Can a targeted antibody calm the immune attack in Huntington's disease?

NCT ID NCT04514367

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 25, 2026 · Last updated Aug 26, 2026 · Updated 1 time

Summary

This phase 2 trial is testing an experimental drug called ANX005 in about 28 people who have Huntington's disease or are at risk of developing it. ANX005 is given as an intravenous infusion and works by blocking a complement protein, C1q, which is part of the immune system and may contribute to nerve damage in Huntington's. The study aims to assess the drug's safety, how it moves through the body, and whether it affects certain biomarkers linked to the disease.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ANX005, an experimental antibody given by intravenous infusion that targets the complement protein C1q
What this could lead to
If successful, this could point toward a new way to slow or alter the course of Huntington's disease, a currently incurable genetic condition.
What could go wrong
This is an early, small phase 2 trial focused mainly on safety and dosing, not proof of effectiveness. The drug may not show meaningful benefit, and side effects are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

28 people

The number who actually took part.

Started

Sep 2020

Finished

Aug 2022

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Diagnosis of or at risk for HD: Genetically confirmed disease by direct deoxyribonucleic acid (DNA) testing, total cytosine-adenine-guanine (CAG)-Age Product (CAP) score \> 400 and UHDRS independence score ≥ 80. 2. Able to walk independently and self-sufficient in basic activities of daily living (for example, eating, dressing, bathing). 3. All HD concomitant medications stable. 4. If female, must be postmenopausal (no menses for at least 2 years without an alternative medical cause), surgically sterilized (bilateral tubal ligation, bilateral oophorectomy, or hysterectomy), or agree to use highly effective methods of contraception. 5. Males with a woman of childbearing potential partner must agree to use highly effective methods of contraception. 6. Previously vaccinated against encapsulated bacterial pathogens (Neisseria meningitidis, Haemophilus influenzae, and Streptococcus pneumoniae) or willing to undergo vaccination. 7. Able to tolerate electroencephalogram (EEG) and lumbar puncture (LP) procedures. Exclusion Criteria: 1. Be at risk of suicide or self-harm within the preceding 12 months. 2. Chorea and/or cognitive deficits severe enough to interfere with study assessments. 3. Participants with body weight \> 150 kilograms (kg). 4. Clinically significant findings on the screening laboratory testing or physical examination that are not specific to HD and may interfere with the conduct of the study or the interpretation of the data or increase participant risk. 5. Signs and symptoms of, or a diagnosis consistent with a chronic autoimmune disorder and/or an antinuclear antibody (ANA) titer ≥ 1:160. 6. History of previous infusion reactions, sensitivities, allergic, or anaphylactic reactions to previous medications, environmental stimuli or other substances. 7. Use of an experimental agent within 60 days or five half-lives prior to Screening or anytime over the duration of this study. 8. Prior treatment with any monoclonal antibody. 9. Presence of an implanted deep brain stimulation device. 10. Any history of gene therapy, ribonucleic acid (RNA) or DNA targeted HD specific investigational agents such as antisense oligonucleotides, cell transplantation or any experimental brain surgery. 11. Brain and spinal pathology that may interfere with cerebrospinal fluid homeostasis and circulation, increases intracranial pressure (implanted shunt or catheter), malformations or tumor. 12. Contraindication to undergoing an LP. 13. Hypersensitivity to any of the excipients in the ANX005 drug product. 14. Clinically significant intercurrent illness, medical condition, or medical history (including neurological or mental illness, human immunodeficiency virus \[HIV\], any active infection, including Hepatitis B or C) that would jeopardize the safety of the participant, limit participation, or compromise the interpretation of the data derived from the participant. 15. Any known genetic deficiencies of the complement-cascade system. 16. History of chronic oral or intravenous steroid use or immunosuppressant medication use. 17. Hemoglobin, bilirubin, or lactate dehydrogenase (LDH) values that are outside normal limits and clinically significant or suggestive of hemolytic anemia.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Annexon Investigational Site 02

    Birmingham, Alabama, 35294, United States

  • Annexon Investigational Site 03

    Englewood, Colorado, 80113, United States

  • Annexon Investigational Site 04

    Washington D.C., District of Columbia, 20057, United States

  • Annexon Investigational Site 06

    Cincinnati, Ohio, 45221, United States

  • Annexon Investigational Site 07

    Durham, North Carolina, 27710, United States

  • Annexon Investigational Site 08

    Kirkland, Washington, 98034, United States

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