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

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
Sponsor:
Information provided by (Responsible Party):
Annexon, Inc.

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

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: ANX005 Phase 2

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.

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Study Type : Interventional
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.
Drug: ANX005
IV Infusion




Primary Outcome Measures :
  1. Number of Participants Who Experienced Treatment-Emergent Adverse Events [ Time Frame: Up to Week 36 ]

Secondary Outcome Measures :
  1. Serum Concentrations of ANX005 [ Time Frame: Up to Week 36 ]
  2. Maximum Observed Serum Concentration (Cmax) of ANX005 [ Time Frame: Day 1 and Week 24 ]
  3. Area Under the ANX005 Serum Concentration-Time Curve [ Time Frame: Day 1 and Week 24 ]
  4. Change from Baseline in Free Complement (C) 1q Serum Concentrations [ Time Frame: Baseline, up to Week 36 ]


Information from the National Library of Medicine

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.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 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.

Information from the National Library of Medicine

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


Contacts
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Contact: Study Coordinator 650-822-5500 clinicaltrials@annexonbio.com

Locations
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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   
Sponsors and Collaborators
Annexon, Inc.
Investigators
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Study Director: Benjamin Hoehn, MD, PhD Annexon, Inc.
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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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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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