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    Summary
    EudraCT Number:2019-001346-17
    Sponsor's Protocol Code Number:ALN-GO1-005
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2019-07-23
    Trial 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 ConcernedUK - MHRA
    A.2EudraCT number2019-001346-17
    A.3Full title of the trial
    ILLUMINATE-C: A Single Arm Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1 (PH1)
    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) in patients with advanced primary hyperoxaluria Type 1
    A.4.1Sponsor's protocol code numberALN-GO1-005
    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/ cityCambrigde, 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 CodeSUB194008
    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
    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
    Cohort A
    • Evaluate the effect of lumasiran on plasma oxalate in patients who are not on dialysis therapy
    Cohort B
    • Evaluate the effect of lumasiran on plasma oxalate levels in patients who are on dialysis therapy
    E.2.2Secondary objectives of the trial
    • Evaluate the effect of lumasiran on change in plasma oxalate area under the curve (AUC) between dialysis sessions
    • Evaluate the long-term effects of lumasiran on change in plasma oxalate
    • Evaluate the effect of lumasiran on change in urinary oxalate excretion
    • Evaluate quality of life in patients with PH1
    • Characterize the PK of lumasiran
    • Evaluate the effect of lumasiran on nephrocalcinosis
    • Evaluate the effect of lumasiran on dialysis requirements
    • Evaluate the effect of lumasiran on the occurrence of renal stones
    • Evaluate the effect of lumasiran on renal function
    • Evaluate the effect of lumasiran on change in measures of systemic oxalosis
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Cohorts A and B
    1. Have reached at least 37 weeks estimated gestational age (full-term infant) at consent (or assent).
    2. Documentation or confirmation of PH1 as determined by genetic analysis prior to initial dosing.
    3. Patients with eGFR ≤45 mL/min/1.73 m2 as calculated by the MDRD formula if ≥18 years or Schwartz Bedside Formula if ≥12 months to <18 years, or patients <12 months of age with serum creatinine that is considered elevated for age at consent.
    4. The mean of the three most recent screening plasma oxalate samples collected prior to Day 1 is ≥20 μmol/L. In Cohort B, these 3 collections may include the first pre-dialysis sample from each plasma oxalate profile.
    5. If taking therapeutic vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days before consent, and is able and willing to remain on this stable regimen until at least the Month 6 visit. Dose adjustments for interval weight gain are acceptable.
    6. Patient 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.

    Cohort B Only
    1. On a stable hemodialysis regimen for at least 4 weeks prior to Screening plasma oxalate assessment; able and willing to maintain this regimen through Month 6 visit, with changes to dialysis regimen permitted only when medically indicated.
    E.4Principal exclusion criteria
    1. Has any of the following laboratory parameter assessments at Screening:
    a. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2× ULN for age
    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) >1.5 for patients not on anticoagulants. Patients on oral anticoagulants (eg, warfarin) with an INR <3.5 will be allowed.
    d. Hemoglobin <8.0 dL
    2. Has known active human immunodeficiency virus (HIV) infection; or evidence of current or chronic hepatitis C virus (HCV) or hepatitis B (HBV) infection.
    3. 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 during Screening. Consultation with the Medical Monitor is required if treated with unapproved products prior to consent regardless of the time interval prior to the first dose of study drug.
    4. Known history of allergic reaction to an oligonucleotide or GalNAc.
    5. Diagnosis of conditions other than PH1 contributing to renal insufficiency such as glomerulonephritis, nephrotic syndrome, or lupus nephritis.
    6. Any condition or comorbidity, which in the opinion of the Investigator, would make the patient unsuitable for dosing or would interfere with study compliance, data interpretation, patient safety, and/or patient participation in the study. This includes significant active and poorly controlled (unstable) cardiovascular, neurologic, gastrointestinal, endocrine, renal or psychiatric disorders unrelated to PH1 identified by key laboratory abnormalities or medical history.
    7. 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].
    8. History of alcohol abuse, within the last 12 months before screening, in the opinion of the investigator.
    9. Patients with a previous liver transplant or for whom a liver transplant is anticipated in the next 6 months.
    10. Patients with a previous kidney transplant who are currently receiving immunosuppression to prevent transplant rejection.
    11. Patients maintained on a peritoneal dialysis regimen.
    12. Patients who in the opinion of the Investigator plan to start dialysis replacement therapy in the next 6 months.
    13. Any comorbidity or condition that, in the opinion of the Investigator, is anticipated to prevent participation in at least 12 months of the study
    14. Is not willing to comply with the contraceptive requirements during the study period, as described in study protocol.
    15. Female patient is pregnant, planning a pregnancy, or breast feeding.
    E.5 End points
    E.5.1Primary end point(s)
    Cohort A
    • Percent change in plasma oxalate from Baseline to Month 6

    Cohort B
    • Percent change in pre-dialysis plasma oxalate from Baseline to Month 6
    E.5.1.1Timepoint(s) of evaluation of this end point
    6 months
    E.5.2Secondary end point(s)
    Primary Analysis Period (Baseline to 6 months)
    • Percent change in plasma oxalate AUC between dialysis sessions
    • Absolute change in plasma oxalate
    • Change in the following parameters:
    *Urinary oxalate
    *QoL assessed by the PedsQL Total Score for patients ≥2 to <18 years of age at consent, and as assessed by KDQOL Burden of Kidney Disease and Effect of Kidney Disease on Daily Life subscales, and SF-12 Physical Component Summary and Mental Component Summary, in patients ≥18 years at consent
    • Plasma PK parameters of lumasiran

    Long-term Extension Period (6 months to end of study)
    • Percent change in plasma oxalate AUC between dialysis sessions
    • Percent and absolute change in plasma oxalate
    • Change in the following parameters:
    *Nephrocalcinosis as assessed by renal ultrasound
    *Frequency and mode of dialysis
    *Frequency of renal stone events
    *Urinary oxalate
    *Renal function as assessed by eGFR
    *Measures of systemic oxalosis in the following systems:
    -Cardiac
    -Dermatologic
    -Skeletal
    -Ocular
    *QoL(Quality of life) assessed by the PedsQL Total Score for patients ≥2 to <18 years of age at consent, and as assessed by KDQOL Burden of Kidney Disease and Effect of Kidney Disease on Daily Life subscales, and SF-12 Physical Component Summary and Mental Component Summary, in patients ≥18 years at consent
    E.5.2.1Timepoint(s) of evaluation of this end point
    Primary Analysis Period: 6 months
    Long-term Extension Period: 6 months to 5 years
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    Belgium
    Egypt
    France
    Germany
    India
    Israel
    Lebanon
    Netherlands
    Switzerland
    Turkey
    United Arab Emirates
    United States
    Jordan
    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 months4
    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: 6
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 14
    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
    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.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 8
    F.4.2.2In the whole clinical trial 20
    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 Decision2019-08-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-01-22
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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