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

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

Sponsors

Lead Sponsor: Annexon, Inc.

Source Annexon, Inc.
Brief Summary

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

Detailed Description

The objective of this study is to evaluate the effects of intravenous ANX005 administered for up to 10 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 10, with follow up visits on Weeks 12, 16 and 24.

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

Overall Status Recruiting
Start Date August 17, 2020
Completion Date December 2021
Primary Completion Date September 2021
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Incidence of treatment emergent adverse events (Safety and Tolerability) of ANX005 Up to Week 24
Secondary Outcome
Measure Time Frame
Pharmacokinetics (PK) of ANX005 Up to Week 24
Pharmacodynamics (PD) effects of ANX005 Up to Week 24
Enrollment 24
Condition
Intervention

Intervention Type: Drug

Intervention Name: ANX005

Description: Intravenous Infusion

Arm Group Label: ANX005

Eligibility

Criteria:

Inclusion Criteria:

1. Diagnosis of or at risk for Huntington's disease: 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 (for at least 2 years), 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. Hemoglobin, bilirubin, and LDH within normal limits.

8. Able to tolerate EEG and 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 medication infusions.

7. Receipt 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 CSF 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 steroid or immunosuppressant medication use

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Rajeev Kumar, MD Principal Investigator Rocky Mountain Movement Disorders Center, Englewood, CO
Overall Contact

Last Name: Maria Hernandez

Phone: 1-650-822-5523

Email: [email protected]

Location
Facility: Status: Contact:
Annexon Investigational Site 02 | Birmingham, Alabama, 35294, United States Not yet recruiting Maria Hernandez
Annexon Investigational Site 05 | Sacramento, California, 95816, United States Not yet recruiting Maria Hernandez
Annexon Investigational Site 03 | Englewood, Colorado, 80113, United States Recruiting Maria Hernandez
Annexon Investigational Site 04 | Washington, District of Columbia, 20057, United States Not yet recruiting Maria Hernandez
Annexon Investigational Site 07 | Durham, North Carolina, 27710, United States Not yet recruiting Maria Hernandez
Annexon Investigational Site 06 | Cincinnati, Ohio, 45221, United States Not yet recruiting Maria Hernandez
Annexon Investigational Site 08 | Kirkland, Washington, 98034, United States Recruiting Maria Hernandez
Annexon Investigational Site 01 | Spokane, Washington, 99202, United States Recruiting Maria Hernandez
Location Countries

United States

Verification Date

October 2020

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: ANX005

Type: Experimental

Description: IV

Patient Data No
Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Intervention Model Description: ANX005 administered for up to 10 weeks

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov