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

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

Detailed Description
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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.

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

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: ANX005


Participation Criteria
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Eligibility Criteria
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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 agree to use highly effective methods of contraception from Screening through Week 36. Males with a woman partner of childbearing potential must agree to use highly effective methods of contraception from Screening through Week 36. 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, ongoing or intercurrent illness, medical condition, or medical history that would jeopardize the safety of the participant, limit participation, or compromise the interpretation of the safety 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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ANX005<br>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<br>* IV Infusion<br>|

What is the study measuring?
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Primary Outcome Measures

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

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Serum Concentrations of ANX005 |  | Up to Week 36 | 
| Maximum Observed Serum Concentration (Cmax) of ANX005 |  | Day 1 and Week 24 | 
| Area Under the ANX005 Serum Concentration-Time Curve |  | Day 1 and Week 24 | 
| Change from Baseline in Free Complement (C) 1q Serum Concentrations |  | Baseline, up to Week 36 |