Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ANX005 in Participants With Warm Autoimmune Hemolytic Anemia (wAIHA)

NCT04691570 | Completed Phase 2 FDA Drug

• 1 other identifier: ANX005-wAIHA-02
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

This study will evaluate the safety and tolerability of ANX005 in participants with Warm Autoimmune Hemolytic Anemia (wAIHA).

Conditions

Warm Autoimmune Hemolytic Anemia (wAIHA)

Keywords

AIHA; C1q; complement; RBC lysis

Outcome Measures

Primary Outcomes (2)

• Safety: Treatment-emergent adverse events (TEAEs)

  Number of participants with TEAEs, defined as any adverse event with an onset on or after the day of infusion through 16 weeks after the infusion

  Up to Week 16

• Change in disease activity biomarkers

  Change in hemoglobin, lactate dehydrogenase, bilirubin, reticulocyte count and haptoglobin from baseline

  Baseline to Day 71

Secondary Outcomes (2)

• Plasma concentrations

  Up to Day 71

• Change in complement system biomarkers

  Baseline to Day 71

Study Arms (1)

ANX005

EXPERIMENTAL

Participants will receive two once-weekly doses of ANX005 at specific time points

Drug: ANX005

Interventions

ANX005 DRUG

ANX005 is provided as a solution for IV infusion

ANX005

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or non-pregnant, non-lactating female ≥18 years of age (no maximum age).
• Diagnosis of wAIHA at least 3 months prior to screening with a direct antiglobulin test (DAT) ≥1 positive for immunoglobulin G (IgG)±C3, or a diagnosis of mixed autoimmune hemolytic anemia (AIHA) that is DAT positive for both IgG and C3, with a presence of a cold antibody with a thermal amplitude ≥30ºCelcius.
• Hemoglobin (Hgb) level ≤10.0 grams/deciliter (pre-transfusion).
• Evidence of classical complement pathway activation.
• Evidence of active hemolysis.
• Stable use of glucocorticoids and immunosuppressants are permitted.
• Vaccinations against encapsulated bacterial organisms within 5 years prior to screening or participant must be willing to receive prophylaxis against infections with encapsulated bacteria via vaccination and/or the use of prophylactic antibiotics in accordance with local standards of practice and/or guidelines.

You may not qualify if:

• Elevated aspartate aminotransferase or alanine aminotransferase levels \>2.5 times the upper limit of normal.
• Platelet count \<30 X 10\^9/liter.
• History of cold agglutinin disease.
• History of solid organ, bone marrow, or stem cell transplantation.
• History of splenectomy within the 3 months prior to screening.
• Received rituximab or other anti-CD20 monoclonal antibody \<3 months prior to screening.
• Intravenous immunoglobulin (IVIg) treatment within 3 months prior to screening or plasmapheresis or immunoadsorption treatment within 60 days prior to screening.
• Clinically significant, recent, or ongoing illness or medical condition, including coexistent autoimmune disorder, malignancy, HIV, hepatitis B virus, and hepatitis C virus.
• History of meningitis or septicemia within the past 2 years.
• Treatment with an investigational therapeutic agent within 30 days prior to screening.
• Hypersensitivity to any drug product or excipients used in this study or to previous IV medication administration.
• Body weight less than 50 kilograms (kg) or greater than 100 kg

Sponsors & Collaborators

• Annexon, Inc.: lead

Study Sites (1)

Investigational Site 01

Rochester, Minnesota, 55905, United States

Location

Study Officials

• Study Director

  Annexon, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 28, 2020
• First Posted: December 31, 2020
• Study Start: November 10, 2021
• Primary Completion: January 17, 2023
• Study Completion: January 17, 2023
• Last Updated: January 19, 2024

Record last verified: 2024-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)