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    Summary
    EudraCT Number:2021-000037-14
    Sponsor's Protocol Code Number:NEOD001301
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-05-26
    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 ConcernedGermany - PEI
    A.2EudraCT number2021-000037-14
    A.3Full title of the trial
    A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, Efficacy and Safety Study of Birtamimab Plus Standard of Care vs. Placebo Plus Standard of Care in Mayo Stage IV Subjects with Light Chain (AL) Amyloidosis
    Eine randomisierte, multizentrische, doppelblinde, placebokontrollierte Phase-III-Studie zur Wirksamkeit und Sicherheit von Birtamimab plus Standardtherapie im Vergleich zu Placebo plus Standardtherapie bei Patienten mit Leichtketten-(AL)-Amyloidose im Mayo-Stadium IV
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Global Phase 3 Double-Blind, Placebo-Controlled study to assess Efficacy and Safety of Birtamimab Plus Standard of Care vs. Placebo Plus Standard of Care in Mayo Stage IV Subjects with Light Chain (AL) Amyloidosis
    A.4.1Sponsor's protocol code numberNEOD001301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04973137
    A.5.4Other Identifiers
    Name:INDNumber:146070
    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 SponsorProthena Biosciences Limited
    B.1.3.4CountryIreland
    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 supportProthena Biosciences Limited
    B.4.2CountryIreland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationProthena Biosciences Limited
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street Address77 Sir John Rogerson’s Quay, Block C
    B.5.3.2Town/ cityGrand Canal Docklands, Dublin 2
    B.5.3.3Post codeD02 T804
    B.5.3.4CountryIreland
    B.5.6E-mailregaffairs@prothena.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/13/1100
    D.3 Description of the IMP
    D.3.1Product nameBirtamimab
    D.3.2Product code Birtamimab
    D.3.4Pharmaceutical form Powder for 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 INNBIRTAMIMAB
    D.3.9.1CAS number 1608108-91-3
    D.3.9.2Current sponsor codebirtamimab
    D.3.9.3Other descriptive nameHumanized IgG1 kappa antiamyloid
    D.3.9.4EV Substance CodeSUB198016
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    AL amyloidosis involves a hematologic disorder caused by clonal plasma cells that produce misfolded immunoglobulin light chains. Overproduction of misfolded light chains results in both soluble, aggregated forms of light chains and insoluble, fibrillar deposits of abnormal AL protein (amyloid), in the tissues and organs. This can cause a range of symptoms and organ dysfunction including cardiac, renal, and hepatic dysfunction, gastrointestinal involvement and neuropathy and macroglossia
    E.1.1.1Medical condition in easily understood language
    Light chain (AL) amyloidosis is a blood disorder, which causes progressive organ damage as a result of the misfolding of proteins. This disease can produce a range of symptoms and organ dysfunction
    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 PT
    E.1.2Classification code 10036673
    E.1.2Term Primary amyloidosis
    E.1.2System Organ Class 10021428 - Immune 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
    Double-blind Phase
    • To evaluate the efficacy of birtamimab plus standard of care compared to placebo plus standard of care when administered intravenously in Mayo Stage IV subjects with AL amyloidosis by assessing time to all-cause mortality.

    Open-label Extension Phase
    • To evaluate the long-term safety of birtamimab plus standard of care
    in Mayo Stage IV subjects with AL amyloidosis
    E.2.2Secondary objectives of the trial
    To evaluate birtamimab plus standard of care compared to placebo plus standard of care on the following:
    • Change from baseline to Month 9 in the 6-Minute Walk Test (6MWT) distance
    • Change from baseline to Month 9 in health-related quality of life using the Short Form-36 questionnaire Version 2 (SF-36v2)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Aged ≥18 years and legal age of consent according to local regulations
    2.Newly diagnosed and AL amyloidosis treatment naive with cardiac involvement
    3.Confirmed diagnosis of AL amyloidosis
    4.Confirmed Mayo Stage IV AL Amyloidosis as defined by NT-proBNP ≥ 1800 pg/mL and Troponin-T ≥0.025 ng/mL (mcg/L) or high sensitivity cardiac troponin T ≥40 ng/L and dFLC ≥18 mg/dL
    5.Planned first-line chemotherapy contains bortezomib administered subcutaneously weekly
    A comprehensive list of inclusion criteria can be found in the protocol.
    E.4Principal exclusion criteria
    1.Non-AL amyloidosis
    2.NT-proBNP >8500 pg/mL
    3.Meets the International Myeloma Working Group (IMWG) definition of multiple myeloma, except for malignancy biomarker of involved/and uninvolved serum free light chain ratio ≥100
    4.Subject is eligible for and plans to undergo ASCT or organ transplant during the study
    5.Myocardial infarction, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or ECG evidence of acute ischemia, within 6 months prior to the Month 1-Day 1 Visit
    6.Severe valvular stenosis (e.g., aortic or mitral stenosis with a valve area <1.0 cm2) or severe congenital heart disease
    7.ECG evidence of acute ischemia or active conduction system abnormalities
    8.Prior treatment with hematopoietic growth factors, transfusions of blood or blood products within 1 week of Month 1-Day 1
    9.Prior radiotherapy within 4 weeks of Month 1-Day 1
    10.Prior treatment with plasma cell-directed chemotherapy, birtamimab, daratumumab, 11-1F4, anti-serum amyloid P antibody, doxycycline for amyloid, or other investigational treatment directed at amyloid
    11.Waldenström's macroglobulinemia and/or immunoglobulin M monoclonal gammopathy
    A comprehensive list of exclusion criteria can be found in the protocol.
    E.5 End points
    E.5.1Primary end point(s)
    Time to all-cause mortality during the Double-blind Phase
    E.5.1.1Timepoint(s) of evaluation of this end point
    Time from the first dose of study drug until death or end of the Double-blind Phase over 9 months
    E.5.2Secondary end point(s)
    •Change from baseline to Month 9 of the Double-blind Phase in the 6MWT distance (meters)
    •Change from baseline to Month 9 of the Double-blind Phase in the Physical Component Summary (PCS) score of the SF-36v2
    E.5.2.1Timepoint(s) of evaluation of this end point
    from baseline until Month 9 of the Double-blind Phase
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    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) 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 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA60
    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
    Taiwan
    Australia
    Canada
    Israel
    Japan
    Korea, Republic of
    United Kingdom
    United States
    Austria
    Belgium
    Czechia
    Denmark
    France
    Germany
    Greece
    Hungary
    Ireland
    Italy
    Netherlands
    Poland
    Portugal
    Spain
    Türkiye
    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
    The Double-blind Phase of the study will end when a protocol defined number of primary endpoint events have occurred and EOT Visits have been completed for all subjects who remain in the Double-blind Phase
    at that time.
    The end of the study is defined as the date when the last subject last visit occurs in the OLE Phase.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days27
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months9
    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: 110
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 110
    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 state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 132
    F.4.2.2In the whole clinical trial 220
    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)
    At the time of study completion all subjects still on study (subjects still receiving study drug treatment [birtamimab or placebo]) may be considered for entry into an open-label extension study of birtamimab.
    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 Decision2021-09-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-11-12
    P. End of Trial
    P.End of Trial StatusOngoing
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