Study of ANX005 in Adults With Amyotrophic Lateral Sclerosis (ALS)

Phase 2a Safety, Tolerability, Pharmacokinetic (PK), and Pharmacodynamic (PD) Study of Intravenous ANX005 in Subjects With Amyotrophic Lateral Sclerosis (ALS)

This study is a multi-center, open-label study of intravenous (IV) ANX005 in participants with ALS.

Study Overview

• Status: Completed
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: ANX005

Detailed Description

In this Phase 2a, multi-center, open label, proof-of-biology study, ANX005 will be administered to participants with ALS.

The study consists of approximately 22 weeks of treatment and approximately 14 weeks of follow-up. All participants will be contacted by phone 6 months after study completion.

• Study Type: Interventional
• Enrollment (Actual): 17
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G 2R7
      • Annexon Investigational Site 10
  • Ontario
    • Toronto, Ontario, Canada, M4N 3M5
      • Annexon Investigational Site 09
  • Quebec
    • Montréal, Quebec, Canada, H2X 0A9
      • Annexon Investigational Site 07
    • Montréal, Quebec, Canada, H3A 2B4
      • Annexon Investigational Site 08
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85013
      • Annexon Investigational Site 04
  • California
    • Orange, California, United States, 9492868
      • Annexon Investigational Site 01
  • Florida
    • Gainesville, Florida, United States, 32608
      • Annexon Investigational Site 02
    • Tampa, Florida, United States, 33612
      • Annexon Investigational Site 03

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Key Inclusion Criteria:

• Diagnosis of ALS according to the World Federation of Neurology revised EI Escorial criteria.
• Onset of weakness within 3 years prior to Day 1 visit.
• Slow Vital Capacity ≥ 50% of predicted normal adjusted for sex, age, and height (from the sitting position).
• ALS Functional Rating Scale-Revised (ALSFRS-R) ≥ 30 at the Screening visit (Week -2).
• If female, must be postmenopausal, surgically sterilized, or childbearing potential must agree to use highly effective methods of contraception from Screening until 3 months after the last infusion with study medication.
• Males with a woman partner of childbearing potential must agree to use highly effective methods of contraception from Screening until Week until 3 months after the last infusion with study medication.
• Documented history of vaccinations within 5 years prior to Screening visit against encapsulated bacterial pathogens or willing to undergo vaccinations.

Key Exclusion Criteria:

• Clinically significant intercurrent illness, medical condition, or medical history that would jeopardize the safety of the participant, limit participation, or compromise the interpretation of the data derived from the participant.
• Participants with body weight > 150 kilograms.
• Antinuclear antibodies (ANA) titer ≥ 1:160 (for either of the 2 ANA results a minimum of 2 weeks apart) during the Screening Period.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ANX005; Participants will receive induction dosing of ANX005 on Days 1 and 5 or 6, followed by maintenance doses of ANX005 every 2 weeks up to Week 22.	Drug: ANX005; IV Infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Participants Who Experienced Treatment-Emergent Adverse Events	Up to Week 36

Secondary Outcome Measures

Outcome Measure	Time Frame
Serum Concentrations of ANX005	Up to Week 36
Change from Baseline in Free Complement (C) 1q Serum Concentrations	Baseline, up to Week 36
Maximum Observed Serum Concentration (Cmax) of ANX005	Up to Week 36
Area Under the ANX005 Serum Concentration-Time Curve	Day 1 and Week 22

Collaborators and Investigators

• Sponsor: Annexon, Inc.
• Investigators: Study Director: Benjamin Hoehn, MD, PhD, Annexon, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): January 15, 2021
• Primary Completion (Actual): January 10, 2024
• Study Completion (Actual): April 23, 2024

Study Registration Dates

• First Submitted: September 23, 2020
• First Submitted That Met QC Criteria: September 24, 2020
• First Posted (Actual): September 29, 2020

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 2, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Pathologic Processes; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: ANX005-ALS-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No