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    The EU Clinical Trials Register currently displays   43873   clinical trials with a EudraCT protocol, of which   7294   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2021-001519-10
    Sponsor's Protocol Code Number:ALN-GO1-008
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2021-11-30
    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 ConcernedSpain - AEMPS
    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
    Estudio aleatorizado, con doble enmascaramiento y controlado con placebo para evaluar la eficacia, la seguridad, la farmacodinámica y la farmacocinética de lumasiran en pacientes con enfermedad renal recurrente por cálculos de oxalato de calcio y concentraciones elevadas de oxalato en la orina
    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
    Estudio para evaluar lumasiran en adultos con enfermedad renal recurrente por cálculos de oxalato de calcio y concentraciones elevadas de oxalato en la orina
    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 number34667 978 443
    B.5.6E-mailatorres@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
    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.3.8INN - Proposed INNLumasiran sodium
    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 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 placeboSolution for injection
    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
    Enfermedad renal recurrente por cálculos de oxalato de calcio
    E.1.1.1Medical condition in easily understood language
    recurrent kidney stone disease
    Enfermedad renal recurrente por cálculos
    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
    Evaluar el efecto de lumasiran en el cambio porcentual de eliminación de oxalato a través de la orina
    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
    • Evaluar el porcentaje de pacientes que logran una reducción ≥20 % del oxalato en la orina en 24 horas con lumasiran
    • Evaluar el efecto de lumasiran en la sobresaturación de oxalato cálcico en la orina
    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.
    • Edad de 18 años o más (o edad de consentimiento legal, la que sea mayor).
    • Nefrolitiasis recurrente, definida como ≥2 episodios de cálculos en el plazo de los 5 años anteriores a la selección. Para la inclusión, los antecedentes de episodios de cálculos renales se definen como:
    1. el tránsito visible de un cálculo renal
    2. una intervención quirúrgica para la extracción de un cálculo asintomático o sintomático
    3. cálculo renal nuevo (≥1 mm) o con aumento de tamaño (de ≥2 mm) en el TAC.
    • Los 2 cálculos renales analizados más recientemente antes de la aleatorización contenían un 50 % o más de oxalato cálcico. Si solo se dispone de uno de estos análisis, entonces debe haber contenido un 50 % o más de oxalato cálcico.
    • Las concentraciones de oxalato en la orina en 24 horas recogidas en 2 muestras de orina válidas en 24 horas obtenidas durante la selección son >LSN.
    • Estar en disposición de cumplir las recomendaciones alimentarias adecuadas respecto a los alimentos que estimulan la formación de cálculos, incluida la limitación del aporte de vitamina C a <200 mg al día.
    • Si está tomando medicamentos o hidrantes para la profilaxis de los cálculos renales o está tomando medicamentos que alteren la eliminación de oxalato en la orina o la formación de cálculos renales, debe haber recibido una pauta estable durante al menos 60 días antes de la aleatorización y estar en disposición de continuar con esta pauta estable durante todo el estudio.
    • Índice de masa corporal (el peso en kilogramos dividido por la altura en metros elevada al cuadrado) de 20 a <40 kg/m2.
    • El paciente es capaz de entender y está dispuesto a cumplir los requisitos del estudio y a otorgar su consentimiento informado por escrito.
    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 [CKD-EPI] creatinine formula;
    • Received an investigational agent within the last 30 days or 5 half-lives, 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.
    • Presentar cualquiera de las siguientes evaluaciones en los parámetros analíticos en la selección:
    a. Alanina aminotransferasa (ALAT) o aspartato aminotransferasa (AST) >2 veces el LSN.
    b. Bilirrubina total >1,5 veces el LSN. Los pacientes con bilirrubina total elevada que es secundaria al síndrome de Gilbert documentado serán aptos si la bilirrubina total es <2 veces el LSN.
    c. Índice internacional normalizado (IIN) >2,0 (se permitirán pacientes que tomen anticoagulantes orales [p. ej., warfarina] con un IIN <3,5).
    • Presentar una TFGe de <30 ml/min/1,73 m2 en la selección (el cálculo se basará en la fórmula de creatinina de la Colaboración en epidemiología de la enfermedad renal crónica [Chronic Kidney Disease Epidemiology Collaboration, CKD-EPI];
    • Haber recibido un fármaco en investigación en los últimos 30 días o 5 semividas, lo que sea más prolongado, antes de la primera dosis del fármaco del estudio o estar en el periodo de seguimiento de otro estudio clínico antes de la inclusión en el estudio.
    • Pacientes con antecedentes conocidos de causas secundarias de aumento en la concentración de oxalato en la orina o de cálculos renales recurrentes, incluidos:
    a. Hiperoxaluria primaria
    b. Trastornos de la alimentación graves (anorexia o bulimia)
    c. Enfermedad inflamatoria intestinal crónica
    d. Intervención quirúrgica intestinal con malabsorción o diarrea crónica
    e. Sarcoidosis
    f. Hiperparatiroidismo primario
    g. Acidosis tubular renal distal completa
    • Presentar otras enfermedades o enfermedades concomitantes que, a juicio del investigador, podrían alterar el cumplimiento del estudio o la interpretación de los datos
    • Antecedentes de alergias a varios fármacos o antecedentes de reacción alérgica a un oligonucleótido o a GalNAc.
    • Antecedentes de intolerancia a la inyección s.c.
    • No estar en disposición de cumplir con los requisitos anticonceptivos durante el periodo del estudio
    • La paciente está embarazada, prevé quedarse embarazada o está en periodo de lactancia.
    • No estar en disposición o ser incapaz de limitar el consumo de bebidas alcohólicas durante el transcurso del estudio
    • Antecedentes de abuso de bebidas alcohólicas en el plazo de los 12 meses anteriores a la selección a juicio del investigador.
    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)
    Cambio porcentual del oxalato en la orina en 24 horas desde el inicio hasta el mes 6 (promedio entre los meses 4 y 6)
    E.5.1.1Timepoint(s) of evaluation of this end point
    From baseline to Month 6 (average across Months 4 through 6)
    Desde el inicio hasta el mes 6 (promedio entre los meses 4 y 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)
    • Porcentaje de pacientes que logran una reducción ≥20 % del oxalato en la orina en 24 horas desde el inicio hasta el mes 6 (promedio entre los meses 4 y 6)
    • Cambio porcentual en la sobresaturación de oxalato cálcico en la orina desde el inicio hasta el mes 6 (promedio entre los meses 4 y 6)
    E.5.2.1Timepoint(s) of evaluation of this end point
    From baseline to Month 6 (average across Months 4 through 6)
    Desde el inicio hasta el mes 6 (promedio entre los meses 4 y 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 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
    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
    última visita del último sujeto reclutado
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    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 state16
    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
    Tratamiento estándar
    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 Decision2022-04-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-02-09
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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