Trial Outcomes & Findings for Study of ARC-520 in Participants With Hepatitis B Virus e Antigen (HBeAg) Positive Chronic Hepatitis B Virus (NCT NCT02452528)

Results Overview

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

Baseline, Day 85

Results posted on

2025-11-03

Participant Flow

Participant milestones

Participant milestones
Measure	ARC-520 Intravenous administration of 1.0 mg/kg ARC-520 once every 4 weeks for 3 total doses, plus entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg/day), taken throughout the study. Pretreatment with diphenhydramine 50 mg 2 hours (±30 minutes) prior to administration of study drug.	Placebo Intravenous administration of normal saline (0.9%) once every 4 weeks for 3 total doses, plus entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg/day), taken throughout the study. Pretreatment with diphenhydramine 50 mg 2 hours (±30 minutes) prior to administration of placebo.
Overall Study STARTED	2	2
Overall Study COMPLETED	1	1
Overall Study NOT COMPLETED	1	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of ARC-520 in Participants With Hepatitis B Virus e Antigen (HBeAg) Positive Chronic Hepatitis B Virus

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	ARC-520 n=2 Participants Intravenous administration of 1.0 mg/kg ARC-520 once every 4 weeks for 3 total doses, plus entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg/day), taken throughout the study. Pretreatment with diphenhydramine 50 mg 2 hours (±30 minutes) prior to administration of study drug.	Placebo n=2 Participants Intravenous administration of normal saline (0.9%) once every 4 weeks for 3 total doses, plus entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg/day), taken throughout the study. Pretreatment with diphenhydramine 50 mg 2 hours (±30 minutes) prior to administration of placebo.	Total n=4 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=3 Participants	0 Participants n=15 Participants	0 Participants n=18 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=3 Participants	0 Participants n=15 Participants	0 Participants n=18 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=3 Participants	2 Participants n=15 Participants	4 Participants n=18 Participants
Age, Categorical >=65 years	0 Participants n=3 Participants	0 Participants n=15 Participants	0 Participants n=18 Participants
Sex: Female, Male Female	0 Participants n=3 Participants	1 Participants n=15 Participants	1 Participants n=18 Participants
Sex: Female, Male Male	2 Participants n=3 Participants	1 Participants n=15 Participants	3 Participants n=18 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=3 Participants	0 Participants n=15 Participants	0 Participants n=18 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	2 Participants n=3 Participants	2 Participants n=15 Participants	4 Participants n=18 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=3 Participants	0 Participants n=15 Participants	0 Participants n=18 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=3 Participants	0 Participants n=15 Participants	0 Participants n=18 Participants
Race (NIH/OMB) Asian	1 Participants n=3 Participants	1 Participants n=15 Participants	2 Participants n=18 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=3 Participants	0 Participants n=15 Participants	0 Participants n=18 Participants
Race (NIH/OMB) Black or African American	1 Participants n=3 Participants	0 Participants n=15 Participants	1 Participants n=18 Participants
Race (NIH/OMB) White	0 Participants n=3 Participants	1 Participants n=15 Participants	1 Participants n=18 Participants
Race (NIH/OMB) More than one race	0 Participants n=3 Participants	0 Participants n=15 Participants	0 Participants n=18 Participants
Region of Enrollment United States	2 Participants n=3 Participants	2 Participants n=15 Participants	4 Participants n=18 Participants

PRIMARY outcome

Timeframe: Baseline, Day 85

Population: At the time of study termination only 2 ARC-520-treated and 2 placebo-treated participants had been enrolled. Due to the small sample size, analysis of this primary endpoint was not performed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From time of informed consent through Day 147 ± 3 days

Population: All participants who received at least 1 dose of ARC-520 or placebo

An AE was defined as any untoward medical occurrence in a participant which does not necessarily have to have a causal relationship with treatment. An SAE was defined as any untoward medical occurrence that, at any dose: 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. An AE was classified as a TEAE if the AE was not present prior to the first study medication administration and started at or after the time of initiation of administration of study medication, or if the AE presented prior to initiation of administration of study medication, continued and increased in intensity after administration of study medication.

Outcome measures

Outcome measures
Measure	Placebo n=2 Participants Intravenous administration of normal saline (0.9%) once every 4 weeks for 3 total doses, plus entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg/day), taken throughout the study. Pretreatment with diphenhydramine 50 mg 2 hours (±30 minutes) prior to administration of placebo.	ARC-520 n=2 Participants Intravenous administration of 1.0 mg/kg ARC-520 once every 4 weeks for 3 total doses, plus entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg/day), taken throughout the study. Pretreatment with diphenhydramine 50 mg 2 hours (±30 minutes) prior to administration of study drug.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and Discontinuations Due to Adverse Events (AEs) TEAEs	1 Participants	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and Discontinuations Due to Adverse Events (AEs) SAEs	0 Participants	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and Discontinuations Due to Adverse Events (AEs) Deaths	0 Participants	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Deaths, and Discontinuations Due to Adverse Events (AEs) Discontinuations due to AEs	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Days 1 and 57

Population: Pharmacokinetic evaluation was to be performed only on participants receiving ARC-520. At the time of study termination only 2 ARC-520-treated participants had been enrolled and therefore pharmacokinetic samples were not processed and no statistical analysis was performed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Days 1 and 57

Population: Pharmacokinetic evaluation was to be performed only on participants receiving ARC-520. At the time of study termination only 2 ARC-520-treated participants had been enrolled and therefore pharmacokinetic samples were not processed and no statistical analysis was performed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Days 1 and 57

Population: Pharmacokinetic evaluation was to be performed only on participants receiving ARC-520. At the time of study termination only 2 ARC-520 treated participants had been enrolled and therefore pharmacokinetic samples were not processed and no statistical analysis was performed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Days 1 and 57

Population: Pharmacokinetic evaluation was to be performed only on participants receiving ARC-520. At the time of study termination only 2 ARC-520 treated participants had been enrolled and therefore pharmacokinetic samples were not processed and no statistical analysis was performed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Days 1 and 57

Population: Pharmacokinetic evaluation was to be performed only on participants receiving ARC-520. At the time of study termination only 2 ARC-520 treated participants had been enrolled and therefore pharmacokinetic samples were not processed and no statistical analysis was performed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Days 1 and 57

Population: Pharmacokinetic evaluation was to be performed only on participants receiving ARC-520. At the time of study termination only 2 ARC-520 treated participants had been enrolled and therefore pharmacokinetic samples were not processed and no statistical analysis was performed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Days 1 and 57

Population: Pharmacokinetic evaluation was to be performed only on participants receiving ARC-520. At the time of study termination only 2 ARC-520 treated participants had been enrolled and therefore pharmacokinetic samples were not processed and no statistical analysis was performed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 48 hours post-dosing on Days 1 and 57

Population: Pharmacokinetic evaluation was to be performed only on participants receiving ARC-520. At the time of study termination only 2 ARC-520 treated participants had been enrolled and therefore pharmacokinetic samples were not processed and no statistical analysis was performed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 24 hours post-dosing on Days 1 and 57

Population: Pharmacokinetic evaluation was to be performed only on participants receiving ARC-520. At the time of study termination only 2 ARC-520 treated participants had been enrolled and therefore pharmacokinetic samples were not processed and no statistical analysis was performed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 24 hours post-dosing on Days 1 and 57

Population: Pharmacokinetic evaluation was to be performed only on participants receiving ARC-520. At the time of study termination only 2 ARC-520 treated participants had been enrolled and therefore pharmacokinetic samples were not processed and no statistical analysis was performed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 24 hours post-dosing on Days 1 and 57

Population: Pharmacokinetic evaluation was to be performed only on subjects receiving ARC-520. At the time of study termination only 2 ARC-520 treated subjects had been enrolled and therefore pharmacokinetic samples were not processed and no statistical analysis was performed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 24 hours post-dosing on Days 1 and 57

Population: Pharmacokinetic evaluation was to be performed only on participants receiving ARC-520. At the time of study termination only 2 ARC-520 treated participants had been enrolled and therefore pharmacokinetic samples were not processed and no statistical analysis was performed.

Outcome measures

Outcome data not reported

Adverse Events

ARC-520

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	ARC-520 n=2 participants at risk Intravenous administration of 1.0 mg/kg ARC-520 once every 4 weeks for 3 total doses, plus entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg/day), taken throughout the study. Pretreatment with diphenhydramine 50 mg 2 hours (±30 minutes) prior to administration of study drug.	Placebo n=2 participants at risk Intravenous administration of normal saline (0.9%) once every 4 weeks for 3 total doses, plus entecavir (0.5 or 1.0 mg/day) or tenofovir (300 mg/day), taken throughout the study. Pretreatment with diphenhydramine 50 mg 2 hours (±30 minutes) prior to administration of placebo.
General disorders Fatigue	0.00% 0/2 • From time of informed consent through Day 147 ± 3 days	50.0% 1/2 • Number of events 1 • From time of informed consent through Day 147 ± 3 days

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