Clinical trial NCT04569435: Study of ANX005 in Adults With Amyotrophic Lateral Sclerosis (ALS)
Official title: Phase 2a Safety, Tolerability, Pharmacokinetic (PK), and Pharmacodynamic (PD) Study of Intravenous ANX005 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Annexon, Inc.
Sponsor class: INDUSTRY
First posted: 2020-09-29
Enrollment: 17
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants Who Experienced Treatment-Emergent Adverse Events
timeFrame: Up to Week 36
Secondary outcomes:
measure: Serum Concentrations of ANX005
timeFrame: Up to Week 36
measure: Maximum Observed Serum Concentration (Cmax) of ANX005
timeFrame: Up to Week 36
measure: Area Under the ANX005 Serum Concentration-Time Curve
timeFrame: Day 1 and Week 22
measure: Change from Baseline in Free Complement (C) 1q Serum Concentrations
timeFrame: Baseline, up to Week 36
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Annexon Investigational Site 04
state: Arizona
city: Orange
country: United States
facility: Annexon Investigational Site 01
state: California
city: Gainesville
country: United States
facility: Annexon Investigational Site 02
state: Florida
city: Tampa
country: United States
facility: Annexon Investigational Site 03
state: Florida
city: Edmonton
country: Canada
facility: Annexon Investigational Site 10
state: Alberta
city: Toronto
country: Canada
facility: Annexon Investigational Site 09
state: Ontario
city: Montréal
country: Canada
facility: Annexon Investigational Site 07
state: Quebec
city: Montréal
country: Canada
facility: Annexon Investigational Site 08
state: Quebec
Study officials:
affiliation: Annexon, Inc.
name: Benjamin Hoehn, MD, PhD
role: STUDY_DIRECTOR
References: