
2/21 • Through Day 169
Infections and infestations
Nasopharyngitis
0.00%
0/9 • Through Day 169
9.1%
1/11 • Through Day 169
0.00%
0/17 • Through Day 169
4.8%
1/21 • Through Day 169
Infections and infestations
Tonsillitis
0.00%
0/9 • Through Day 169
0.00%
0/11 • Through Day 169
5.9%
1/17 • Through Day 169
0.00%
0/21 • Through Day 169
Infections and infestations
Upper respiratory tract infection
11.1%
1/9 • Through Day 169
9.1%
1/11 • Through Day 169
5.9%
1/17 • Through Day 169
14.3%
3/21 • Through Day 169
Investigations
Aspartate aminotransferase increased
11.1%
1/9 • Through Day 169
0.00%
0/11 • Through Day 169
0.00%
0/17 • Through Day 169
0.00%
0/21 • Through Day 169
Investigations
Blood cholesterol increased
0.00%
0/9 • Through Day 169
0.00%
0/11 • Through Day 169
5.9%
1/17 • Through Day 169
0.00%
0/21 • Through Day 169
Investigations
Blood creatine phosphokinase increased
11.1%
1/9 • Through Day 169
0.00%
0/11 • Through Day 169
0.00%
0/17 • Through Day 169
0.00%
0/21 • Through Day 169
Musculoskeletal and connective tissue disorders
Arthralgia
11.1%
1/9 • Through Day 169
0.00%
0/11 • Through Day 169
0.00%
0/17 • Through Day 169
0.00%
0/21 • Through Day 169
Musculoskeletal and connective tissue disorders
Myalgia
0.00%
0/9 • Through Day 169
0.00%
0/11 • Through Day 169
5.9%
1/17 • Through Day 169
0.00%
0/21 • Through Day 169
Nervous system disorders
Headache
0.00%
0/9 • Through Day 169
0.00%
0/11 • Through Day 169
5.9%
1/17 • Through Day 169
4.8%
1/21 • Through Day 169
Nervous system disorders
Lethargy
11.1%
1/9 • Through Day 169
0.00%
0/11 • Through Day 169
0.00%
0/17 • Through Day 169
0.00%
0/21 • Through Day 169
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
0.00%
0/9 • Through Day 169
9.1%
1/11 • Through Day 169
0.00%
0/17 • Through Day 169
0.00%
0/21 • Through Day 169
Skin and subcutaneous tissue disorders
Pruritus
0.00%
0/9 • Through Day 169
9.1%
1/11 • Through Day 169
0.00%
0/17 • Through Day 169
0.00%
0/21 • Through Day 169
Skin and subcutaneous tissue disorders
Rash
0.00%
0/9 • Through Day 169
0.00%
0/11 • Through Day 169
5.9%
1/17 • Through Day 169
0.00%
0/21 • Through Day 169
Vascular disorders
Hypotension
11.1%
1/9 • Through Day 169
0.00%
0/11 • Through Day 169
0.00%
0/17 • Through Day 169
0.00%
0/21 • Through Day 169
General disorders
Chest discomfort
0.00%
0/9 • Through Day 169
9.1%
1/11 • Through Day 169
0.00%
0/17 • Through Day 169
4.8%
1/21 • Through Day 169
General disorders
Chills
0.00%
0/9 • Through Day 169
0.00%
0/11 • Through Day 169
5.9%
1/17 • Through Day 169
0.00%
0/21 • Through Day 169
Cardiac disorders
Sinus bradycardia
11.1%
1/9 • Through Day 169
0.00%
0/11 • Through Day 169
0.00%
0/17 • Through Day 169
0.00%
0/21 • Through Day 169
Gastrointestinal disorders
Abdominal pain upper
11.1%
1/9 • Through Day 169
0.00%
0/11 • Through Day 169
0.00%
0/17 • Through Day 169
0.00%
0/21 • Through Day 169
Gastrointestinal disorders
Diarrhoea
11.1%
1/9 • Through Day 169
0.00%
0/11 • Through Day 169
0.00%
0/17 • Through Day 169
0.00%
0/21 • Through Day 169
Gastrointestinal disorders
Toothache
0.00%
0/9 • Through Day 169
0.00%
0/11 • Through Day 169
5.9%
1/17 • Through Day 169
0.00%
0/21 • Through Day 169

Additional Information

Chief Operating Officer

Arrowhead Pharmaceuticals, Inc.

Phone: 626-304-3400

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place