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    Summary
    EudraCT Number:2021-001519-10
    Sponsor's Protocol Code Number:ALN-GO1-008
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2021-12-01
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2021-001519-10
    A.3Full title of the trial
    A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, Pharmacodynamics, and Pharmacokinetics of Lumasiran in Patients with Recurrent Calcium Oxalate Kidney Stone Disease and Elevated Urinary Oxalate Levels
    Studio randomizzato, in doppio cieco, controllato con placebo per valutare l’efficacia, la sicurezza, la farmacodinamica e la farmacocinetica di lumasiran in pazienti con calcolosi renale da calcio ossalato recidivante e livelli elevati di ossalato urinari
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate Lumasiran in Adults with Recurrent Calcium Oxalate
    Kidney Stone Disease and Elevated Urinary Oxalate Levels
    Uno studio per valutare Lumasiran in adulti con ricorrenti calcoli di ossalato di calcio Malattia da calcoli renali ed elevati livelli di ossalato urinario
    A.3.2Name or abbreviated title of the trial where available
    Lumasiran (ALN-GO1)
    Lumasiran (ALN-GO1)
    A.4.1Sponsor's protocol code numberALN-GO1-008
    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 SponsorALNYLAM PHARMACEUTICALS, 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 supportAlnylam Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlnylam Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointClinical Trials Information Line
    B.5.3 Address:
    B.5.3.1Street Address300 Third Street
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02142
    B.5.3.4CountryUnited States
    B.5.4Telephone number+31203697861
    B.5.5Fax number000000
    B.5.6E-mailclinicaltrials@alnylam.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 namelumasiran
    D.3.2Product code [ALN-GO1]
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1834612-06-4
    D.3.9.2Current sponsor codeALN-G01
    D.3.9.3Other descriptive nameALN-65585, AD-65585
    D.3.9.4EV Substance CodeSUB180822
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number189
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 placeboSolution for injection/infusion
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Recurrent calcium oxalate kidney stone disease
    Recidiva di calcolosi renale da ossalato di calcio
    E.1.1.1Medical condition in easily understood language
    recurrent kidney stone disease
    Calcoli renali ricorrenti
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10023436
    E.1.2Term Kidney stone
    E.1.2System Organ Class 100000004857
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the effect of lumasiran on the percent change in urinary
    oxalate excretion
    Valutare l’effetto di lumasiran sulla variazione percentuale nell’escrezione di ossalato urinario
    E.2.2Secondary objectives of the trial
    • To evaluate the percentage of patients who achieve a =20% reduction
    in 24-hour urinary oxalate with lumasiran
    • To evaluate the effect of lumasiran on urinary calcium oxalate
    supersaturation
    • Valutare la percentuale di pazienti che raggiungono una riduzione del >20% di ossalato urinario nelle 24 ore con lumasiran
    • Valutare l’effetto di lumasiran sulla sovrasaturazione dell’ossalato di calcio urinario
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Age 18 years or older (or age of legal consent, whichever is older).
    • Recurrent kidney stone disease, defined as =2 stone events within the
    5 years prior to screening. For inclusion, a historical kidney stone event
    is defined as:
    1. the visible passage of a kidney stone
    2. a procedural intervention for removal of an asymptomatic or
    symptomatic stone
    3. a new (=1 mm) or enlarged (by =2 mm) kidney stone on CT imaging
    • The 2 most recently analyzed kidney stones prior to randomization
    contained 50% or more of calcium oxalate; if only one stone analysis is
    available, then it must have contained 50% or more of calcium oxalate.
    • 24-hour urinary oxalate levels from 2 valid 24-hour urine collections
    obtained during screening are >ULN.
    • Willing to adhere to dietary recommendations appropriate for stone
    formers including limiting vitamin C supplementation to <200 mg daily.
    • If taking medications and/or hydrating for kidney stone prophylaxis,
    or taking medications that alter urinary oxalate excretion and/or kidney
    stone formation, must have been on a stable regimen for at least 60 days
    before randomization, and willing to remain on this stable regimen for
    the duration of the study.
    • Body mass index (the weight in kilograms divided by the square of the
    height in meters) of 20 to <40 kg/m2.
    • Patient is able to understand and is willing and able to comply with the
    study requirements and to provide written informed consent.
    • Età pari o superiore a 18 anni (o età del consenso legale, a seconda di quale sia maggiore).
    • Recidiva di calcolosi renale, definita come >2 eventi nei 5 anni precedenti allo screening. Per l’inclusione, un evento storico di calcolosi renale è definito come:
    1. il passaggio visibile di un calcolo renale
    2. un intervento procedurale per la rimozione di un calcolo asintomatico o sintomatico
    3. un nuovo calcolo renale (>1 mm) o ingrossato (di >2 mm) visibile tramite TAC
    • I 2 calcoli renali analizzati più recentemente prima della randomizzazione contenevano il 50% o più di ossalato di calcio; se è disponibile l'analisi di un solo calcolo doveva contenere il 50% o più di ossalato di calcio.
    • I livelli di ossalato urinario nelle 24 ore derivati da 2 raccolte di urine valide nelle 24 ore ottenute durante lo screening sono >ULN (limite superiore di normalità).
    • Disponibilità ad aderire alle appropriate raccomandazioni alimentari in relazione alla formazione dei calcoli, tra cui limitare l’integrazione di vitamina C a <200 mg al giorno.
    • Se si stanno assumendo farmaci e/o idratanti per la profilassi della calcolosi renale, o farmaci che alterano l’escrezione urinaria di ossalato e/o la formazione di calcoli renali, si deve aver seguito un regime stabile per almeno 60 giorni prima della randomizzazione e si deve essere disposti a mantenere tale regime stabile per l’intera durata dello studio.
    • Indice di massa corporea (il peso espresso in chilogrammi diviso per il quadro dell’altezza in metri) da 20 a <40 kg/m2.
    • Il/la paziente è in grado di comprendere ed è disposto/a e in grado di attenersi ai requisiti dello studio e di fornire il consenso informato scritto.
    E.4Principal exclusion criteria
    • Has any of the following laboratory parameter assessments at
    screening:
    a. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST)
    >2 × ULN
    b. Total bilirubin >1.5 × ULN. Patients with elevated total bilirubin that is
    secondary to documented Gilbert's syndrome are eligible if the total
    bilirubin is <2 × ULN
    c. International normalized ratio (INR) >2.0 (patients on oral
    anticoagulant [eg, warfarin] with an INR <3.5 will be allowed)
    • Has an eGFR of <30 mL/min/1.73m2 at screening (calculation will be
    based on the Chronic Kidney Disease Epidemiology Collaboration [CKDEPI]
    creatinine formula;
    • Received an investigational agent within the last 30 days or 5 halflives,
    whichever is longer, prior to the first dose of study drug, or are in
    follow-up of another clinical study prior to study enrollment
    • Patients with a known history of secondary causes of elevated urinary
    oxalate and/or recurrent kidney stones including:
    a. Primary hyperoxaluria
    b. Severe eating disorders (anorexia or bulimia)
    c. Chronic inflammatory bowel disease
    d. Intestinal surgery with malabsorption or chronic diarrhea
    e. Sarcoidosis
    f. Primary hyperparathyroidism
    g. Complete distal renal tubular acidosis
    • Has other medical conditions or comorbidities which, in the opinion of
    the Investigator, would interfere with study compliance or data
    interpretation.
    • History of multiple drug allergies or history of allergic reaction to an
    oligonucleotide or GalNAc.
    • History of intolerance to SC injection(s)
    • Is not willing to comply with the contraceptive requirements during
    the study period
    • Female patient is pregnant, planning a pregnancy, or breast-feeding
    • Unwilling or unable to limit alcohol consumption throughout the
    course of the study
    • History of alcohol abuse, within the last 12 months before screening,
    in the opinion of the Investigator.
    • Preseza di una qualsiasi delle seguenti valutazioni dei parametri di laboratorio allo screening:
    a. Alanina aminotransferasi (ALT) o aspartato aminotransferasi (AST) >2 x ULN
    b. Bilirubina totale >1,5 x ULN. Pazienti con livelli elevati di bilirubina totale, collegata alla sindrome di Gilbert documentata sono idonei se la bilirubina totale è <2 × ULN
    c. Rapporto normalizzato internazionale (INR) >2,0 (è consentita la partecipazione a pazienti che assumono anticoagulante orale [ad es. warfarin] con INR <3,5)
    • Presenza di una eGFR pari a <30 ml/min/1,73 m2 allo screening (il calcolo si baserà sulla formula della creatinina secondo la Chronic Kidney Disease Epidemiology Collaboration [CKD- EPI];
    • Precedente trattamento con un agente sperimentale negli ultimi 30 giorni o 5 emivite, a seconda di quale periodo sia più lungo, anteriormente alla prima dose del farmaco dello studio, o in corso di follow-up di un altro studio clinico prima dell’arruolamento nello studio
    • Pazienti con anamnesi nota di cause secondarie di livelli elevati di ossalato urinario e/o recidive di calcolosi renale, compresi:
    a. Iperossaluria primaria
    b. Disturbi alimentari gravi (anoressia o bulimia)
    c. Malattia infiammatoria intestinale cronica
    d. Chirurgia intestinale con malassorbimento o diarrea cronica
    e. Sarcoidosi
    f. Iperparatiroidismo primario
    g. Acidosi tubulare renale distale completa
    • Presenza di altre condizioni mediche o comorbidità che, a giudizio dello sperimentatore, potrebbero interferire con la conformità allo studio o l’interpretazione dei dati.
    • Anamnesi di allergie farmacologiche multiple o anamnesi di reazione allergica a un oligonucleotide o a GalNAc.
    • Anamnesi di intolleranza alla/e iniezione/i SC
    • Indisponibilità ad attenersi agli obblighi contraccettivi durante il periodo dello studio
    • Paziente di sesso femminile in gravidanza, sta pianificando una gravidanza o in allattamento
    • Indisponibilità o incapacità di limitare il consumo di alcol per tutta la durata dello studio
    • Anamnesi di abuso di alcol negli ultimi 12 mesi prima dello screening, a parere dello sperimentatore.
    E.5 End points
    E.5.1Primary end point(s)
    Percent change in 24-hour urinary oxalate from baseline to Month 6
    (average across Months 4 through 6)
    Variazione percentuale nell’ossalato urinario nelle 24 ore dal basale al Mese 6 (media tra i Mesi da 4 a 6)
    E.5.1.1Timepoint(s) of evaluation of this end point
    From baseline to Month 6 (average across Months 4 through 6)
    Dal basale al Mese 6 (media tra i Mesi da 4 a 6)
    E.5.2Secondary end point(s)
    • Percentage of patients who achieve a =20% reduction in 24-hour
    urinary oxalate from baseline to Month 6 (average across Months 4
    through 6)
    • Percent change in urinary calcium oxalate supersaturation from
    baseline to Month 6
    (average across Months 4 through 6)
    • Percentuale di pazienti che raggiungono una riduzione =20% nell’ossalato urinario nelle 24 ore dal basale al Mese 6 (media tra i Mesi da 4 a 6)
    • Variazione percentuale della supersaturazione dell'ossalato di calcio urinario dal basale al Mese 6
    (media tra i Mesi da 4 a 6)
    E.5.2.1Timepoint(s) of evaluation of this end point
    From baseline to Month 6 (average across Months 4 through 6)
    Dal basale al Mese 6 (media tra i Mesi da 4 a 6)
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    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 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 Yes
    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 trial3
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA14
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Belgium
    Italy
    Spain
    Switzerland
    United Kingdom
    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
    last patient last visit
    last patient last visit
    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 months1
    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 months0
    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: 90
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 30
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 92
    F.4.2.2In the whole clinical trial 120
    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)
    Standard of care
    Standard of care
    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 Decision2022-02-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-12-16
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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