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    Summary
    EudraCT Number:2021-000325-26
    Sponsor's Protocol Code Number:ANX005-ALS-01
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-02-19
    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 number2021-000325-26
    A.3Full title of the trial
    Phase 2a Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of Intravenous ANX005 in Subjects with Amyotrophic Lateral Sclerosis
    Étude de phase 2a sur l'innocuité, la tolérance, la pharmacocinétique et la pharmacodynamique de l'ANX005 par voie intraveineuse chez des patients atteints de sclérose latérale amyotrophique
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 2 safety and tolerability study using ANX005 for ALS
    A.4.1Sponsor's protocol code numberANX005-ALS-01
    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 Biosciences
    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 Biosciences
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAnnexon Biosciences
    B.5.2Functional name of contact pointSharon Turner-Rinehardt
    B.5.3 Address:
    B.5.3.1Street Address180 Kimball Way, 2nd floor
    B.5.3.2Town/ citySouth San Francisco
    B.5.3.3Post code94080
    B.5.3.4CountryUnited States
    B.5.4Telephone number1650822 5545
    B.5.6E-mailsturner-rinehardt@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 injection/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.3.9.1CAS number 308067-58-5
    D.3.9.2Current sponsor codeANX005
    D.3.9.3Other descriptive nameIMMUNOGLOBULIN G
    D.3.9.4EV Substance CodeSUB20618
    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
    Amyotrophic Lateral Scelrosis (ALS)
    E.1.1.1Medical condition in easily understood language
    Amyotrophic Lateral Scelrosis (ALS)
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10002026
    E.1.2Term Amyotrophic lateral sclerosis
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • To assess the safety and tolerability of ANX005 administered for up to 12 weeks in subjects with ALS
    • To assess the pharmacokinetics (PK) of ANX005 in the serum and cerebrospinal fluid (CSF)
    • To assess the pharmacodynamic (PD) effects of ANX005 in blood and CSF through the assessment of C1q
    E.2.2Secondary objectives of the trial
    • To assess the pharmacodynamic (PD) effects of ANX005 in blood and CSF through the assessment of NfL and pNFH
    • To assess complement activation markers (C4a, CH50) and additional neuronal, glial, and inflammatory biomarkers in blood and CSF
    • To assess the antibody response (immunogenicity) to ANX005
    • To assess clinical and functional status using the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
    • To assess motor strength using handheld dynamometry
    • To assess slow vital capacity (SVC)
    • To assess changes in muscle structure and composition using electrical impedance myography (EIM)
    • To assess quality of life as measured by the 40 item Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Males or females aged 18 and above at the time of informed consent.
    2. Diagnosis of ALS (probable, possible, laboratory-supported probable or definite) according to the World Federation of Neurology revised E1 Escorial criteria
    3. Onset of weakness within 3 years prior to enrollment
    4. Slow Vital Capacity ≥ 60% of predicted normal adjusted for sex, age, and height (from the sitting position).
    5. Able to perform reproducible pulmonary function tests
    6. ALS concomitant medications (e.g., riluzole, edaravone) stable for at least 2 months prior to Screening and expected to remain at that dose until Week 24, the end of study visit.
    7. ALSFRS-R ≥ 30 at the Screening Visit
    8. If female, must be postmenopausal (for at least 2 years), surgically sterilized (bilateral tubal ligation, bilateral oophorectomy, or hysterectomy), or agree to use highly effective methods of
    contraception from screening through Week 24
    9. Males with a woman of childbearing potential partner must agree to use highly effective methods of contraception from Screening through Week 24 (section 6.3).
    10. Documented evidence of previous vaccinations within 5 years prior to screening visit against encapsulated bacterial pathogens (Neisseria meningitidis including serogroup B meningococcus, Haemophilus influenzae, and Streptococcus pneumoniae) or willing to undergo vaccination.
    11. Able to comply with the requirements of the study and complete the full sequence of protocol related procedures and evaluations, including lumbar punctures for collection of cerebrospinal fluid.
    12. If a caregiver/study partner is required, per Investigator discretion, must have a competent caregiver/study partner who is at least 18 years of age and is willing to accompany the subject to select trial visits and to be available by phone if needed, and who is and will remain sufficiently knowledgeable of subject’s ongoing condition to respond to Study Center inquiries about the subject.
    13. Able to understand and provide written, informed consent
    E.4Principal exclusion criteria
    1. Be at risk of suicide or self-harm within the preceding 12 months, in the opinion of the investigator.
    2. Subjects with body weight > 150 kg.
    3. Clinically significant findings on screening laboratory testing, physical examination or vital signs that are not specific to ALS that could interfere with the conduct of the study, the interpretation of the data or increase subject risk.
    4. Signs and symptoms of, or a diagnosis consistent with a chronic
    autoimmune disorder and/or an ANA titer ≥ 1:160.
    5. History of previous infusion reactions, sensitivities, allergic, or anaphylactic reactions to previous medications, environmental stimuli or other substances
    6. Receipt of an experimental agent within 60 days or five half-lives, whichever is longer, prior to Screening or through Week 24.
    7. Prior treatment with any monoclonal antibody within 6 months of screening.
    8. Hypersensitivity to any of the excipients in the ANX005 drug product
    9. Clinically significant intercurrent illness, medical condition, or medical history (including neurological or mental illness, HIV, any active infection, including Hepatitis B or C) that would jeopardize the safety of the subject, limit participation, or compromise the
    interpretation of the data derived from the subject.
    10. Any known genetic deficiencies of the complement-cascade system.
    11. History of chronic systemic steroid use or immunosuppressant medication ending less than 1 month prior to screening.
    12. Active alcohol, drug abuse or substance abuse, or any other reason that makes it unlikely that the subject will comply with study procedures per Investigator discretion.
    13. Females who are pregnant (positive pregnancy test at screening or prior to infusion of ANX005), breast feeding, or unable or unwilling to use highly effective methods of contraception throughout the study.
    14. Contraindication to undergoing an LP including, but not limited to: inability to tolerate an appropriately flexed position for the time necessary to perform an LP; international normalized ratio (INR) >
    1.4 or other coagulopathy; platelet count of < 120,000/μL; infection at the desired lumbar puncture site; taking anti-platelet or coagulant medication within 60 days of screening (Note: low dose aspirin is permitted); history of severe degenerative arthritis of the lumbar spine; suspected non-communicating hydrocephalus or intracranial mass; prior history of spinal mass or trauma
    E.5 End points
    E.5.1Primary end point(s)
    1) to assess the safety and tolerability of ANX005 administered for up to 12 weeks in subjects with ALS
    2) to assess the pharmacokinetics (PK) of ANX005 in the serum and cerebrospinal fluid (CSF)
    3) to assess the pharmacodynamic (PD) effects of ANX005 in blood and CSF through the assessment of C1q
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 12
    E.5.2Secondary end point(s)
    N/A
    E.5.2.1Timepoint(s) of evaluation of this end point
    N/A
    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 No
    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 No
    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.8.3 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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
    Canada
    United States
    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
    LVLP
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months8
    E.8.9.2In all countries concerned by the trial days0
    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: 19
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 6
    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 Information not present in EudraCT
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 25
    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)
    None
    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 Decision2021-04-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-04-06
    P. End of Trial
    P.End of Trial StatusOngoing
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