A Phase 2a Safety, Tolerability, Pharmacokinetic (PK), and Pharmacodynamic (PD) Study of Intravenous ANX005 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of Intravenous ANX005 in Subjects with Amyotrophic Lateral Sclerosis (ALS).
Disease:Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALS
Study Type:Interventional Trial
Study Category:Drug Trial
Study Chair(s)/Principal Investigator(s):
Clinicaltrials.gov ID (11 digit #):NCT04569435
Coordinating Center Contact Information
Full Study Summary:
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 Sponsor:Annexon, Inc.
Estimated Study Start Date:04/15/2021
Estimated Study Completion Date:12/31/2021
Posting Last Modified Date:04/27/2021
Date Study Added to alsconsortium.org:04/27/2021
Time since Symptom Onset:<36 months
Time since Diagnosis:
Can participants use Riluzole?Yes
Subjects must meet all of the following criteria:
- Males or females aged 18 and above at the time of informed consent.
- Diagnosis of ALS according to the World Federation of Neurology revised E1 Escorial criteria.
- Onset of weakness within 3 years prior to enrollment.
- Slow Vital Capacity ≥ 60% of predicted normal adjusted for sex, age, and height (from the sitting position).
- Able to perform reproducible pulmonary function tests.
- ALS concomitant medications stable for at least 2 months prior to Screening.
- ALSFRS-R ≥ 30 at the Screening Visit.
- If female, must be postmenopausal, surgically sterilized, or agree to use highly effective methods of contraception from screening through Week 24.
- Males with a woman partner of childbearing potential must agree to use highly effective methods of contraception from Screening through Week 24.
- Documented history of vaccinations within 5 years prior to screening visit against encapsulated bacterial pathogens.
- Complete the full sequence of protocol related procedures and evaluations, including lumbar punctures for collection of cerebrospinal fluid.
- Ability to understand and provide written, informed consent.
Subjects must not meet any of the following criteria:
- 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.
- Subjects with body weight > 150 kg.
- Clinically significant findings on screening laboratory testing, physical examination or vital signs that are not specific to ALS that could interfere with the conduct of the study, the interpretation of the data or increase subject risk.
- An ANA titer ≥ 1:160.
- History of previous infusion reactions, sensitivities, allergic, or anaphylactic reactions to previous medications, environmental stimuli or other substances.
- Receipt of an experimental agent within 60 days prior to Screening or through Week 24.
- Prior treatment with any monoclonal antibody within 6 months of screening.
- Hypersensitivity to any of the excipients in the ANX005 drug product.
- 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.
- Any known genetic deficiencies of the complement-cascade system.
- History of chronic systemic steroid use or immunosuppressant medication ending less than 1 month prior to screening.
- Active alcohol, drug abuse or substance abuse.
- Females who are pregnant or unwilling to use highly effective methods of contraception.
- Contraindication to undergoing an LP.
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