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    Summary
    EudraCT Number:2020-005755-20
    Sponsor's Protocol Code Number:ALZ-801-AD301
    National Competent Authority:Iceland - IMCA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2024-05-27
    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 ConcernedIceland - IMCA
    A.2EudraCT number2020-005755-20
    A.3Full title of the trial
    A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study of the Efficacy, Safety and Biomarker Effects of ALZ-801 in Subjects with Early Alzheimer's Disease and APOE4/4 Genotype
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 3 study of ALZ-801 in patients with early Alzheimer's Disease and APOE4/4 genotype
    A.3.2Name or abbreviated title of the trial where available
    APOLLOE4
    A.4.1Sponsor's protocol code numberALZ-801-AD301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04770220
    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 SponsorAlzheon, 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 supportNational Institute on Aging
    B.4.2CountryUnited States
    B.4.1Name of organisation providing supportAlzheon, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlzheon, Inc.
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address111 Speen Street, Suite 306
    B.5.3.2Town/ cityFramingham, MA
    B.5.3.3Post code01701
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicaltrialsinfo@alzheon.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.3.1Product nameALZ-801
    D.3.2Product code ALZ-801
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNnot yet assigned
    D.3.9.1CAS number 1034190-08-3
    D.3.9.2Current sponsor codeALZ-801
    D.3.9.3Other descriptive name3-(((2S)-2-AMINO-3-METHYL-1-OXOBUTYL)AMINO)-1-PROPANESULFONIC ACID
    D.3.9.4EV Substance CodeSUB218066
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number265
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Early Alzheimer’s Disease (AD)
    E.1.1.1Medical condition in easily understood language
    Early Alzheimer’s Disease (AD)
    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 20.0
    E.1.2Level HLT
    E.1.2Classification code 10001897
    E.1.2Term Alzheimer's disease (incl subtypes)
    E.1.2System Organ Class 100000004852
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Primary Clinical Objectives:
    To evaluate the efficacy of oral ALZ-801 on cognition in subjects with Early AD who are homozygous for the ε4 variant of the APOE gene (APOE4 homozygous or APOE4/4) using ADAS-Cog 13
    and
    To evaluate the safety and tolerability of ALZ-801 over 78 weeks in Early AD subjects with the APOE4/4 genotype.
    See protocol for Primary Objectives on Primary Fluid Biomarkers and Primary Imaging Biomarker.
    E.2.2Secondary objectives of the trial
    Secondary Clinical Objectives:
    To evaluate the effects of ALZ-801 on cognitive and/or functional outcomes: A-IADL, CDR-SB, DAD.
    To evaluate the effects of ALZ-801 on neuropsychiatric symptoms of AD: NPI (12-item form)
    To evaluate the effects of ALZ-801 on additional measures of cognition: ADAS-Cog 11, MMSE
    To evaluate the effects of ALZ-801 on QoL: QoL-AD
    To assess levels of healthcare and caregiver resource usage: RUD Lite
    see protocol for Secondary objectives on Secondary Fluid Biormarkers, Secondary Imaging Biomarkers and Pharmacokinetics.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Cerebrospinal Fluid (CSF) Biomarder sub-study.
    To evaluate the effects of ALZ-801 on other CSF biomarkers of core AD pathology, neurodegeneration, and neuroinflammation in the CSF sub-study:
    - Core AD pathology: tau phosphorylated at threonine 217 (p-tau217), beta amyloid (Aβ40, Aβ42)
    - Neurodegeneration in AD: Neurofilament Light (NfL) and total tau (t-tau)
    - Synaptic toxicity: neurogranin
    - Neuroinflammation: sTREM2 (microglia), and astrocytic markers YKL-40 and glial fibrillary acidic protein (GFAP)
    E.3Principal inclusion criteria
    1. Male or female between the ages of 50 and 80 years (inclusive).
    2. Clinical diagnosis of MCI or Mild Dementia due to AD consistent with the NIA-AA Working Group Criteria
    3. Homozygous for the ε4 allele of the APOE gene (APOE4/4).
    4. MMSE score at Screening of 22 to 30 (inclusive).
    5. CDR Global score at Screening of 0.5 or 1, and a CDR Memory Box score ≥ 0.5.
    6. RBANS delayed memory index score ≤ 85.
    7. Evidence of progressive memory loss over the last 12 months per investigator assessment as captured on the diagnostic verification form.
    8. Can complete the cognitive testing and all other required study procedures.
    9. Has completed at least 6 years of formal education after age of 5 years, and is able to read at minimum of 6th grade level or equivalent per investigator assessment.
    10. Lives at home independently, in a senior living facility, or in an assisted living facility.
    11. Body weight between 48-120 kg (inclusive), and body mass index (BMI) between 17-35 (inclusive).
    12. Except for a diagnosis of AD and the presence of stable medical conditions, is, in the opinion of the Investigator, in good general medical health based upon the results of medical history, physical examination, laboratory tests, vital signs, and ECG.
    13. Has a reliable caregiver or study partner who is willing and able to sign an ICF, to accompany the subject to study visits, and adhere to study requirements. Caregiver or study partner must be able to provide accurate information on subject’s cognitive and functional ability and have face to face contact with subject of at least 10 hours per week.
    See protocol for further inclusion criteria
    E.4Principal exclusion criteria
    1. Screening brain MRI indicative of significant abnormality per central reader, other than AD related atrophy, including, but not limited to; prior hemorrhage > 1 cm3, prior large vascular territorial infarct, > 2 lacunar infarcts (size > 1.5 cm) outside the brain stem, severe white matter changes (deep white matter changes Fazekas grade = 3), superficial hemosiderosis > 1 cm, ventriculomegaly related to normal pressure hydrocephalus, or aneurysm, vascular malformation, subdural hematoma, space-occupying lesion (e.g., meningiomas ≥ 1 cm, abscess or brain tumor).
    2. Diagnosis of neurodegenerative disorder other than AD.
    3. Diagnosed with MDD according to the criteria of the Diagnostic and Statistical Manual of Mental Disorders – Fifth Edition (DSM-5), within one year prior to screening. A subject who does not meet current criteria for MDD and who is on stable doses of antidepressants or mood stabilizers may be included in the study at the discretion of the Investigator.
    4. Currently taking memantine or has taken memantine within 12 weeks prior to the Screening – Part 2 Visit.
    5. History of suicidal behavior within one year prior to screening; or has ongoing suicidal ideation with intent, with or without a specific plan or method (e.g., positive response to C-SSRS items 4 or 5 during the past 6 months).
    6. History of seizures, excluding febrile seizures of childhood or a single distant seizure (> 10 years).
    7. Medically confirmed history of recent cerebral infarct or transient ischemic attack within one year prior to screening.
    8. Medically confirmed history of recent myocardial infarction or unstable, untreated coronary artery disease, or angina pectoris (within 1 year prior to the Screening – Part 2 Visit).
    9. Lifetime history of schizophrenia, schizoaffective disorder, or bipolar disorder.
    10. History of, or currently has, any clinically significant ECG finding, or a QT interval corrected by Fridericia’s method (QTcF) of > 450 msec for males and > 470 msec for females.
    11. History of cancer, diagnosed and treated within the last 3 years prior to the Screening – Part 2 Visit, with the exception of the following: (a) treated basal cell carcinoma of the skin, (b) treated cutaneous squamous cell carcinoma in situ, (c) treated in situ or Stage 1 prostate cancer, and (d) treated in situ cervical cancer (all require approval by the Sponsor’s Medical Officer).
    12. Has donated blood > 250 mL within 6 weeks prior to the Screening – Part 2 Visit.
    13. History of alcohol or drug dependence or abuse within 2 years prior to the Screening – Part 2 Visit or tests positive for drugs of abuse [i.e., amphetamines, MDMA, barbiturates, benzodiazepines, cannabinoids, cocaine, phencyclidine, ethanol, and opiates] at the Screening – Part 2 Visit.
    14. Any significant medical condition or infection (e.g., uncontrolled cardiovascular, GI, endocrinologic, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, renal, or other major disease or malignancy) that is unstable and that would either: (a) place the subject at undue risk from administration of study drug or from undergoing study procedures, or (b) interfere with the interpretation of safety or efficacy evaluations obtained in the course of the study.
    15. Unable to swallow ALZ-801 tablets or has a known intolerance or hypersensitivity to tramiprosate or any of the excipients contained in the ALZ-801 tablets.
    16. Hemoglobin level < 11 g/dL in male subjects or < 10 g/dL in female subjects, or a hemoglobin level > 16 g/dL at the Screening – Part 2 Visit.
    17. Clinically relevant abnormalities in serum thyroid-stimulating hormone (TSH) or calcium. If the subject is taking thyroid hormone replacement therapy, corresponding Screening test values must be considered not clinically significant by the investigator.
    18. Serum vitamin B12 below the lower limit of normal at the Screening – Part 2 Visit. A subject can be rescreened if he/she receives vitamin B12 treatment, and has a normal vitamin B12 level at least four weeks post-treatment.
    19. Any clinical chemistry laboratory value greater than or equal to Common Terminology Criteria for Adverse Events (CTCAE; version 5.0; National Cancer Institute 2018) Grade 2, unless considered not clinically relevant by the Investigator.
    20. One or more of the following at the Screening – Part 2 Visit: Alanine aminotransferase (ALT) > 3 × upper limit of normal (ULN), Aspartate aminotransferase (AST) > 3 × ULN, or Total bilirubin (TBL) > 1.5 × ULN.
    21. Estimated glomerular filtration rate (eGFR) < 40 mL/min per 1.73 m2 according to the Modification of Diet in Renal Disease (MDRD) formula [see National Institute of Diabetes and Digestive and Kidney Diseases website for formula MDRD formula for eGFR (SI units)].
    See protocol for further exclusion criteria
    E.5 End points
    E.5.1Primary end point(s)
    Primary Efficacy Endpoint:
    Cognitive Endpoint: Difference between study drug and placebo in the mean CBL to Week 78 in ADAS-Cog 13 scores
    Primary Fluid Biomarker Endpoints:
    CBL to Week 78 in CSF p-tau181 (in CSF sub-study)
    CBL to Week 78 in plasma p-tau181 in all subjects
    Primary Imaging Biomarker Endpoint:
    CBL to Week 78 in total hippocampal volume as assessed by vMRI
    Safety and Tolerability:
    Incidence and nature of TEAEs, serious TEAEs, and TEAEs leading to withdrawal.
    CBL in vital signs and physical exam, including body weight
    CBL in laboratory parameters (clinical chemistry, hematology, coagulation tests)
    CBL in 12-lead ECG parameters.
    CBL in MRI central readings for ARIA-E or ARIA-H
    CBL in the C-SSRS
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 78
    E.5.2Secondary end point(s)
    Key Secondary Efficacy Endpoint:
    CBL to Week 78 in the following scales: A-IADL, CDR-SB, DAD
    Additional Secondary Efficacy Endpoints:
    CBL to Week 78 in the following scales: NPI, ADAS-Cog 11, MMSE, QoL-AD, RUD Lite
    CBL for the following scales and time points: ADAS-Cog 13 and ADAS-Cog 11 scores to Weeks 13, 26, and 52, A-IADL, CDR-SB, DAD, and NPI scores to Weeks 26 and 52, MMSE scores to Weeks 13, 26, 39, 52, and 65, QoL-AD and RUD Lite scores to Week 52
    Secondary Fluid Biomarker Endpoints:
    CBL to Week 78 in CSF p-tau217, Aβ40, Aβ42, NfL, t-tau, neurogranin, sTREM2, YKL-40, and GFAP (in CSF sub-study)
    CBL to Week 78 in plasma p-tau217, Aβ40, Aβ42, NfL, and GFAP in all subjects
    CBL to 52 weeks in all CSF biomarkers
    CBL to Weeks 13, 26, 39, 52, and 65 in all plasma biomarkers
    CBL to Weeks 13, 26, 39, 52, 65, and/or 78 for other potential biomarkers of interest in plasma or CSF, including assay of CSF Aβ oligomers (to be specified and included in the final SAP)
    Secondary Imaging Biomarker Endpoints:
    CBL for the following MRI measures: Total hippocampal volume to Weeks 26 and 52, Cortical thickness and whole brain volume to Weeks 26, 52, and 78
    E.5.2.1Timepoint(s) of evaluation of this end point
    78 weeks (see protocol for further detail)
    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 No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Biomarker Effects
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA23
    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 Kingdom
    United States
    France
    Germany
    Iceland
    Netherlands
    Spain
    E.8.7Trial has a data monitoring committee Yes
    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 years3
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    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: 90
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 210
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Elderly patients, requiring a caregiver.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 81
    F.4.2.2In the whole clinical trial 300
    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)
    A possible separate extension research study is planned
    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 Decision2021-10-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-11-02
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2024-07-29
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