A Single and Multiple Ascending Dose Study of ANX009 in Normal Healthy Volunteers (NHV)

NCT04535752 | Completed Phase 1

• 1 other identifier: ANX009-NHV-01
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the safety, tolerability, pharmakokinetics and pharmacodynamics of single and repeated doses of ANX009

Conditions

Safety and Tolerability in Healthy Volunteers

Outcome Measures

Primary Outcomes (1)

• Safety: Number of Participants Who Experienced Treatment-Emergent Adverse Events

  Incidence and severity of treatment-emergent adverse events (AEs). AEs will be coded using MedDRA and severity of AEs will be graded using National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI CTCAE).

  [Time Frame: Up to Day 29 for SAD; up to Day 43 for MAD]

Secondary Outcomes (6)

• Pharmacodynamics: Total Amount of Complement Protein in Blood (CH50)

  Up to Week 6

• Pharmacodynamics: Amount of C1 in Blood (C1q)

  Up to Week 6

• Pharmacokinetic: Maximum Observed Serum Concentration (Cmax) of ANX009

  Pre-dose, immediately after dose, and 0.5, 1, 2, 4, 6, 8, 12, 24 hours post-dose on Day 1 (SAD and MAD) and 36, 48, and 72 hours post-dose Day 1 (SAD)

• Pharmacokinetic: Time to Maximum Observed Serum Concentration (Tmax) of ANX009

  Pre-dose, immediately after dose, and 0.5, 1, 2, 4, 6, 8, 12, 24 hours post-dose on Day 1 (SAD and MAD) and 36, 48, and 72 hours post-dose Day 1 (SAD)

• Pharmacokinetic: Area Under the ANX009 Serum Concentration-Time Curve to Last Sample (AUC 0-t) and extrapolated through infinity (AUC 0-inf)

  Pre-dose, immediately after dose, and 0.5, 1, 2, 4, 6, 8, 12, 24 hours post-dose on Day 1 (SAD and MAD) and 36, 48, and 72 hours post-dose Day 1 (SAD)

Study Arms (4)

ANX009, Single Ascending Doses

EXPERIMENTAL

Single dose of ANX009 with a 7-day follow-up before escalation to the next dose level.

Drug: ANX009

Placebo, Single Ascending Doses

PLACEBO COMPARATOR

Single doses of matching placebo

Drug: Placebo

ANX009, Multiple Ascending Doses

EXPERIMENTAL

ANX009 once daily on Days 1-14

Drug: ANX009

Placebo, Multiple doses

PLACEBO COMPARATOR

Matching placebo once daily on Days 1-14

Drug: Placebo

Interventions

ANX009 DRUG

Single or multiple ascending dose

Also known as: 009

ANX009, Multiple Ascending Doses; ANX009, Single Ascending Doses

Placebo DRUG

Single or multiple ascending dose

Also known as: matching placebo

Placebo, Multiple doses; Placebo, Single Ascending Doses

Eligibility Criteria

• Age: 18 Years - 59 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Healthy male and non-pregnant, non-lactating female volunteers ≥18 to 59 years of age.
• Females must be postmenopausal, surgically sterilized or willing and able to use highly effective methods of contraception from screening through the final study visit.
• Males with a partner of childbearing potential must agree to use contraception from Screening through the final study visit.
• Documented history within 5 years of screening of previous vaccination against encapsulated bacterial pathogens (MAD cohorts only).
• Complete the full sequence of protocol-related doses, procedures and evaluations.
• No alcohol and drugs of abuse at screening and baseline or through study completion.
• Discontinue use of nutritional supplements and prescription and over-the-counter medications (vitamins are allowed).
• No new tattoos/piercings or elective surgery from screening through the End of Study visit
• Ability to understand and provide written informed consent.

You may not qualify if:

• 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.
• Clinically significant findings on the screening or Baseline ECG or physical examination.
• Clinically significant abnormalities on screening or Baseline laboratory assessments.
• An ANA titer ≥ 1:160.
• History of any autoimmune disease.
• History of meningitis or septicemia.
• Clinically significant infection that required medical intervention (not including antibiotic prophylaxis) within 1 month prior to study drug dosing.
• Known genetic deficiencies of the complement cascade system or immunodeficiency.
• Treatment with an investigational therapeutic agent within 30 days prior to study drug dosing.
• Use of immunosuppressants or corticosteroids within 30 days prior to study drug dosing.
• Active alcohol abuse, drug abuse or substance abuse.
• Hypersensitivity to any of the excipients in the ANX009 drug product or active substance.
• History of previous sensitivities or allergic or anaphylactic reactions to previous medication injections.
• Positive for HIV Ab, Hepatitis C Ab or Hepatitis B surface antigen (HBsAg) at screening.

Sponsors & Collaborators

• Annexon, Inc.: lead
• Nucleus Network Ltd: collaborator

Study Sites (1)

Site 1

Melbourne, Australia

Location

Study Officials

• Eric Humphriss, MBA

  Annexon Director, Global Clinical Operations

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: BASIC SCIENCE
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 24, 2020
• First Posted: September 2, 2020
• Study Start: October 30, 2020
• Primary Completion: June 26, 2021
• Study Completion: June 26, 2021
• Last Updated: August 30, 2021

Record last verified: 2021-08

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)