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    Summary
    EudraCT Number:2020-003675-18
    Sponsor's Protocol Code Number:ANX005-wAIHA-02
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-02-11
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedFrance - ANSM
    A.2EudraCT number2020-003675-18
    A.3Full title of the trial
    A Phase 2, Open-Label, Repeat Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Proof-of-Concept of Intravenous ANX005 in Subjects with Warm Autoimmune Hemolytic Anemia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Assess the Safety, Tolerability, Efficacy, PK, PD and POC of Intravenous ANX005 in Patients With wAIHA
    A.4.1Sponsor's protocol code numberANX005-wAIHA-02
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04691570
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAnnexon, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAnnexon, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAnnexon, Inc.
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address180 Kimball Way
    B.5.3.2Town/ citySouth San Francisco
    B.5.3.3Post codeCA 94080
    B.5.3.4CountryUnited States
    B.5.4Telephone number16508225500
    B.5.5Fax number16506369773
    B.5.6E-mailinfo@annexonbio.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code ANX005
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNANX005
    D.3.9.3Other descriptive nameANX005
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Warm Autoimmune Hemolytic Anemia
    E.1.1.1Medical condition in easily understood language
    Warm Autoimmune Hemolytic Anemia
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10003825
    E.1.2Term Autoimmune hemolytic anemia
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this repeat dose Phase 2 study are as follows:
    • To evaluate the safety and tolerability of two once-weekly intravenous infusions of 100 mg/kg ANX005 in patients with wAIHA
    • To evaluate the clinical effect of two once-weekly intravenous infusions of 100 mg/kg ANX005 in patients with wAIHA
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are as follows:
    • To evaluate the pharmacokinetic (PK) profile of ANX005 in subjects with wAIHA
    • To evaluate the effect of ANX005 on classical complement pathway inhibition as measured by complement system related biomarkers in subjects with wAIHA
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients 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 and have relapsed or are refractory to standard of care (SoC) therapy (eg, immunosuppressants, corticosteroids, mAbs such as rituximab, or splenectomy)
    3. Hemoglobin level ≤10.0 g/dL (pre-transfusion) at screening with no alternative explanation for anemia apart from wAIHA
    4. Positive DAT ≥1+ for C3d and IgG within 6 months prior to dosing
    5. Evidence of classical complement pathway activation by at least one of the following within 6 months prior to dosing :
    a. Serum complement component 4 (C4) below the lower limit of normal
    b. CH50 below the lower limit of normal (LLN)
    c. Complement component deposition on the cell surface assessed by flow cytometry
    6. Evidence of active hemolysis at screening 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
    7. If on corticosteroids, patients must be receiving a stable dose ≤1 mg/kg prednisone or equivalent. Tapering of corticosteroids is permitted during the screening period, but the patient’s dose must be stable for at least 14 days prior to ANX005 dosing. Enrolled patients should remain on a stable prescribed dose throughout the study. For patients who are not currently receiving corticosteroids, use should be discontinued within 30 days prior to dosing.
    8. If on immunosuppressants (eg, azathioprine, mycophenolate mofetil, cyclosporine) must be on stable dose for at least 4 weeks prior to dosing.
    9. 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.
    10. If female, must be either postmenopausal (no menses for at least 12 months without an alternative medical cause) or surgically sterilized; OR if a woman of childbearing potential (WOCBP), patient must use a highly effective (ie, failure rate of <1%) method of contraception during the study and for 3 months after the last infusion with study medication.
    11. If male, must agree to either abstain from heterosexual intercourse as the preferred and usual lifestyle (ie, abstinent on a long term and persistent basis) and agree to remain abstinent OR use a male condom during the study and for at least 3 months after the last infusion with study medication; and should also be advised of the benefit for a female partner to use a highly effective (ie, failure rate of <1%) method of contraception
    12. Able to comply with the requirements of the study and complete the full sequence of protocol-related doses, procedures, and evaluations
    13. Ability to understand and provide written informed consent
    E.4Principal exclusion criteria
    Patients must NOT meet any of the following criteria to be eligible to participate in the study:
    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
    3. Platelet count < 30 × 109/L at screening
    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 90 days prior to screening
    7. Received rituximab or other B cell depleting monoclonal antibody (eg, daratumumab) <90 days prior to screening
    8. Clinically significant, ongoing illness or medical condition, including mental illness, suicide attempt, seizures, cardiovascular disease, malignancy, or a medical history that would jeopardize the safety of the patient, limit participation, or compromise the interpretation of the safety data derived from the patient
    9. History of a systemic autoimmune disorder (eg, lupus) or primary immunodeficiency disorder (eg, common variable immune deficiency)
    10. History of meningitis or septicemia within the past 2 years
    11. Clinically significant infection (ie, viral, including symptomatic or asymptomatic SARS-CoV2 [COVID-19], bacterial, fungal, or mycobacterial) that required medical intervention (not including antibiotic prophylaxis) within 1 month prior to screening
    12. Known genetic deficiencies of the complement cascade system
    13. Treatment with an investigational therapeutic agent within 30 days or five half-lives, whichever is longer, prior to screening
    14. Active alcohol or substance abuse or any other reason that makes it unlikely that the patient will comply with study procedures
    15. Females who are pregnant or breastfeeding. Note: To be eligible, WOCBP must have a negative pregnancy test at screening (serum) and Day 1 (urine).
    16. Hypersensitivity to ANX005 or any of the excipients in the ANX005 drug product
    17. Hypersensitivity to or previous allergic reaction to the active substance or to any of the excipients in any of the immunizations required for this study
    18. History of previous sensitivities or allergic or anaphylactic reactions to previous IV medication including, but not limited to, monoclonal antibodies
    19. Positive serology for human immunodeficiency virus (HIV) 1 or 2 at screening
    20. Evidence of hepatitis C virus (HCV) antibody at screening requires reflex testing for HCV ribonucleic acid (RNA). Patients with positive HCV RNA viral load at screening will be excluded. Patients with positive HCV antibody, but negative HCV RNA viral load are eligible but should be monitored throughout the study
    21. Evidence of active or prior hepatitis B virus (HBV) infection. Patients with positive hepatitis B surface antigen (HBsAg) at screening are excluded from the study. Patients with positive HBV core antibody and deoxyribonucleic acid (DNA) viral load are excluded from the study
    22. History of intravenous immunoglobulin (IVIg) treatment within 90 days prior to dosing
    23. History of plasmapheresis or immunoadsorption treatment within 60 days prior to dosing
    24. Received any complement inhibitor (eg, eculizumab, berinert) within 90 days or 5 half-lives, whichever is longer, prior to dosing
    25. Body weight less than 50 kg or greater than 100 kg
    E.5 End points
    E.5.1Primary end point(s)
    • Number and percent of patients experiencing treatment-emergent adverse events (TEAEs)
    • Change from baseline in hemoglobin over time up to Day 71
    • Change from baseline in biomarkers of hemolysis (reticulocyte count, haptoglobin, LDH, total and indirect bilirubin) over time up to Day 71
    E.5.1.1Timepoint(s) of evaluation of this end point
    10 weeks
    E.5.2Secondary end point(s)
    • ANX005 plasma concentration profiles and PK parameters such as AUC, Cmax, Tmax, accumulation ratio, t1/2, λz, CL and Vss
    • Inhibition of classical complement activity as measured by serum CH50 from baseline over time up to Day 71
    • Change and percent change from baseline in serum C4 concentrations over time up to Day 71
    • Change and percent change from baseline in serum free C1q concentrations over time up to Day 71
    E.5.2.1Timepoint(s) of evaluation of this end point
    10 weeks
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Repeat Dose Study
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA13
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    United States
    Austria
    Bulgaria
    France
    Germany
    Italy
    Spain
    United Kingdom
    Czechia
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 9
    F.4.2.2In the whole clinical trial 12
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Once study participation is completed, the subject can discuss with their personal physician about treatment options.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-06-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-04-07
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-06-18
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