An Open Label Study of ANX005 in Subjects With, or at Risk for, Manifest Huntington's Disease

NCT04514367 | Completed Phase 2 FDA Drug

• 1 other identifier: ANX005-HD-01
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 6

Brief Summary

This study is a multi-center, open-label study of intravenous (IV) ANX005 in subjects with, or at risk for, manifest Huntington's Disease (HD).

Conditions

Huntington Disease

Outcome Measures

Primary Outcomes (3)

• Safety and tolerability of intravenous ANX005 administered for up to 22 weeks in subjects with, or at risk for, manifest Huntington's Disease

  As measured by incidence of TEAEs, SAEs, AEs related to ANX005, SAEs related to ANX005, Grade 3 or higher AEs, Grade 3 or higher AEs related to ANX005, AEs leading to study or treatment discontinuation.

  Up to Week 36

• Pharmacokinetics (PK) of ANX005

  As measured by ANX005 serum and cerebrospinal fluid concentrations

  Up to Week 36

• Pharmacodynamics (PD) effects of ANX005

  As measured by C1q, C4a, and NfL levels in blood and/or cerebrospinal fluid concentrations

  Up to Week 36

Other Outcomes (1)

• Exploratory effects of ANX005 on measures of efficacy

  Up to Week 36

Study Arms (1)

ANX005

EXPERIMENTAL

IV

Drug: ANX005

Interventions

ANX005 DRUG

Intravenous Infusion

ANX005

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of or at risk for Huntington's disease: Genetically confirmed disease by direct DNA testing, total CAG-Age Product (CAP) score \> 400 and UHDRS independence score ≥ 80.
• Able to walk independently and self-sufficient in basic activities of daily living (e.g. eating, dressing, bathing).
• All HD concomitant medications stable.
• 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.
• Males with a woman of childbearing potential partner must agree to use highly effective methods of contraception.
• Previously vaccinated against encapsulated bacterial pathogens (Neisseria meningitidis, Haemophilus influenzae, and Streptococcus pneumoniae) or willing to undergo vaccination.
• Able to tolerate EEG and lumbar puncture (LP) procedures.

You may not qualify if:

• Be at risk of suicide or self-harm within the preceding 12 months.
• Chorea and/or cognitive deficits severe enough to interfere with study assessments.
• Subjects with body weight \> 150 kg.
• 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 subject risk.
• Signs and symptoms of, or a diagnosis consistent with a chronic autoimmune disorder and/or an ANA titer ≥ 1:160.
• History of previous infusion reactions, sensitivities, allergic, or anaphylactic reactions to previous medications, environmental stimuli or other substances.
• Use of an experimental agent within 60 days or five half-lives prior to Screening or anytime over the duration of this study.
• Prior treatment with any monoclonal antibody.
• Presence of an implanted deep brain stimulation device.
• Any history of gene therapy, RNA or DNA targeted HD specific investigational agents such as antisense oligonucleotides, cell transplantation or any experimental brain surgery.
• Brain and spinal pathology that may interfere with cerebrospinal fluid homeostasis and circulation, increases intracranial pressure (implanted shunt or catheter), malformations or tumor.
• Contraindication to undergoing an LP.
• Hypersensitivity to any of the excipients in the ANX005 drug product.
• Clinically significant intercurrent illness, medical condition, or medical history (including neurological or mental illness, HIV, any active infection, including Hepatitis B or C) that would jeopardize the safety of the subject, limit participation, or compromise the interpretation of the data derived from the subject.
• Any known genetic deficiencies of the complement-cascade system.

Sponsors & Collaborators

• Annexon, Inc.: lead

Study Sites (6)

Annexon Investigational Site 02

Birmingham, Alabama, 35294, United States

Location

Annexon Investigational Site 03

Englewood, Colorado, 80113, United States

Location

Annexon Investigational Site 04

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

Location

Annexon Investigational Site 07

Durham, North Carolina, 27710, United States

Location

Annexon Investigational Site 06

Cincinnati, Ohio, 45221, United States

Location

Annexon Investigational Site 08

Kirkland, Washington, 98034, United States

Location

MeSH Terms

Conditions

Huntington Disease

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Chorea; Dyskinesias; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Study Officials

• Benjamin Hoehn, MD

  Annexon, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: ANX005 administered for up to 22 weeks

Study Record Dates

• First Submitted: July 27, 2020
• First Posted: August 14, 2020
• Study Start: August 17, 2020
• Primary Completion: January 28, 2022
• Study Completion: January 28, 2022
• Last Updated: January 31, 2023

Record last verified: 2023-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)