Trial Outcomes & Findings for An Open Label Study of ANX005 in Participants With, or at Risk for, Manifest Huntington's Disease (NCT NCT04514367)
NCT ID: NCT04514367
Last Updated: 2026-08-25
Results Overview
An adverse event (AE) was defined as any untoward medical occurrence that developed or worsened in severity during the conduct of a clinical study and does not necessarily have a causal relationship to study drug. Serious AEs (SAEs) included death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was any AE with an onset on or after the day of infusion through Week 36 (end of study). AEs were graded per National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE): Grade: 1=Mild, 2=Moderate, 3=Severe or medically significant but not immediately life-threatening. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.
COMPLETED
PHASE2
28 participants
From first dose of study drug up to end of study (Week 36)
2026-08-25
Participant Flow
Participant milestones
| Measure |
ANX005
Participants received induction dosing of ANX005 administered by intravenous (IV) infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Overall Study
STARTED
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28
|
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Overall Study
Received at Least 1 Dose of Study Drug
|
28
|
|
Overall Study
Per Protocol Set
|
23
|
|
Overall Study
Pharmacokinetic (PK) Analysis Set
|
28
|
|
Overall Study
COMPLETED
|
25
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Overall Study
NOT COMPLETED
|
3
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Reasons for withdrawal
| Measure |
ANX005
Participants received induction dosing of ANX005 administered by intravenous (IV) infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Overall Study
Withdrawal by Subject
|
1
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Overall Study
Adverse Event
|
2
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Baseline Characteristics
Per protocol set
Baseline characteristics by cohort
| Measure |
ANX005
n=28 Participants
Participants received induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Age, Continuous
|
49.7 years
STANDARD_DEVIATION 12.51 • n=28 Participants
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Sex: Female, Male
Female
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12 Participants
n=28 Participants
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Sex: Female, Male
Male
|
16 Participants
n=28 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=28 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
27 Participants
n=28 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=28 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=28 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=28 Participants
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|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=28 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=28 Participants
|
|
Race (NIH/OMB)
White
|
28 Participants
n=28 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=28 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=28 Participants
|
|
Complement C4a in Cerebrospinal Fluid (CSF)
|
15.16 micrograms (µg)/liter (L)
STANDARD_DEVIATION 6.092 • n=23 Participants • Per protocol set
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|
CSF Neurofilament Light Chain (NfL) Level
|
3236.1 nanograms (ng)/L
STANDARD_DEVIATION 816.74 • n=23 Participants • Per protocol set
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Blood NfL Level
|
39.51 ng/L
STANDARD_DEVIATION 11.661 • n=23 Participants • Per protocol set
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|
Blood C1q
|
85.589 µg/mL
STANDARD_DEVIATION 14.0165 • n=28 Participants
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CSF C1q
|
0.2297 µg/mL
STANDARD_DEVIATION 0.06239 • n=28 Participants
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PRIMARY outcome
Timeframe: From first dose of study drug up to end of study (Week 36)Population: Safety population included all enrolled participants who received at least 1 dose of study drug.
An adverse event (AE) was defined as any untoward medical occurrence that developed or worsened in severity during the conduct of a clinical study and does not necessarily have a causal relationship to study drug. Serious AEs (SAEs) included death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was any AE with an onset on or after the day of infusion through Week 36 (end of study). AEs were graded per National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE): Grade: 1=Mild, 2=Moderate, 3=Severe or medically significant but not immediately life-threatening. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.
Outcome measures
| Measure |
ANX005
n=28 Participants
Participants received induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Any TEAEs
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28 Participants
|
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
SAEs
|
2 Participants
|
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
AEs related to ANX005
|
28 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
SAEs related to ANX005
|
2 Participants
|
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Grade ≥3 AEs
|
12 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Grade ≥3 AEs related to ANX005
|
11 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
AEs leading to study discontinuation
|
2 Participants
|
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
AEs leading to discontinuation of study medications
|
4 Participants
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PRIMARY outcome
Timeframe: Day 1Population: Safety population included all enrolled participants who received at least 1 dose of study drug.
Outcome measures
| Measure |
ANX005
n=28 Participants
Participants received induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Actual Dose of ANX005 Administered on Day 1
|
75.0624 milligrams (mg)/kilogram (kg)
Standard Deviation 2.6694
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PRIMARY outcome
Timeframe: Week 22Population: Safety population included all enrolled participants who received at least 1 dose of study drug. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Outcome measures
| Measure |
ANX005
n=23 Participants
Participants received induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Actual Dose of ANX005 Administered on Week 22
|
102.6609 mg/kg
Standard Deviation 2.7408
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PRIMARY outcome
Timeframe: Pre-dose up to 4 hours post-dose on Day 1Population: PK analysis set included all participants who received any amount of ANX005 and had evaluable ANX005 concentration data.
Outcome measures
| Measure |
ANX005
n=28 Participants
Participants received induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Area Under the Concentration Versus Time Curve From Time 0 to t (AUC0-t) of ANX005 at Day 1
|
4290 days*µg/milliliter (mL)
Standard Deviation 818
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PRIMARY outcome
Timeframe: Predose at Baseline (Day 1)Population: PK analysis set included all participants who received any amount of ANX005 and had evaluable ANX005 concentration data.
Outcome measures
| Measure |
ANX005
n=28 Participants
Participants received induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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CSF Free Complement Component 1q (C1q) at Day 1
|
0.2297 µg/mL
Standard Deviation 0.06239
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PRIMARY outcome
Timeframe: Predose at Baseline (Day 1)Population: PK analysis set included all participants who received any amount of ANX005 and had evaluable ANX005 concentration data.
Outcome measures
| Measure |
ANX005
n=28 Participants
Participants received induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Blood Free C1q at Day 1
|
85.589 µg/mL
Standard Deviation 14.0165
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PRIMARY outcome
Timeframe: Baseline, Week 24Population: Per protocol set included all participants who completed all 13 infusions of ANX005 and did not have major protocol violations. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Outcome measures
| Measure |
ANX005
n=22 Participants
Participants received induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Change From Baseline in Complement C4a in CSF at Week 24
|
38.18 µg/L
Standard Deviation 14.423
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PRIMARY outcome
Timeframe: Baseline, Week 36Population: Per protocol set included all participants who completed all 13 infusions of ANX005 and did not have major protocol violations. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Outcome measures
| Measure |
ANX005
n=20 Participants
Participants received induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Change From Baseline in Complement C4a in CSF at Week 36
|
23.28 µg/L
Standard Deviation 9.570
|
PRIMARY outcome
Timeframe: Baseline, Week 24Population: Per protocol set included all participants who completed all 13 infusions of ANX005 and did not have major protocol violations. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Outcome measures
| Measure |
ANX005
n=22 Participants
Participants received induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Change From Baseline in CSF NfL Level at Week 24
|
318.0 ng/L
Standard Deviation 618.66
|
PRIMARY outcome
Timeframe: Baseline, Week 36Population: Per protocol set included all participants who completed all 13 infusions of ANX005 and did not have major protocol violations. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Outcome measures
| Measure |
ANX005
n=20 Participants
Participants received induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Change From Baseline in CSF NfL Level at Week 36
|
508.6 ng/L
Standard Deviation 919.43
|
PRIMARY outcome
Timeframe: Baseline, Week 24Population: Per protocol set included all participants who completed all 13 infusions of ANX005 and did not have major protocol violations.
Outcome measures
| Measure |
ANX005
n=23 Participants
Participants received induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Change From Baseline in Blood NfL Level at Week 24
|
-6.53 ng/L
Standard Deviation 10.112
|
PRIMARY outcome
Timeframe: Baseline, Week 36Population: Per protocol set included all participants who completed all 13 infusions of ANX005 and did not have major protocol violations. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.
Outcome measures
| Measure |
ANX005
n=22 Participants
Participants received induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Change From Baseline in Blood NfL Level at Week 36
|
-2.20 ng/L
Standard Deviation 13.146
|
Adverse Events
ANX005
Serious adverse events
| Measure |
ANX005
n=28 participants at risk
Participants received induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Musculoskeletal and connective tissue disorders
Systemic lupus erythematosus
|
3.6%
1/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
3.6%
1/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
Other adverse events
| Measure |
ANX005
n=28 participants at risk
Participants received induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks (Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, and 22) with follow up visits on Weeks 24, 28, and 36.
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|---|---|
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Skin and subcutaneous tissue disorders
Rash
|
53.6%
15/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
35.7%
10/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
28.6%
8/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Rash macular
|
10.7%
3/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Skin and subcutaneous tissue disorders
Rash pruritic
|
10.7%
3/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Nervous system disorders
Dizziness
|
21.4%
6/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Nervous system disorders
Headache
|
17.9%
5/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Nervous system disorders
Dizziness postural
|
7.1%
2/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Nervous system disorders
Lethargy
|
7.1%
2/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Nervous system disorders
Syncope
|
7.1%
2/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Nausea
|
17.9%
5/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Vomiting
|
14.3%
4/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Oral pain
|
7.1%
2/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Infections and infestations
COVID-19
|
14.3%
4/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Infections and infestations
Urinary tract infection
|
10.7%
3/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Investigations
Blood chloride increased
|
7.1%
2/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Investigations
White blood cell count decreased
|
7.1%
2/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
General disorders
Fatigue
|
10.7%
3/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Injury, poisoning and procedural complications
Fall
|
10.7%
3/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Injury, poisoning and procedural complications
Puncture site pain
|
10.7%
3/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Psychiatric disorders
Irritability
|
10.7%
3/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Cardiac disorders
Tachycardia
|
14.3%
4/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
7.1%
2/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.1%
2/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
7.1%
2/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
7.1%
2/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Vascular disorders
Flushing
|
10.7%
3/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Vascular disorders
Hypotension
|
7.1%
2/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
|
Vascular disorders
Orthostatic hypotension
|
7.1%
2/28 • From first dose of study drug up to end of study (Week 36)
Safety population included all enrolled participants who received at least 1 dose of study drug.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Written approval from the Sponsor is required before disclosing any information relative to this clinical study, and no publications initiated by Investigators may be published until all protocol-defined results are published in a manuscript. The details and processes of producing and reviewing reports, manuscripts, and presentations based on the data from this study are described in the clinical study agreement between the Sponsor and the institution of the Investigator.
- Publication restrictions are in place
Restriction type: OTHER