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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04514367
Recruitment Status : Recruiting
First Posted : August 14, 2020
Last Update Posted : May 11, 2021
Sponsor:
Information provided by (Responsible Party):
Annexon, Inc.

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).

Condition or disease Intervention/treatment Phase
Huntington Disease Drug: ANX005 Phase 2

Detailed Description:

The objective of this study is to evaluate the effects of intravenous ANX005 administered for up to 22 weeks in subjects with, or at risk for, manifest Huntington's Disease.

Subjects will receive induction dosing of ANX005 administered by IV infusion on Days 1 and 5 or 6, followed by maintenance dosing every 2 weeks through Week 22, with follow up visits on Weeks 24, 28, and 36.

All subjects will be contacted (in clinic visit or phone call) 6 months after study completion.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: ANX005 administered for up to 22 weeks
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2a Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous ANX005 in Subjects With, or at Risk for, Manifest Huntington's Disease
Actual Study Start Date : August 17, 2020
Estimated Primary Completion Date : March 2022
Estimated Study Completion Date : June 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: ANX005
IV
Drug: ANX005
Intravenous Infusion




Primary Outcome Measures :
  1. Incidence of treatment emergent adverse events (Safety and Tolerability) of ANX005 [ Time Frame: Up to Week 36 ]
    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.


Secondary Outcome Measures :
  1. Pharmacokinetics (PK) of ANX005 [ Time Frame: Up to Week 36 ]
    As measured by ANX005 serum and cerebrospinal fluid concentrations

  2. Pharmacodynamics (PD) effects of ANX005 [ Time Frame: Up to Week 36 ]
    As measured by C1q, C4a, and NfL blood and/or cerebrospinal fluid concentrations



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. 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.
  2. Able to walk independently and self-sufficient in basic activities of daily living (e.g. 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 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. Subjects with body weight > 150 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 subject risk.
  5. Signs and symptoms of, or a diagnosis consistent with a chronic autoimmune disorder and/or an 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, 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, 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.
  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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04514367


Contacts
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Contact: Study Coordinator 1-650-822-5500 clinicaltrials@annexonbio.com

Locations
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United States, Alabama
Annexon Investigational Site 02 Recruiting
Birmingham, Alabama, United States, 35294
Contact: Study Coordinator    650-822-5500    clinicaltrials@annexonbio.com   
United States, California
Annexon Investigational Site 05 Not yet recruiting
Sacramento, California, United States, 95816
Contact: Study Coordinator    650-822-5500    clinicaltrials@annexonbio.com   
United States, Colorado
Annexon Investigational Site 03 Recruiting
Englewood, Colorado, United States, 80113
Contact: Study Coordinator    650-822-5500    clinicaltrials@annexonbio.com   
United States, District of Columbia
Annexon Investigational Site 04 Recruiting
Washington, District of Columbia, United States, 20057
Contact: Study Coordinator    650-822-5500    clinicaltrials@annexonbio.com   
United States, North Carolina
Annexon Investigational Site 07 Recruiting
Durham, North Carolina, United States, 27710
Contact: Study Coordinator    650-822-5500    clinicaltrials@annexonbio.com   
United States, Ohio
Annexon Investigational Site 06 Not yet recruiting
Cincinnati, Ohio, United States, 45221
Contact: Study Coordinator    650-822-5500    clinicaltrials@annexonbio.com   
United States, Washington
Annexon Investigational Site 08 Recruiting
Kirkland, Washington, United States, 98034
Contact: Study Coordinator    650-822-5500    clinicaltrials@annexonbio.com   
Annexon Investigational Site 01 Recruiting
Spokane, Washington, United States, 99202
Contact: Study Coordinator    650-822-5500    clinicaltrials@annexonbio.com   
Sponsors and Collaborators
Annexon, Inc.
Investigators
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Principal Investigator: Rajeev Kumar, MD Rocky Mountain Movement Disorders Center, Englewood, CO
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Responsible Party: Annexon, Inc.
ClinicalTrials.gov Identifier: NCT04514367    
Other Study ID Numbers: ANX005-HD-01
First Posted: August 14, 2020    Key Record Dates
Last Update Posted: May 11, 2021
Last Verified: May 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Huntington Disease
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
Cognition Disorders
Neurocognitive Disorders
Mental Disorders