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    The EU Clinical Trials Register currently displays   44257   clinical trials with a EudraCT protocol, of which   7344   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:2018-001981-40
    Sponsor's Protocol Code Number:ALN-GO1-003
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-09-11
    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 ConcernedGermany - BfArM
    A.2EudraCT number2018-001981-40
    A.3Full title of the trial
    ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study with an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults with Primary Hyperoxaluria Type 1
    ILLUMINATE-A: eine randomisierte, doppelt verblindete, placebokontrollierte Studie der Phase III mit verlängerter Behandlungsdauer zur Beurteilung der Wirksamkeit und Unbedenklichkeit von Lumasiran bei Kindern und Erwachsenen mit primärer Hyperoxalurie Typ 1
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 Study of an Investigational Drug, Lumasiran (ALN-GO1) with an Extended Dosing Period in Children and Adults with Primary Hyperoxaluria Type 1 Disease
    Eine Studie der Phase III zur Beurteilung der Wirksamkeit und Unbedenklichkeit von Lumas
    Kindern und Erwachsenen mit primärer Hyperoxalurie Typ 1
    A.4.1Sponsor's protocol code numberALN-GO1-003
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/373/2017
    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 Trial Information Line
    B.5.3 Address:
    B.5.3.1Street Address300 Third Street
    B.5.3.2Town/ cityCambridge, Massachusetts
    B.5.3.3Post code02142
    B.5.3.4CountryUnited States
    B.5.4Telephone number001877256 9526
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/16/1637
    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
    D.3.9.2Current sponsor codeALN-GO1
    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
    Primary Hyperoxaluria Type 1 (PH1)
    E.1.1.1Medical condition in easily understood language
    A rare genetic metabolic disorder leading to kidney stones and/or excess calcium deposition in the kidneys, eventually resulting in kidney failure.
    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 20.1
    E.1.2Level PT
    E.1.2Classification code 10020703
    E.1.2Term Hyperoxaluria
    E.1.2System Organ Class 10038359 - Renal and urinary 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 effect of lumasiran on percent reduction in urinary oxalate excretion
    E.2.2Secondary objectives of the trial
    • To characterize the effect of lumasiran on absolute levels of urinary oxalate excretion and the oxalate:creatinine ratios, and plasma oxalate
    • To evaluate the effect of lumasiran, on renal function
    • To evaluate the long-term treatment effect of lumasiran
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age 6 years or older.
    2. Documentation or confirmation of PH1 as determined by genetic analysis prior to randomization.
    3. Mean 24-hour urinary oxalate excretion from the first 2 valid 24-hour urine collections is ≥0.7 mmol/24h/1.73m2
    4. If taking pyridoxine (vitamin B6) for the treatment of PH1, must have been on stable regimen for at least 90 days before randomization, and willing to remain on this stable regimen for 12 months from first study drug administration.
    5. Patient is able to understand and is willing and able to comply with the study requirements and to provide written informed consent. In the case of patients under the age of legal consent, the legal guardian(s) must provide informed consent and the patient should provide assent per local and national requirements.
    E.4Principal exclusion criteria
    1. Medical history includes clinical evidence of extrarenal systemic oxalosis, as determined by the Investigator.
    2. Has any of the following laboratory parameter assessments at screening: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2 × upper limit of normal (ULN), total bilirubin >1.5 x ULN (patients with elevated total bilirubin that is secondary to documented Gilbert’s syndrome are eligible if the total bilirubin is <2 x ULN), international normalized ratio (INR) >1.5 (patients on oral anticoagulant [eg, warfarin] with an INR <3.5 will be allowed)
    3. Has known or clinical laboratory evidence of human immunodeficiency virus (HIV) infection; or evidence of current or chronic hepatitis C virus (HCV), or hepatitis B virus (HBV) infection (local laboratory values are acceptable)
    4. Estimated GFR of <30 mL/min/1.73m2 at screening (calculation will be based on the Modification of Diet in Renal Disease [MDRD] formula for patients ≥18 years of age and the Schwartz Bedside Formula for patients <18 years of age).
    5. 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 randomization
    Medical Conditions
    6. History of renal or liver transplant
    7. Has other medical conditions or comorbidities, which in the opinion of the Investigator, would interfere with study compliance or data interpretation
    8. History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc.
    9. History of intolerance to subcutaneous (SC) injection(s)
    10. Is not willing to comply with the contraceptive requirements during the study period.
    11. Female patient is pregnant, planning a pregnancy, or breast-feeding.
    12. Unwilling or unable to limit alcohol consumption throughout the course of the study. Alcohol intake of >2 units/day is excluded during the study (unit: 1 glass of wine [approximately 125 mL] = 1 measure of spirits [approximately 1 fluid ounce] = ½ pint of beer [approximately 284 mL]
    13. History of alcohol abuse, within the last 12 months before screening, in the opinion of the investigator.
    E.5 End points
    E.5.1Primary end point(s)
    Percent change in 24-hour urinary oxalate excretion from baseline to Month 6
    E.5.1.1Timepoint(s) of evaluation of this end point
    24-hour urine and blood samples will be collected for assessment of pharmacodynamic parameters each month. Urinary oxalate concentrations will be analyzed using a validated central assay at baseline and 6 months to assess the primary endpoint.
    E.5.2Secondary end point(s)
    • Absolute change in 24-hour urinary oxalate corrected for body surface area (BSA) from baseline to Month 6
    • Change in 24-hour urinary oxalate:creatinine ratio (value/upper limit of normal [ULN]) from baseline to Month 6
    • Proportion of patients with 24-hour urinary oxalate level at or below 1.5 x ULN at Month 6
    • Proportion of patients with 24-hour urinary oxalate level at or below ULN at Month 6
    • Change in estimated glomerular filtration rate (eGFR) from baseline to Month 6
    • Percent change in plasma oxalate from baseline to Month 6
    • Absolute change in plasma oxalate from baseline to Month 6
    • Change from baseline (percent and absolute) in 24-hour urinary oxalate excretion, percentage of time that 24-hour urinary oxalate is ≤ 1.5 × ULN, 24-hour urinary oxalate:creatinine ratios and eGFR in the extension periods
    E.5.2.1Timepoint(s) of evaluation of this end point
    24-hour urine and blood samples will be collected for assessment of pharmacodynamic parameters each month. Urinary and plasma oxalate concentrations will be analyzed using a validated central assay at baseline and 6 months to assess the secondary endpoints.
    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 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    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
    Israel
    Jordan
    United Arab Emirates
    United States
    France
    Germany
    United Kingdom
    Netherlands
    Switzerland
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months10
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 20
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Patient or legal guardian (if patient is under the age of legal consent) must be able to understand and be able to comply with the study requirements to provide written informed consent.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 30
    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)
    Expected 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 Decision2018-12-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-12-11
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2024-01-12
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