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

Primary Purpose

Warm Autoimmune Hemolytic Anemia (wAIHA)

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

ANX005

About this trial

This is an interventional treatment trial for Warm Autoimmune Hemolytic Anemia (wAIHA) focused on measuring AIHA, C1q, complement, RBC lysis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Investigational Site 01

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ANX005

Arm Description

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

Outcomes

Primary Outcome Measures

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

Change in disease activity biomarkers

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

Secondary Outcome Measures

Plasma concentrations

Plasma concentrations of ANX005 over time

Change in complement system biomarkers

Change in CH50 and C1q from baseline

Full Information

NCT ID

NCT04691570

First Posted

December 28, 2020

Last Updated

March 3, 2023

Sponsor

Annexon, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT04691570

Brief Title

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

Official 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)

Study Type

Interventional

2. Study Status

Record Verification Date

February 2023

Overall Recruitment Status

Completed

Study Start Date

November 10, 2021 (Actual)

Primary Completion Date

January 17, 2023 (Actual)

Study Completion Date

January 17, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Annexon, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

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

Detailed Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Warm Autoimmune Hemolytic Anemia (wAIHA)

Keywords

AIHA, C1q, complement, RBC lysis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

6 (Actual)

8. Arms, Groups, and Interventions

Arm Title

ANX005

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

ANX005

Intervention Description

ANX005 is provided as a solution for IV infusion

Primary Outcome Measure Information:

Title

Safety: Treatment-emergent adverse events (TEAEs)

Description

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

Time Frame

Up to Week 16

Title

Change in disease activity biomarkers

Description

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

Time Frame

Baseline to Day 71

Secondary Outcome Measure Information:

Title

Plasma concentrations

Description

Plasma concentrations of ANX005 over time

Time Frame

Up to Day 71

Title

Change in complement system biomarkers

Description

Change in CH50 and C1q from baseline

Time Frame

Baseline to Day 71

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Study Director

Organizational Affiliation

Annexon, Inc.

Official's Role

Study Director

Facility Information:

Facility Name

Investigational Site 01

City

Rochester

State/Province

Minnesota

ZIP/Postal Code

55905

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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