Study to Assess the Safety, Tolerability, Efficacy, PK, PD and POC of Intravenous ANX005 in Patients With wAIHA

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 12

Inclusion Criteria

Subjects must meet ALL the following criteria to be eligible to participate in the study:
1. Male or female = 18 years of age on the day of signing informed consent
2. Diagnosis of wAIHA at least 3 months prior to screening with a DAT =1 positive for IgG ±C3, or a diagnosis of mixed AIHA that is DAT positive for both IgG and C3, with a presence of a cold antibody with a thermal amplitude =30ºC
3. Hemoglobin level =10.0 g/dL (pre-transfusion) in wAIHA-01 within 6 weeks of the Day 1 dosing day or during the Week -6 or rescreening visits
4. Evidence of classical complement pathway activation in wAIHA-01 within 6 weeks of the Day 1 dosing date or during the wAIHA-02 screening:
a. Serum complement component 4 (C4) level no greater than 1.5 times the lower limit of normal and/or
b. CH50 below the lower limit of normal (LLN)
5. Evidence of active hemolysis based on at least one of the following:
a. LDH above the upper limit of normal (ULN)
b. Indirect bilirubin above the ULN
c. Haptoglobin below the LLN
6. Patients must have been previously vaccinated for encapsulated bacteria within 5 years prior to screening or 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.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 10
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

Subjects must not meet any of the following criteria:
1. Active lymphoma, lymphoproliferative disorder, or other malignancy
requiring therapy and/or not clinically stable or in remission for at least
3 months prior to screening
2. Elevated aspartate aminotransferase or alanine aminotransferase levels > 2.5 times the upper
limit of normal at screening unless associated with wAIHA
3. Platelet count < 30 x 109/L
4. History of isolated cold agglutinin disease
5. History of solid organ, bone marrow, or stem cell transplantation
6. History of splenectomy within the 3 months prior to screening
7. Received rituximab or other B cell depleting monoclonal antibody (eg.
daratumumab) <90 days prior to screening
8. Signs and symptoms of, or a diagnosis consistent with, a chronic autoimmune disorder and/or an ANA titer =1:160
9. Known genetic deficiencies of the complement cascade system

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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