Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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 : October 7, 2021
Sponsor:
Information provided by (Responsible Party):
Annexon, Inc.

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
This study is a multi-center, open-label study of intravenous (IV) ANX005 in subjects with Amyotrophic Lateral Sclerosis ((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 of ANX005 will be administered to up to approximately 24 subjects with Amyotrophic Lateral Sclerosis (ALS) for up to 12 weeks.

Induction Dose: Each subject will receive induction dosing of ANX005 on Days 1 and 5 or 6 (5/6).

Maintenance Dose: Each subject will receive a maintenance dose of ANX005 every 2 weeks (Weeks 2, 4, 6, 8, and 10). Maintenance doses are expected to be completed over approximately 4-5 hours.

Follow-up visits will be on Weeks 12, 16, and 24. All subjects will be contacted by phone 6 months after study completion.

Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: ANX005 administered for up to 12 weeks
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 2022
Estimated Study Completion Date : May 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
Experimental: ANX005
IV
 Drug: ANX005
Intravenous Infusion


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Incidence of treatment emergent adverse events (Safety and Tolerability) of ANX005 [ Time Frame: Up to week 12 ]
    As measured by incidence of TEAEs, SAEs, AEs related to ANX005, SAEs related to ANX005, Grade 3 or higher AEs, Grade 3 or higher AEs related to ANX005, AEs leading to study or treatment discontinuation.


Secondary Outcome Measures :
  1. Pharmacokinetics (PK) of ANX005 [ Time Frame: Up to Week 24 ]
    Concentrations of ANX005 in serum over time (up to Week 24)

  2. Pharmacokinetics (PK) of ANX005 [ Time Frame: Day 1 and Week 10 ]
    ANX005 Day 1 and Week 10 PK parameters (eg, Cmax)

  3. Pharmacokinetics (PK) of ANX005 [ Time Frame: Day 1 and Week 10 ]
    ANX005 Day 1 and Week 10 PK parameters (eg, AUC0-t)


Other Outcome Measures:
  1. Pharmacodynamics (PD) effects of ANX005 [ Time Frame: up to Week 24 ]
    Change in free C1q concentrations in serum over time (up to Week 24)


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Diagnosis of ALS according to the World Federation of Neurology revised E1 Escorial criteria.
  2. Onset of weakness within 3 years prior to enrollment.
  3. Slow Vital Capacity ≥ 60% of predicted normal adjusted for sex, age, and height (from the sitting position).
  4. ALSFRS-R ≥ 30 at the Screening Visit.
  5. If female, must be postmenopausal, surgically sterilized, or agree to use highly effective methods of contraception from screening through Week 24.
  6. Males with a woman partner of childbearing potential must agree to use highly effective methods of contraception from Screening through Week 24.
  7. Documented history of vaccinations within 5 years prior to screening visit against encapsulated bacterial pathogens.

Exclusion Criteria:

  1. Clinically significant, ongoing illness or medical condition that would jeopardize the safety of the subject, limit participation, or compromise the interpretation of the safety data derived from the subject.
  2. Subjects with body weight > 150 kg.
  3. An ANA titer ≥ 1:160.
  4. Clinically significant intercurrent illness, medical condition, or medical history that would jeopardize the safety of the subject, limit participation, or compromise the interpretation of the data derived from the subject.
Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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
Layout table for location contacts
Contact: Study Coordinator 650-822-5500 clinicaltrials@annexonbio.com

Locations
Layout table for location information
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
Layout table for investigator information
Study Director: Lahar Mehta, MD Annexon, Inc.
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
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: October 7, 2021
Last Verified: October 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
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