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Completed

NCT Number: NCT04691570

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

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

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Key information

Conditions

Warm Autoimmune Hemolytic Anemia (wAIHA)

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Investigational Site 03, Melbourne, Australia

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About this study

After being informed of study details and potential risks, all participants who provide written informed consent will undergo an up to 6-week screening period to determine eligibility. Participants who meet the eligibility criteria will receive two once-weekly intravenous (IV) infusions of ANX005. Participants will return to the clinic weekly through Week 10 for study assessments. The total duration of individual participation in this study will be up to 16 weeks.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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

Treatment and study plan

ANX005

Drug

ANX005 is provided as a solution for IV infusion

Primary outcomes

  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    Time frame: Day 1 through Day 71

    An adverse event (AE) was any untoward medical occurrence in a participant who had been administered a pharmaceutical product. An AE did not necessarily have a causal relationship with the product and therefore could be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a pharmaceutical product. An AE could arise with any use, route of administration, formulation, dose (including an overdose), or when used in combination with another pharmaceutical product. A TEAE was an AE with an onset date/time after the first infusion of ANX005 until the end of the study.

    A summary of serious and all other non-serious adverse events regardless of causality is located in the Adverse Events module.

  2. Maximum Change From Baseline in Hemoglobin Levels

    Time frame: Baseline up to Day 71

    Maximum change from Baseline was calculated as the maximum post-Baseline value observed up to Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.

  3. Change From Baseline in Lactate Dehydrogenase Levels at Day 71

    Time frame: Baseline, Day 71

    Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.

  4. Change From Baseline in Percentage of Reticulocytes/Total Cells Count at Day 71

    Time frame: Baseline, Day 71

    Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value.

    Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.

  5. Change From Baseline in Haptoglobin Levels at Day 71

    Time frame: Baseline, Day 71

    Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.

  6. Change From Baseline in Total Bilirubin Levels at Day 71

    Time frame: Baseline, Day 71

    Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.

  7. Change From Baseline in Indirect Bilirubin Levels at Day 71

    Time frame: Baseline, Day 71

    Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.

Secondary outcomes

  1. Change in Percent Inhibition Complement CH50 From Baseline Through Day 71

    Time frame: Baseline, Days 2, 4, 8, 15, 22, 29, 36, 43, 50, 57, and 71

    Change in percent inhibition complement CH50 from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005. A decrease form baseline indicated a better outcome.

  2. Change From Baseline in Complement C4 Level Through Day 71

    Time frame: Baseline, Days 2, 4, 8 (pre-dose and end of infusion), 15, 22, 29, 36, 43, 50, 57, and 71

    Change from Baseline was calculated as the post-Baseline value at Day 71 minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.

  3. Change From Baseline in Complement C1q Level Through Day 71

    Time frame: Baseline, Days 2 (4 hours [hr] after Infusion and end of infusion), 4, 8 (pre-dose, 4 hr after Infusion, and end of infusion), 15, 22, 29, 36, 43, 50, 57, and 71

    Change from Baseline was calculated as the post-Baseline value minus the Baseline value. Baseline was defined as the last non-missing observation recorded before the first dose of ANX005.

Sponsors and collaborators

Lead sponsor

Annexon, Inc.

Industry

Registry information

Official study title

A Phase 2, Open-Label, Repeat Dose Study to Assess the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of Intravenous ANX005 in Subjects With Warm Autoimmune Hemolytic Anemia (wAIHA)

Important dates

Study start
2021
Primary completion
2023
Study completion
2023
First posted
Dec 31, 2020
Registry last updated
Jun 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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