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    Summary
    EudraCT Number:2016-005141-23
    Sponsor's Protocol Code Number:021FSGS16010
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2018-06-25
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2016-005141-23
    A.3Full title of the trial
    A Randomized, Multicenter, Double-blind, Parallel, Active-control Study of the Effects of Sparsentan, a Dual Endothelin Receptor and Angiotensin Receptor Blocker, on Renal Outcomes in Patients with Primary Focal Segmental Glomerulosclerosis (FSGS)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Effectiveness and Safety of Sparsentan as treatment for Primary Focal Segmental Glomerulosclerosis (FSGS)
    A.4.1Sponsor's protocol code number021FSGS16010
    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 SponsorTravere 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 supportTravere Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTravere Therapeutics, Inc.
    B.5.2Functional name of contact pointTravere Call Center
    B.5.3 Address:
    B.5.3.1Street Address3611 Valley Centre Drive, Suite 300
    B.5.3.2Town/ citySan Diego, CA
    B.5.3.3Post code92130
    B.5.3.4CountryUnited States
    B.5.4Telephone number+18776595518
    B.5.6E-mailmedinfo@travere.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/3/15/1574
    D.3 Description of the IMP
    D.3.1Product nameSparsentan/Over-encapsulated Sparsentan tablet
    D.3.2Product code RE-021
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSparsentan
    D.3.9.1CAS number 254740-64-2
    D.3.9.2Current sponsor codeRE-021
    D.3.9.3Other descriptive nameSPARSENTAN
    D.3.9.4EV Substance CodeSUB168607
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name Irbesartan tablets
    D.2.1.1.2Name of the Marketing Authorisation holderMilipharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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 nameover-encapsulated Irbesartan Tablets
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNIrbesartan
    D.3.9.1CAS number 138402-11-6
    D.3.9.3Other descriptive nameIRBESARTAN
    D.3.9.4EV Substance CodeSUB08293MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number75
    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.IMP: 3
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name Irbesartan tablets
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 nameover-encapsulated Irbesartan Tablets
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNIrbesartan
    D.3.9.1CAS number 138402-11-6
    D.3.9.3Other descriptive nameIRBESARTAN
    D.3.9.4EV Substance CodeSUB08293MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number75
    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.IMP: 4
    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/3/15/1574
    D.3 Description of the IMP
    D.3.1Product nameSparsentan
    D.3.2Product code RE-021
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSparsentan
    D.3.9.1CAS number 254740-64-2
    D.3.9.2Current sponsor codeRE-021
    D.3.9.3Other descriptive nameSPARSENTAN
    D.3.9.4EV Substance CodeSUB08293MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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.IMP: 5
    D.1.2 and D.1.3IMP RoleComparator
    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 Yes
    D.2.1.1.1Trade name Irbesartan tablets
    D.2.1.1.2Name of the Marketing Authorisation holderCamber Pharmaceuticals Inc.
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 nameOver-encapsulated Irbesartan Tablets
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNIrbesartan
    D.3.9.1CAS number 138402-11-6
    D.3.9.3Other descriptive nameIRBESARTAN
    D.3.9.4EV Substance CodeSUB08293MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number75
    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
    Focal segmental glomerulosclerosis (FSGS)
    E.1.1.1Medical condition in easily understood language
    Scarring of the tissue in the filtering unit of the kidney (glomerulus).
    E.1.1.2Therapeutic area Diseases [C] - Symptoms and general pathology [C23]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10067757
    E.1.2Term Focal segmental glomerulosclerosis
    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
    The efficacy objective of the study is to determine the long-term nephroprotective potential of treatment with sparsentan as compared with an ARB in patients with primary and genetic FSGS.

    The safety objective of the study is to assess the safety and tolerability of sparsentan by double-blind monitoring of safety endpoints.

    The open-label objective of the study is to assess the long-term efficacy,
    safety, and tolerability of open label sparsentan in patients with FSGS.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. The patient or parent/legal guardian (as appropriate) is willing and
    able to provide signed informed consent/assent
    2. Biopsy-proven primary FSGS or documentation of a genetic mutation in a podocyte protein associated with FSGS. The biopsy may have been
    performed at any time in the past. The patient will be enrolled based on
    light microscopy diagnosis of FSGS and supportive findings on either
    electron microscopy (EM) or immunofluorescence (IF) analysis
    (preferably both). In exceptional cases, the patient may be enrolled
    based on light microscopy diagnosis of FSGS lesion(s) in the absence of
    EM or IF analysis, provided the history and/or the course of the disease
    are indicative of primary FSGS and the case has been reviewed by the
    Medical Monitor and Investigator.
    3. Male or female aged 18 to 75 years, inclusive weighting at least 20kgat screening (Note: patients under 18 may be recruited only in United States and United Kingdom).
    4. UP/C ≥1.5 g/g (170 mg/mmol) at screening.
    5. eGFR ≥30 mL/min/1.73 m2 at screening.
    6. Mean seated blood pressure ≥100/60 mmHg and ≤160/100 mmHg.
    7. WOCBP agree to the use of contraception and pregnancy testing as described in the protocol.

    Inclusion Criteria for the Open-Label Extension:
    Based on assessments at the Week 108 visit, a patient will meet all of the following criteria to be eligible for the open-label extension.
    1.The patient completed participation in the double-blind period, including the Week 112 visit.
    2.The patient or parent/legal guardian (as appropriate) is willing and able to provide signed informed consent for participation in the open label extension.
    3.The patient received blinded study medication throughout the duration of the double blind period (ie, did not permanently discontinue study medication).
    E.4Principal exclusion criteria
    1. FSGS secondary to another condition.
    2. Positive findings on serological tests of primary or secondary glomerular injury.
    3. History of type 1 diabetes mellitus, uncontrolled type 2 diabetes mellitus (hemoglobin A1c [HbA1c] >8%), or nonfasting blood glucose >180 mg/dL (10.0 mmol/L) at screening.
    4. Any organ transplantation, with the exception of corneal transplants.
    5. Treatment with any of the prohibited concomitant medications.
    6. Treatment with rituximab, cyclophosphamide, or abatacept within ≤3 months prior to screening. If a patient is taking other chronic immunosuppressive medications, the dosage must be stable for ≥1 month prior to randomization.
    7. Documented history of heart failure and/or previous hospitalization for heart failure or unexplained dyspnea, orthopnea, paroxysmal nocturnal dyspnea, ascites, and/or peripheral edema.
    8. Clinically significant cerebrovascular disease and/or coronary artery disease.
    9. Hemodynamically significant valvular disease.
    10. Jaundice, hepatitis, or known hepatobiliary disease (excluding asymptomatic cholelithiasis), or transaminase levels >2 times the upper limit of the normal range at screening.
    11. Positive at screening for the human immunodeficiency virus (HIV) or markers indicating acute or chronic hepatitis B (HBV) infection or hepatitis C virus (HCV) infection.
    12. History of malignancy other than adequately treated basal cell or squamous cell skin cancer or cervical carcinoma within the past 2 years.
    13. A screening hematocrit value <27% (0.27 L/L) or hemoglobin value <9 g/dL (90g/L).
    14. A screening potassium value of >5.5 mEq/L (5.5 mmol/L)
    15. Body mass index (BMI) >40 and there is a casual relationship to the FSGS lesion.
    16. History of alcohol or illicit drug use, or excessive alcohol intake (>21 units per week within 2 years of screening)
    17. History of serious side effect or allergic response to any Ang II antagonist or ERA or hypersensitivity to any of the excipients.
    18. Female patients is pregnant, breastfeeding, or planning to conceive during the study.
    19. Participation in a study of another investigational product within 28 days prior to screening.
    20. Prior exposure to sparsentan.
    21. Unable to adhere to the requirements of the study, including swallowing the study medication capsules whole.

    Exclusion Criteria for the Open-Label Extension:
    Based on assessments at the Week 108 and Week 112 visits, a patient
    who meets any of the following criteria will be excluded from the open
    label
    extension.
    1.The patient has progressed to ESRD requiring RRT.
    2.The patient developed criteria for discontinuation as defined in Section
    6.4.2 or Section 6.5 between Week 108 and Week 112.
    3.The patient was unable to initiate, or developed contraindications to,
    treatment with RAAS inhibitors between Week 108 and Week 112.
    4.The patient has an eGFR =20 mL/min/1.73 m2 at Week 108. NOTE: If,
    in the Investigator's opinion, the eGFR value at Week 108 is deemed
    unlikely to be representative of the patient's true status, the
    Investigator may repeat the eGFR measurement prior to Week 112
    through the central laboratory to assess patient eligibility. Patients with
    an eGFR <30 mL/min/1.73 m2 will require close monitoring of eGFR and
    serum potassium throughout the open-label extension.
    5. The female patient is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy End points:
    The primary efficacy endpoint is the slope of eGFR over approximately 2 years of randomized treatment assessed at the final analysis.

    The surrogate efficacy endpoint is the proportion of patients achieving a target reduction in proteinuria.

    Safety End points:
    • Descriptive statistics will be used to summarize the safety data.

    Open-Label Endpoints:
    Endpoints for the open-label extension include, but are not necessarily
    limited to:
    •The absolute and percent change from Week 112 in eGFR at each visit
    •The percent change from Week 112 in UP/C at each visit
    •Changes from Week 112 in QOL at each visit
    •Changes from Week 112 in body weight, vital signs, physical
    examinations, peripheral edema, and clinical laboratory parameters
    •Changes from Week 112 in lipid profile (total cholesterol and
    triglycerides, LDL-C, and HDL C
    •The incidence of TEAEs during the open-label extension
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 36 and Week 108 or as defined above
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints in non-US countries are:
    • The percent change from Week 6 in eGFR at Week 108
    • The percent change in eGFR from baseline of the double-blind period to 4 weeks post-cessation of randomized treatment at Week 112
    • The slope of eGFR following the initiation of randomized treatment (ie, from Day 1 to Week 108; total slope over 2 years)
    The secondary efficacy endpoints in the US are:
    • The slope of eGFR following the initial acute effect of randomized treatment (ie, from Week 6 to Week 108; chronic slope over 2 years)
    • The change in eGFR from baseline of the double-blind period to 4 weeks post-cessation of randomized treatment at week 112
    E.5.2.1Timepoint(s) of evaluation of this end point
    Weeks 108 and 112
    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 No
    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 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) Yes
    E.8.2.2Placebo No
    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 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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA128
    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
    Argentina
    Australia
    Belgium
    Brazil
    Canada
    Croatia
    Czechia
    Denmark
    Estonia
    France
    Germany
    Hong Kong
    Italy
    Korea, Republic of
    New Zealand
    Poland
    Portugal
    Spain
    Sweden
    Taiwan
    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
    LVLS
    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 months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months1
    E.8.9.2In all countries concerned by the trial days14
    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: 60
    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: 30
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 30
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 180
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 60
    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 140
    F.4.2.2In the whole clinical trial 300
    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)
    The Investigator should resume standard of care treatment, including treatment with appropriate medications, as deemed appropriate. Patients who complete the study may be eligible to enroll in a separate open-label extension study.
    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 Decision2018-11-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-07-11
    P. End of Trial
    P.End of Trial StatusOngoing
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