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    Summary
    EudraCT Number:2014-003865-11
    Sponsor's Protocol Code Number:NEOD001-CL002
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2015-07-17
    Trial results View 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 number2014-003865-11
    A.3Full title of the trial
    A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled, 2-Arm, Efficacy and Safety Study of NEOD001 Plus Standard of Care vs. Placebo Plus Standard of Care in Subjects with Light Chain (AL) Amyloidosis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to see how effective and safe NEO001 is when given together with the standard treatment (for plasma cell dyscrasia) in comparison to placebo together with the standard treatment in people with Light Chain (AL) Amyloidosis.
    A.4.1Sponsor's protocol code numberNEOD001-CL002
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02312206
    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 Therapeutics 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 Therapeutics Limited
    B.4.2CountryIreland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationProthena Biosciences Inc
    B.5.2Functional name of contact pointSpencer Guthrie
    B.5.3 Address:
    B.5.3.1Street Address650 Gateway Blvd
    B.5.3.2Town/ citySouth San Francisco, California
    B.5.3.3Post code94080
    B.5.3.4CountryUnited States
    B.5.4Telephone number(650) 615-2106
    B.5.5Fax number(650) 837-8560
    B.5.6E-mailspencer.guthrie@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 nameNEOD001
    D.3.2Product code NEOD001
    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 INNN/A
    D.3.9.1CAS number N/A
    D.3.9.2Current sponsor codeNEOD001
    D.3.9.3Other descriptive nameHumanized IgG1, kappa anti-serum amyloid A and anti-AL amyloid antibody
    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
    Light chain (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 dysfuction
    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
    To evaluate the efficacy of NEOD001 plus standard of care vs. placebo plus standard of care when administered intravenously in subjects with AL amyloidosis by assessing time to all- cause mortality or cardiac hospitalization
    E.2.2Secondary objectives of the trial
    In subjects with AL amyloidosis, comparing NEOD001 plus standard of care vs. placebo plus standard of care:
    • To evaluate the cardiac response rate as assessed by NT-proBNP
    • To evaluate time to cardiac mortality or cardiac hospitalization
    • To evaluate cardiac functional response using the 6 Minute Walk Test (6MWT)
    • To evaluate general health-related quality of life using the Short Form 36 questionnaire (SF-36)
    • To evaluate cardiac-specific quality of life using the Kansas City Cardiomyopathy Questionnaire (KCCQ)
    • To evaluate the renal response rate using established criteria (Palladini 2014)
    • To evaluate the hepatic response rate according to consensus criteria (Comenzo 2012)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects must meet all of the following criteria:
    1. Aged ≥ 18 years
    2. Newly diagnosed and AL amyloidosis treatment naïve
    3. Bone marrow consistent with plasma cell dyscrasia within 42 days prior to Month 1-Day 1
    4. Confirmed diagnosis of AL amyloidosis by the following:
    • Histochemical diagnosis of amyloidosis determined by polarizing light microscopy of green birefringent material in Congo red-stained tissue specimens OR characteristic electron microscopy appearance AND
    • Confirmatory immunohistochemistry OR mass spectroscopy of AL amyloidosis
    5. Confirmed diagnosis of AL amyloidosis by mass spectrometry of tissue biopsy if the subject meets any of the following:
    • is black or African American
    • is over 75 years of age with concurrent monoclonal gammopathy
    • has a history of familial amyloidosis and has concurrent monoclonal gammopathy
    6. Cardiac involvement as defined by all of the following:
    • Past documented or presently noted clinical signs and symptoms supportive of a diagnosis of heart failure in the setting of a confirmed diagnosis of AL amyloidosis in the absence of an alternative explanation for heart failure
    • Echocardiogram demonstrating a mean left ventricular wall thickness > 12 mm in the absence of other cardiac cause to explain wall thickening (such as hypertension or cardiac valvular disease)
    • NT-proBNP ≥ 650 pg/mL in the absence of renal failure or atrial fibrillation with an uncontrolled ventricular rate
    7. Planned first-line chemotherapy contains bortezomib administered weekly and SC
    8. Adequate bone marrow reserve, hepatic and renal function, as demonstrated by: absolute neutrophil count (ANC) ≥ 1.0 x10^9/L
    • platelet count ≥ 75 x 10^9/L
    • hemoglobin ≥ 9 g/dL
    • total bilirubin ≤ 2 x upper limit of normal (ULN)
    • aspartate aminotransferase (AST) / serum glutamic oxaloacetic transaminase (SGOT) ≤ 3 x ULN
    • alanine aminotransferase (ALT) / serum glutamic pyruvic transaminase (SGPT) ≤ 3 x ULN
    • alkaline phosphatase (ALP) ≤ 5 x ULN (except for patients with hepatomegaly and isozymes specific to liver, rather than bone)
    • estimated glomerular filtration rate (eGFR) ≥ 30 mL/min
    9. Seated systolic blood pressure 90-180 mmHg
    10. Distance walked during 6MWT is ≥30 meters and ≤550 meters
    11. Women of childbearing potential (WOCBP) must have two negative pregnancy tests during Screening, the second within 24 hours prior to the first administration of study drug and must agree to use physician-approved contraception from Screening to 30 days following the last study drug administration
    12. Male subjects must be surgically sterile or must agree to use physician-approved contraception from Screening to 30 days following the last study drug administration
    13. Ability to understand and willingness to sign an informed consent form prior to initiation of any study procedures
    E.4Principal exclusion criteria
    Subjects must meet none of the following criteria:
    1. Non-AL amyloidosis
    2. NT-proBNP > 8,500 pg/mL
    3. Meets the International Myeloma Working Group (IMWG) definition of Multiple Myeloma (see Appendix 11)
    *Note that subjects who meet the IMWG definition of symptomatic multiple myeloma with signs and/or symptoms attributable only to associated amyloidosis and who do not otherwise meet the criteria for diagnosis of smoldering myeloma are potentially eligible upon approval of the Sponsor.
    4. Subject is eligible for and plans to undergo ASCT
    5. Symptomatic orthostatic hypotension that in the medical judgment of the Investigator would interfere with subject’s ability to safely receive treatment or complete study assessments
    6. Myocardial infarction, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia, within 6 months prior to the Month 1-Day 1 Visit
    7. Severe valvular stenosis (e.g. aortic or mitral stenosis with a valve area <1.0 cm2) or severe congenital heart disease
    8. ECG evidence of acute ischemia or active conduction system abnormalities with the exception of any of the following:
    • First degree AV-block
    • Second degree AV-block Type 1 (Mobitz Type 1 / Wenckebach type)
    • Right or left bundle branch block
    • Atrial fibrillation with a controlled ventricular rate
    Prior to randomization, any ECG screening abnormalities must be noted as “not clinically significant” by the Investigator
    9. Peripheral neuropathy assessed as National Cancer Institute-Common Terminology Criteria for Adverse Events ( NCI-CTCAE) Grade 2 with pain, Grade 3 or Grade 4
    10. Subject is receiving oral or IV antibiotics, antifungals or antivirals within 1 week of Month 1-Day 1 with the exception of prophylactic oral agents
    11. Prior treatment with hematopoietic growth factors, transfusions of blood or blood products within 1 week of Month 1-Day 1
    12. Prior radiotherapy within 4 weeks of Month 1-Day 1
    13. Major surgery within 4 weeks of Month 1-Day 1 or planned major surgery during the study
    14. Active malignancy with the exception of any of the following:
    • Adequately treated basal cell carcinoma, squamous cell carcinoma, or in situ cervical cancer
    • Adequately treated Stage I cancer from which the subject is currently in remission and has been in remission for 2 years
    • Stage I prostate cancer that does not require treatment
    • Any other cancer from which the subject has been disease-free for ≥ 2 years
    15. History of Grade ≥ 3 infusion-associated AEs or hypersensitivity to another monoclonal antibody, or known hypersensitivity to diphenhydramine (or an equivalent H1 antihistamine) or acetaminophen (or its equivalent, paracetamol)
    16. Uncontrolled, active human immunodeficiency virus (HIV), Hepatitis B or Hepatitis C infection
    17. Prior treatment with NEOD001
    18. Treatment with another investigational agent within 30 days of Month 1-Day 1
    19. Women who are pregnant or lactating
    20. Any condition which could interfere with, or the treatment for which might interfere with, the conduct of the study or which would, in the opinion of the Investigator, unacceptably increase the subject’s risk by participating in the study
    E.5 End points
    E.5.1Primary end point(s)
    Time to all-cause mortality or cardiac hospitalization
    E.5.1.1Timepoint(s) of evaluation of this end point
    As adjudicated by the CEC
    E.5.2Secondary end point(s)
    • NT-proBNP
    • Time to cardiac mortality or cardiac hospitalization
    • 6MWT
    • SF-36
    • KCCQ
    • Renal best response
    • Hepatic best response
    E.5.2.1Timepoint(s) of evaluation of this end point
    • NT-proBNP best response from baseline to Month 9
    • Time to cardiac mortality or cardiac hospitalization as adjudicated by the CEC
    • Change from baseline to Month 9 in the 6MWT
    • Change from baseline to Month 9 in the SF-36
    • Change from baseline to Month 9 in the KCCQ
    • Renal best response from baseline to Month 9 (Palladini 2014)
    • Hepatic best response from baseline to Month 9 (Comenzo 2012)
    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 Yes
    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 concerned10
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA25
    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
    Austria
    Belgium
    Canada
    Czech Republic
    France
    Germany
    Greece
    Israel
    Italy
    Netherlands
    Spain
    United Kingdom
    United States
    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 study will end when approximately 156 primary endpoint events have occurred and EOS visits have been completed for all subjects who remain on study at that time. Events are defined as deaths due to any cause, or cardiac hospitalizations as adjudicated by the CEC
    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 days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    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: 200
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 36
    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 state44
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 236
    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 (i.e., once approximately 156 primary endpoint events have been reached), subjects still on treatment will be invited to enrol in an open-label extension study of NEOD001.
    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 Decision2015-06-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-05-07
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2018-05-31
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