Study of ANX005 in Adults With Amyotrophic Lateral Sclerosis (ALS)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04569435 |
Recruitment Status :
Recruiting
First Posted : September 29, 2020
Last Update Posted : July 7, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Amyotrophic Lateral Sclerosis | Drug: ANX005 | Phase 2 |
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.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 24 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 2a Safety, Tolerability, Pharmacokinetic (PK), and Pharmacodynamic (PD) Study of Intravenous ANX005 in Subjects With Amyotrophic Lateral Sclerosis (ALS) |
Actual Study Start Date : | January 15, 2021 |
Estimated Primary Completion Date : | November 30, 2022 |
Estimated Study Completion Date : | May 31, 2023 |

Arm | Intervention/treatment |
---|---|
Experimental: ANX005
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.
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Drug: ANX005
IV Infusion |
- Number of Participants Who Experienced Treatment-Emergent Adverse Events [ Time Frame: Up to Week 36 ]
- Serum Concentrations of ANX005 [ Time Frame: Up to Week 36 ]
- Maximum Observed Serum Concentration (Cmax) of ANX005 [ Time Frame: Day 1 and Week 24 ]
- Area Under the ANX005 Serum Concentration-Time Curve [ Time Frame: Day 1 and Week 24 ]
- Change from Baseline in Free Complement (C) 1q Serum Concentrations [ Time Frame: Baseline, up to Week 36 ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04569435
Contact: Study Coordinator | 650-822-5500 | clinicaltrials@annexonbio.com |
United States, Arizona | |
Annexon Investigational Site 04 | Recruiting |
Phoenix, Arizona, United States, 85013 | |
Contact: Study Coordinator 650-822-5500 clinicaltrials@annexonbio.com | |
United States, California | |
Annexon Investigational Site 01 | Recruiting |
Orange, California, United States, 9492868 | |
Contact: Study Coordinator 650-822-5500 clinicaltrials@annexonbio.com | |
Annexon Investigational Site 05 | Recruiting |
San Francisco, California, United States, 94109 | |
Contact: Study Coordinator 650-822-5500 clinicaltrials@annexonbio.com | |
United States, Florida | |
Annexon Investigational Site 02 | Recruiting |
Gainesville, Florida, United States, 32608 | |
Contact: Study Coordinator 650-822-5500 clinicaltrials@annexonbio.com | |
Annexon Investigational Site 03 | Recruiting |
Tampa, Florida, United States, 33612 | |
Contact: Study Coordinator 650-822-5500 clinicaltrials@annexonbio.com | |
United States, New York | |
Annexon Investigational Site 06 | Recruiting |
New York, New York, United States, 10021 | |
Contact: Study Coordinator 650-822-5500 clinicaltrials@annexonbio.com | |
Canada, Alberta | |
Annexon Investigational Site 10 | Recruiting |
Edmonton, Alberta, Canada, T6G 2R7 | |
Contact: Study Coordinator 6508225500 clinicaltrials@annexonbio.com | |
Canada, New Brunswick | |
Annexon Investigational Site 11 | Recruiting |
Fredericton, New Brunswick, Canada, N8 E3B 0C7 | |
Contact: Study Coordinator 6508225500 Clinicaltrials@annexonbio.com | |
Canada, Ontario | |
Annexon Investigational Site 09 | Recruiting |
Toronto, Ontario, Canada, M4N 3M5 | |
Contact: Study Coordinator 6508225500 clinicaltrials@annexonbio.com | |
Canada, Quebec | |
Annexon Investigational Site 07 | Recruiting |
Montréal, Quebec, Canada, H2X 0A9 | |
Contact: Study Coordinator 6508225500 clinicaltrials@annexonbio.com | |
Annexon Investigational Site 08 | Recruiting |
Montréal, Quebec, Canada, H3A 2B4 | |
Contact: Study Coordinator 6508225500 clinicaltrials@annexonbio.com |
Study Director: | Benjamin Hoehn, MD, PhD | Annexon, Inc. |
Responsible Party: | Annexon, Inc. |
ClinicalTrials.gov Identifier: | NCT04569435 |
Other Study ID Numbers: |
ANX005-ALS-01 |
First Posted: | September 29, 2020 Key Record Dates |
Last Update Posted: | July 7, 2022 |
Last Verified: | June 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Motor Neuron Disease Amyotrophic Lateral Sclerosis Sclerosis Pathologic Processes Neurodegenerative Diseases Nervous System Diseases |
Neuromuscular Diseases Spinal Cord Diseases Central Nervous System Diseases TDP-43 Proteinopathies Proteostasis Deficiencies Metabolic Diseases |