NCT04569435

Brief Summary

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

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
17

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jan 2021

Typical duration for phase_2

Geographic Reach
2 countries

8 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 23, 2020

Completed
6 days until next milestone

First Posted

Study publicly available on registry

September 29, 2020

Completed
4 months until next milestone

Study Start

First participant enrolled

January 15, 2021

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 10, 2024

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

April 23, 2024

Completed
Last Updated

January 6, 2025

Status Verified

January 1, 2025

Enrollment Period

3 years

First QC Date

September 23, 2020

Last Update Submit

January 2, 2025

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

  • Number of Participants Who Experienced Treatment-Emergent Adverse Events

    Up to Week 36

Secondary Outcomes (4)

  • Serum Concentrations of ANX005

    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

  • Change from Baseline in Free Complement (C) 1q Serum Concentrations

    Baseline, up to Week 36

Study Arms (1)

ANX005

EXPERIMENTAL

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

Interventions

ANX005DRUG

IV Infusion

ANX005

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

Annexon Investigational Site 04

Phoenix, Arizona, 85013, United States

Location

Annexon Investigational Site 01

Orange, California, 9492868, United States

Location

Annexon Investigational Site 02

Gainesville, Florida, 32608, United States

Location

Annexon Investigational Site 03

Tampa, Florida, 33612, United States

Location

Annexon Investigational Site 10

Edmonton, Alberta, T6G 2R7, Canada

Location

Annexon Investigational Site 09

Toronto, Ontario, M4N 3M5, Canada

Location

Annexon Investigational Site 07

Montreal, Quebec, H2X 0A9, Canada

Location

Annexon Investigational Site 08

Montreal, Quebec, H3A 2B4, Canada

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Benjamin Hoehn, MD, PhD

    Annexon, Inc.

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 23, 2020

First Posted

September 29, 2020

Study Start

January 15, 2021

Primary Completion

January 10, 2024

Study Completion

April 23, 2024

Last Updated

January 6, 2025

Record last verified: 2025-01

Data Sharing

IPD Sharing
Will not share

Locations

US(4)CA(4)