Amyotrophic Lateral Sclerosis
Conditions
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.
Interventions
DRUGANX005
IV Infusion
Sponsors
Annexon, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants Who Experienced Treatment-Emergent Adverse Events | Up to Week 36 |
Secondary
| Measure | Time frame |
|---|---|
| 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 |
Countries
Canada, United States
Outcome results
None listed