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

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04569435
• Other study ID #: ANX005-ALS-01
• Status: Recruiting
• Phase: Phase 2
• Start date: January 15, 2021
• Est. completion date: January 2024

Study information

• Verified date: March 2023
• Source: Annexon, Inc.
• Contact: Study Coordinator
• Phone: 650-822-5500
• Email: clinicaltrials[@]annexonbio.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 24
• Est. completion date: January 2024
• Est. primary completion date: October 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• ANX005
IV Infusion

Locations

Country	Name	City	State
Canada	Annexon Investigational Site 10	Edmonton	Alberta
Canada	Annexon Investigational Site 11	Fredericton	New Brunswick
Canada	Annexon Investigational Site 07	Montréal	Quebec
Canada	Annexon Investigational Site 08	Montréal	Quebec
Canada	Annexon Investigational Site 09	Toronto	Ontario
United States	Annexon Investigational Site 02	Gainesville	Florida
United States	Annexon Investigational Site 06	New York	New York
United States	Annexon Investigational Site 01	Orange	California
United States	Annexon Investigational Site 04	Phoenix	Arizona
United States	Annexon Investigational Site 05	San Francisco	California
United States	Annexon Investigational Site 03	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Annexon, Inc.

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants Who Experienced Treatment-Emergent Adverse Events		Up to Week 36
Secondary	Serum Concentrations of ANX005		Up to Week 36
Secondary	Maximum Observed Serum Concentration (Cmax) of ANX005		Day 1 and Week 24
Secondary	Area Under the ANX005 Serum Concentration-Time Curve		Day 1 and Week 24
Secondary	Change from Baseline in Free Complement (C) 1q Serum Concentrations		Baseline, up to Week 36