Can a targeted antibody calm the immune attack in Huntington's disease?
NCT ID NCT04514367
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.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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28 people
The number who actually took part.
- Started
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Sep 2020
- Finished
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Aug 2022
- Lead sponsor
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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
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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
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Annexon Investigational Site 02
Birmingham, Alabama, 35294, United States
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Annexon Investigational Site 03
Englewood, Colorado, 80113, United States
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Annexon Investigational Site 04
Washington D.C., District of Columbia, 20057, United States
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Annexon Investigational Site 06
Cincinnati, Ohio, 45221, United States
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Annexon Investigational Site 07
Durham, North Carolina, 27710, United States
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Annexon Investigational Site 08
Kirkland, Washington, 98034, United States
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