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    EudraCT Number:2016-002181-32
    Sponsor's Protocol Code Number:RG012-03
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2016-09-08
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2016-002181-32
    A.3Full title of the trial
    A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, Dose Selection, and Preliminary Efficacy of Weekly RG 012 Injections in Patients with Alport Syndrome
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A research study to evaluate the safety and effectiveness of RG-012 as a treatment for patients with Alport syndrome
    A.3.2Name or abbreviated title of the trial where available
    A Phase 2 Study to Evaluate Weekly RG012 Injections in Alport Syndrome
    A.4.1Sponsor's protocol code numberRG012-03
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02855268
    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 SponsorRegulus Therapeutics 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 supportRegulus Therapeutics Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRegulus Therapeutics Inc.
    B.5.2Functional name of contact pointMark A. Deeg
    B.5.3 Address:
    B.5.3.1Street Address10614 Science Center Drive
    B.5.3.2Town/ citySan Diego,
    B.5.3.3Post codeCA 92121
    B.5.3.4CountryUnited States
    B.5.4Telephone number001858.202.6343
    B.5.5Fax number001858.202.6343
    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/3/15/1451
    D.3 Description of the IMP
    D.3.2Product code RG-012
    D.3.4Pharmaceutical form 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.3.9.4EV Substance CodeAS1
    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. 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 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 placeboInjection
    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
    Alport syndrome is an inherited form of kidney disease caused by mutations in genes coding for the capillary basement membrane
    collagen IV
    E.1.1.1Medical condition in easily understood language
    Alport syndrome is a condition which causes damage to your kidneys over time
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Primary Objective:
    • To assess the safety and tolerability of RG-012 when administered weekly for 24 weeks
    E.2.2Secondary objectives of the trial
    Secondary Objectives:
    • To assess the effect of RG-012 on endogenous renal microRNA-21 (miR-21)
    • To assess the effect of RG-012 on other selected blood, urine, and renal biomarkers
    • To assess pharmacokinetic (PK) parameters of the parent compound (RG456070) and its active major metabolite (RG0005) following administration of RG-012
    • To assess potential formation of anti-drug antibodies (ADAs) following administration of RG 012
    • To assess the preliminary efficacy of RG 012 to reduce the decline in renal function over time

    Exploratory Objective:
    • To assess changes in quality of life (QoL) following administration of RG 012
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Capable of providing written informed consent
    2. Male or female subjects aged 18 to 65 years (inclusive)
    Capable of providing written informed consent
    3. Confirmed diagnosis of Alport syndrome (clinical, histopathologic, and/or genetic diagnosis)
    4. As estimated with the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine or creatinine–cystatin C equation, all 3 of the following eGFR criteria must be met:
    • eGFR at the initial screening measurement between 45 and 90 ml/min/1.73 m2
    • Declining eGFR at a rate ≥5ml/min/1.73m2 per year
    • Final screening/baseline eGFR >30ml/min/1.73m2
    5.Proteinuria ≥500 mg protein/g creatinine at initial screening and baseline visits
    6. On a stable dosing regimen of an ACE inhibitor and/or ARB for ≥30 days prior to screening
    7. Female subjects of childbearing potential must agree to use an acceptable methods of contraception from screening through 30 days after the last dose of study drug
    8. Male subjects with female partners of childbearing potential must agree to use one an acceptable contraception methods from the time of the first dose of study drug through 30 days after the last dose of study drug
    9. Negative drug screen for opiates, cocaine, heroin, phencyclidine, amphetamines (including ecstasy), barbiturates, and benzodiazepines
    10. Negative screening results for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, and human immunodeficiency virus (HIV) antibody
    11. Screening hematology, clinical chemistries, coagulation, and urinalysis are not clinically significant as assessed by the Investigator, and the following criteria are met:
    • Platelets >100x109/L
    • Total white blood cells >3.0x109/L and absolute neutrophil count >1.5x109/L
    • Hemoglobin >10 g/dL for females and >11 g/dL for males
    • Total and direct bilirubin <1.5x upper limit of normal (ULN), unless elevated bilirubin is associated with a known benign condition (e.g., Gilbert's syndrome)
    • Alanine aminotransferase (ALT) <3x ULN
    • Aspartate aminotransferase (AST) <3x ULN
    • Alkaline phosphatase (ALP) <3x ULN
    12. Agree not to start any new prescription medication and to maintain stable regimens of existing medications, except as prescribed or approved by the Investigator, or if required in an emergency
    13. Agree not to take any over-the-counter medication or herbal supplements during the study, except as instructed or approved by the Investigator
    14. Able to understand all study procedures in the informed consent form (ICF) and willing to comply with all aspects of the protocol
    E.4Principal exclusion criteria
    1. Causes of chronic kidney disease aside from Alport syndrome (including, but not limited to, diabetic nephropathy, hypertensive
    nephropathy, lupus, IgA nephropathy)
    2. ESRD as evidenced by ongoing dialysis therapy or history of renal transplantation
    3. Female subjects who are pregnant or lactating
    4. Concurrent clinically significant medical diagnosis (other than Alport
    syndrome) that would potentially interfere with the subject's study compliance or confound the study results, including but not limited to: significant or unstable cardiac disease (unstable angina, myocardial infarction within the last 6 months, coronary heart disease, congestive heart failure, prolonged QT syndrome [torsade de pointes], significant arrhythmia, and/or any other clinically significant ECG abnormalities); cancer; uncontrolled seizure disorder; chronic infection (e.g., tuberculosis); diabetes; and/or significant thyroid disease
    5. Any history of malignancy within the last 1 year (history of localized basal cell or squamous cell carcinoma that has been excised/appropriately treated is not considered exclusionary)
    6. Concurrent social conditions (e.g., drug or alcohol abuse) that would potentially interfere with the subject's study compliance
    7. Use of over-the-counter medications or herbal supplements that may interact with the bioavailability of the investigational product or use of alternative medicines that may have influence on the disease outcome.
    8. Mental disability or history of or current significant psychiatric disease that may impair ability to provide informed consent or impact compliance with study procedures
    9. Clinically significant illness within 30 days prior to screening that in the judgment of the Investigator could interfere with interpretation of study results, impair compliance with study procedures, or impact the safety of the subject
    10. Participation in a recent investigational study and receipt of an investigational drug or investigational use of a licensed drug within 30 days or 5 half lives, whichever is longer, prior to screening
    11. History or presence of hypersensitivity, idiosyncratic reaction, or allergic reaction to the study drug, inactive ingredients, or related compounds
    12. History of any clinically significant reaction (in the judgment of the Investigator) to previous injection of an oligonucleotide product
    13. History or presence of alcoholism or drug abuse within the past 2 years prior to screening
    14. Any other condition or circumstance that, in the opinion of the Investigator, may make the subject unlikely to complete the study or comply with study procedures and requirements, or may pose a risk to the subject's safety and well-being
    E.5 End points
    E.5.1Primary end point(s)
    Safety Endpoints:
    • Incidence of adverse events (AEs), serious AEs (SAEs), severe AEs,
    drug-related AEs, and AEs leading to withdrawal
    • Observed values and changes from baseline in clinical laboratory parameters (e.g., hematology, chemistry, complement, and urinalysis)
    • Observed values and changes from baseline in vital signs
    • 12-Lead electrocardiogram (ECG)
    • Physical examinations
    Pharmacodynamic Endpoints:
    • Change over time in renal biomarkers (including miR-21 in renal biopsy tissue, blood urea nitrogen [BUN], serum creatinine, urine protein/albumin, kidney injury molecule-1 [KIM 1], β-2 microglobulin, clusterin, cystatin C) and exploratory biomarkers (including, but not necessarily limited to, additional microRNAs, asymmetric dimethylarginine [ADMA], transforming growth factor-β [TGFβ], connective tissue growth factor [CTGF], neutrophil gelatinase-associated lipocalin [NGAL])
    Pharmacokinetic Endpoints:
    • Plasma concentrations and calculated PK parameters of the parent compound (RG456070) and its active metabolite (RG0005)
    • Incidence and titer of ADAs
    Efficacy Endpoint:
    • Change in eGFR from baseline to Week 24
    Exploratory Endpoint:
    ​​​​• Change over time in QoL as measured using the Short Form 36 Health Survey® (SF-36)
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 24 weeks of treatment
    E.5.2Secondary end point(s)
    Not applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    United Kingdom
    United States
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    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.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F. of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F. of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F. of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F. of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F. of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F. of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 27
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    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)
    Subjects completing 24 weeks of treatment will be eligible to screen for
    enrollment in an extension study (RG012-04) in which all subjects receive active treatment. For subjects who do not enter the open-label extension, there is no post treatment plan
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2016-11-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-12-07
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2017-08-30
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