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    EudraCT Number:2019-004254-28
    Sponsor's Protocol Code Number:CAEL101-301
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-11-05
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2019-004254-28
    A.3Full title of the trial
    A Phase 3, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of CAEL-101 and Plasma Cell Dyscrasia Treatment Versus Placebo and Plasma Cell Dyscrasia Treatment in Plasma Cell Dyscrasia Treatment-Naïve Patients with Mayo Stage IIIb AL Amyloidosis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A double-blind study to evaluate the effectiveness and safety of CAEL-101 in patients with AL amyloidosis.
    A.4.1Sponsor's protocol code numberCAEL101-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04504825
    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 SponsorCaelum Biosciences, 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 supportCaelum Biosciences, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCaelum Biosciences, Inc.
    B.5.2Functional name of contact pointEileen Daniel
    B.5.3 Address:
    B.5.3.1Street Address1200 Florence-Columbus Road #208
    B.5.3.2Town/ cityBordentown, New Jersey
    B.5.3.3Post code08505-4200
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16093373010
    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/3/19/2222
    D.3 Description of the IMP
    D.3.1Product nameCAEL-101
    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 INNNot assigned
    D.3.9.2Current sponsor codeCAEL-101
    D.3.9.3Other descriptive nameCh11-1F4 (NSC-711516)
    D.3.9.4EV Substance CodeSUB198710
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    stage IIIb cardiac AL amyloidosis
    E.1.1.1Medical condition in easily understood language
    Cardiac amyloidosis is a disorder caused by deposits of an abnormal protein (amyloid) in the heart tissue. These deposits make it hard for the heart to work properly.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10007509
    E.1.2Term Cardiac amyloidosis
    E.1.2System Organ Class 10007541 - Cardiac disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objectives are:
    • To determine if CAEL-101 and treatment for plasma cell dyscrasia improves overall survival in Mayo stage IIIb AL amyloidosis patients who are treatment naïve compared to treatment for plasma cell dyscrasia alone
    • To evaluate the safety and tolerability of CAEL-101 in combination with treatment for plasma cell dyscrasia
    E.2.2Secondary objectives of the trial
    The key secondary objectives in this study are:
    • To assess functional improvement as measured by the distance walked in the six-minute walk test (6MWT)
    • To assess quality of life as measured by the Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS)
    • To assess quality of life as measured by the Short Form 36 version 2 Physical Component Score (SF-36 v2® PCS)
    • To assess cardiac improvement as measured by percent Global Longitudinal Strain (GLS%)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Key Inclusion Criteria:
    1. AL amyloidosis stage IIIb based on the 2013 European Modification of the 2004 Standard Mayo Clinic Staging in patients with advanced cardiac involvement at the time of
    2. Measurable hematologic disease at Screening as defined by at least one of the following:
    a. Involved/uninvolved free light chain difference (dFLC) > 4 mg/dL or
    b. Involved free light chain (iFLC)> 4 mg/dL with abnormal ratio or
    c. Serum protein electrophoresis (SPEP) m-spike > 0.5 g/dL
    3. Histopathological diagnosis of amyloidosis based on polarizing light microscopy of green bi-refringent material in Congo red stained tissue specimens AND confirmation of
    AL derived amyloid deposits by at least one of the following:
    a. Immunohistochemistry or
    b. Mass spectrometry or
    c. Characteristic electron microscopy appearance
    4. Cardiac involvement as defined by:
    a. Documented 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
    b. At least one of the following:
    i. Endomyocardial biopsy demonstrating AL cardiac amyloidosis or
    ii. Echocardiogram demonstrating a mean left ventricular wall thickness (calculated as [IVSd+LPWd]/2) of > 12 mm at diastole in the absence of other
    causes (e.g., severe hypertension, aortic stenosis), which would adequately explain the degree of wall thickening or
    iii. Cardiac magnetic resonance imaging (MRI) with gadolinium contrast agent diagnostic of cardiac amyloidosis
    5. Planned first-line treatment for plasma cell dyscrasia is cyclophosphamide-bortezomibdexamethasone (CyBorD)-based regimen administered as SoC.
    6. Women of childbearing potential (WOCBP) must have a negative pregnancy test during Screening and must agree to use highly effective physician approved contraception
    (Section 6.9) from Screening to 90 days at least 5 months following the last study drug administration or 12 months following the last dose of her PCD therapy, whichever is
    7. Men must be surgically sterile or must agree to use effective physician approved contraception (Section 6.9) and refrain from donating sperm from Screening to 90 days
    at least 5 months following the last study drug administration or 12 months following the last dose of his PCD therapy, whichever is longer
    E.4Principal exclusion criteria
    Key Exclusion Criteria:
    1. Have any other form of amyloidosis other than AL amyloidosis
    2. Received prior therapy for AL amyloidosis or multiple myeloma. A maximum exposure of 2 weeks of a CyBorD-based PCD treatment after Screening laboratory samples are
    obtained and prior to randomization is allowed.
    3. Has POEMS (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin changes) syndrome or multiple myeloma defined as
    clonal bone marrow plasma cells > 10% or biopsy-proven bony or extramedullary plasmacytoma and AND one or more of the following CRAB features:
    a. Evidence of end organ damage that can be attributed to the underlying plasma cell proliferative disorder, specifically:
    i. Hypercalcemia: serum calcium > 0.25 mmol/L (> 1mg/dL) higher than the upper limit of normal (ULN) or > 2.75 mmol/L (> 11mg/dL)
    ii. Renal insufficiency: creatinine clearance < 40 mL per minute or serum creatinine > 177mol/L (> 2mg/dL)
    iii. Anemia: hemoglobin value of > 20g/L below the lowest limit of normal, or a hemoglobin value < 100g/L
    iv. Bone lesions: one or more osteolytic lesion on skeletal radiography, computed tomography (CT), or positron emission tomography (PET)/CT. If bone marrow has < 10%
    clonal plasma cells, more than one bone lesion is required to distinguish from solitary plasmacytoma with minimal marrow involvement
    b. Any one of the following biomarkers of malignancy:
    i. 60% or greater clonal plasma cells on bone marrow examination
    ii. More than one focal lesion on MRI that is at least 5mm or greater in size
    4. Have supine systolic blood pressure < 90 mmHg or symptomatic orthostatic hypotension, defined as a decrease in systolic blood pressure upon standing of > 30 mmHg despite
    medical management (e.g., midodrine, fludrocortisones) in the absence of volume depletion.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is overall survival and will be assessed as the date of randomization to the date of death. Patients living at the end of the study will be censored at their last known date alive. Patients who discontinue study treatment or the study prematurely will be included in the analysis (even if after discontinuation of study treatment or the study) using the death date or censoring time point, as appropriate.
    E.5.1.1Timepoint(s) of evaluation of this end point
    End of Treatment (EoT) and Follow-up every 12 weeks thereafter. The primary endpoint is also evaluated during the Treatment Period.
    E.5.2Secondary end point(s)
    The four key secondary efficacy endpoints are:
    • Changes from baseline to Week 50 in the 6MWT distance
    • Changes from baseline to Week 50 in the KCCQ-OS
    • Changes from baseline to Week 50 in the SF-36 v2 PCS
    • Changes from baseline to Week 50 in GLS%
    E.5.2.1Timepoint(s) of evaluation of this end point
    6MWT, KCCQ-QS & SF-36: Weeks 1, 14, 26, 38 & 50. GLS% (echocardiogram): Screening (Week -4 to 0), Weeks 14, 26 & 50.
    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 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. 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA40
    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
    Russian Federation
    United States
    United Kingdom
    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 be considered complete at the time when the expected number of deaths have been observed, the last surviving patient to start study drug completes at least 50 weeks of treatment or Sponsor decision to terminate the study, whichever comes first.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    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: 66
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 45
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 31
    F.4.2.2In the whole clinical trial 111
    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)
    Patients who are still receiving treatment with study drug when the study is complete may be eligible for enrollment into an open-label, extension study of CAEL-101. Patients enrolling into the extension study will complete all assessments required at the 14-day EOT visit. Patients randomized to placebo in this study will be permitted to enroll in extension study and will start CAEL-101 in that trial. Patients not enrolling into the extension study will complete all visits in the EOT Period.
    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 Decision2021-01-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-01-04
    P. End of Trial
    P.End of Trial StatusOngoing
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