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

Phase 1

• Conditions: warm autoimmune hemolytic anemia; MedDRA version: 20.0Level: LLTClassification code 10003825Term: Autoimmune hemolytic anemiaSystem Organ Class: 100000004851; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2020-003675-18-IT
• Lead Sponsor: Annexon, Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-08-02
• Last Update Posted: 30 August 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

1. Male or non-pregnant, non-lactating female = 18 years of age on the day of signing informed consent
2. Diagnosis of wAIHA at least 3 months prior to screening
3. Hgb level > 10.0 g/dL (pre-transfusion) with no alternative explanation for anemia apart from wAIHA at screening
4. Positive direct antiglobulin test (DAT) = 1 + for C3d and IgG
5. Evidence of classical complement pathway activation by one of the
below:
a. Serum complement component 4 (C4) below the lower limit of normal
b. CH50 below the lower limit of normal
6. Evidence of active hemolysis based on at least one of the following:
a. LDH above the ULN
b. Indirect bilirubin above the ULN
c. Haptoglobin below the LLN
7. Vaccinations against encapsulated bacterial organisms (N. meningitidis, H. influenzae and S. pneumoniae) within 5 years prior to screening or subject must be willing
to complete vaccinations at least 2 weeks prior to dosing with ANX005.
Documentation of vaccinations must be received at the study site in advance of the scheduled screening visit
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

1. History of or current lymphoma or lymphoproliferative disorder requiring therapy
2. Elevated aspartate aminotransferase or alanine aminotransferase levels > 2.5 times the upper limit of normal at screening
3. Platelet count < 30 x 109/L
4. History of cold agglutinin disease or IgM cold agglutinin titer > 1:64
5. History of solid organ, bone marrow, or stem cell transplantation
6. History of splenectomy within the 3 months prior to screening
7. Other wAIHA treatments:
- Received rituximab or other anti-CD20 monoclonal antibody < 3 months prior to screening
- Receiving steroids > 1 mg/kg of prednisone or equivalent daily at screening
8. Signs and symptoms of, or a diagnosis consistent with, a chronic autoimmune disorder not resulting from primary wAIHA and/or an ANA titer of = 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