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

Phase 2

Completed

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: ANX005

• Registration Number: NCT04569435
• Lead Sponsor: Annexon, Inc.

Brief Summary

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

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 Timeline

• Study First Submitted: 2020-09-23
• Study First Submitted QC: 2020-09-24
• Study First Posted: 2020-09-29
• Study Start: 2021-01-15
• Primary Completion: 2024-01-10
• Study Completion: 2024-04-23
• Disposition First Posted: 2024-08-28
• Status Verified: 2025-01
• Disposition First Submitted: 2025-01-02
• Last Update Submitted: 2025-01-02
• Last Update Posted: 2025-01-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 17

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ANX005	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.

Primary Outcome Measures

Name	Time	Method
Number of Participants Who Experienced Treatment-Emergent Adverse Events	Up to Week 36

Secondary Outcome Measures

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

Trial Locations

Locations (8)

Annexon Investigational Site 04; 🇺🇸 Phoenix, Arizona, United States

Annexon Investigational Site 01; 🇺🇸 Orange, California, United States

Annexon Investigational Site 02; 🇺🇸 Gainesville, Florida, United States

Annexon Investigational Site 03; 🇺🇸 Tampa, Florida, United States

Annexon Investigational Site 10; 🇨🇦 Edmonton, Alberta, Canada

Annexon Investigational Site 09; 🇨🇦 Toronto, Ontario, Canada

Annexon Investigational Site 07; 🇨🇦 Montréal, Quebec, Canada

Annexon Investigational Site 08; 🇨🇦 Montréal, Quebec, Canada