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    The EU Clinical Trials Register currently displays   43801   clinical trials with a EudraCT protocol, of which   7272   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:2021-004192-13
    Sponsor's Protocol Code Number:R3R01-ASFSGS-201
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2022-05-19
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2021-004192-13
    A.3Full title of the trial
    A Phase II, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary Steroid-Resistant Focal Segmental Glomerulosclerosis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    R3R01 in Alport Syndrome Patients and Primary Steroid-Resistant Focal Segmental Glomerulosclerosis
    A.3.2Name or abbreviated title of the trial where available
    R3R01 in Alport Syndrome Patients and Primary Steroid-Resistant Focal Segmental Glomerulosclerosis
    A.4.1Sponsor's protocol code numberR3R01-ASFSGS-201
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05267262
    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 SponsorRiver 3 Renal, Corporation
    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 supportRiver 3 Renal, Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationR3R Corporation
    B.5.2Functional name of contact pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street Address7550 Purple Sage
    B.5.3.2Town/ cityPark City
    B.5.3.3Post codeUT 84098
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1 610-937-1932
    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 nameR3R01
    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 INNnot available
    D.3.9.1CAS number not availabl
    D.3.9.2Current sponsor codeR3R01
    D.3.9.3Other descriptive nameR3R01
    D.3.9.4EV Substance CodeSUB271579
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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
    Alport Syndrome (AS) and Primary Steroid-Resistent Focal
    Segmental Glomerulosclerosis (FSGS)
    E.1.1.1Medical condition in easily understood language
    Alport syndrome (AS) and Focal Segmental Glomerulosclerosis (FSGS). AS and FSGS are both a type of kidney disease.
    E.1.1.2Therapeutic area Not possible to specify
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10001843
    E.1.2Term Alport's syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    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
    Primary Objectives:
    For all patients:
    • To evaluate the tolerability and safety of R3R01 administered orally
    for 12 weeks.
    For AS patients:
    • To evaluate the efficacy of R3R01 in reducing proteinuria at 12 weeks
    in the whole AS group.
    For FSGS patients:
    • To evaluate the efficacy of R3R01 in reducing proteinuria at 12 weeks
    in the whole FSGS group.
    E.2.2Secondary objectives of the trial
    For all patients:
    •To evaluate improvement in quality of life as measured by the Short Form SF-36 for adults or the pediatric quality of life inventory
    (PedsQL) for children(ages 12-18)and their parents/legal guardians by assessing the change from baseline to end of treatment (Day 84)
    and end of the follow-up period(Day 168).
    •To evaluate the pharmacokinetics of R3R01.
    For AS patients:
    •To evaluate proteinuria complete response(UPCR<0.3g/g)at all
    timepoints proteinuria is measured.
    •To evaluate proteinuria partial response (decrease in proteinuria from baseline of ≥50%) at all timepoints proteinuria is measured.
    For FSGS patients:
    •To evaluate proteinuria complete remission(UPCR<0.3g/g) at all timepoints proteinuria is measured.
    Full text can be found in the protocol V5.0 dated14Jan2023, text was shortened due to character limit of this field.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    All Patients:
    1. Patient is able to communicate well with the investigator, understands and is willing to comply with all requirements of the study, and understands and signs the written informed consent form (ICF).
    2. For children to be eligible, one or both parents/legal guardians must sign a parental permission form which provides information contained in the ICF. Children capable of assent must express their willingness to participate by signing an assent form.
    3. If patient has received a COVID vaccination, the baseline visit must occur at least one week or more after the second/booster vaccination.
    4. Patients who have had active symptoms of COVID within 3 months prior to screening and are now asymptomatic for the last 2 weeks but have tested COVID PCR positive. If a patient is asymptomatic at screening but is COVID positive, then rescreening can occur after a minimum of two weeks.
    5. Both female patients, as well as female partners of male patients who are of child-bearing potential must be willing to not become pregnant for the complete duration of the study (>180 days) (90 days after the last dose of study medication).
    6. Males (including sterilized subjects) whose female partners have child-bearing potential, must agree to use male contraception (condoms) during the period from the time of signing the informed consent form (ICF) through 90 days after the last dose of study drug. They must agree to immediately inform the investigator if their partner becomes pregnant during the study.
    AS Inclusion Criteria (in addition):
    7. Males and females with X-Linked AS and males and females with autosomal inherited AS.
    a. For countries that are enrolling pediatric patients: patients from age 12 years and older.
    b. For countries that are not enrolling pediatric patients: patients from age 18 years and older.
    8. Confirmed diagnosis of AS by genetic testing and /or kidney biopsy. For patients enrolled in the US who meet all inclusion and exclusion criteria but have not had their diagnosis confirmed by genetic testing or kidney biopsy, the Sponsor will provide for patient’s genetic testing.
    9. UPCR ≥1.0 g/g.
    10. eGFR ≥ 45 mL/min/1.73m2 (using CKD-EPI equation for adults and Bedside Schwartz equation for children).
    11. ACEi/ARB therapy at maximum tolerated dose stable for at least 4 weeks prior to screening. ACEi/ARB dose should remain stable over the course of the study.
    FSGS Inclusion Criteria (in addition):
    12. Male or female patients,
    a. For countries that are enrolling pediatric patients: 12 to 75 years old at the time of signing the informed consent
    b. For countries that are not enrolling pediatric patients: 18 to 75 years old at the time of signing the informed consent
    13. Primary FSGS (without any identifiable cause, and where the FSGS is confirmed by renal biopsy) or FSGS where there is documentation of a genetic mutation in a podocyte protein associated with FSGS.
    14. Steroid-resistance defined as failure to achieve partial or complete remission, or experienced adverse events without acceptable clinical benefit after at least 8 weeks of adequate corticosteroid therapy for children and 12 weeks for adults.
    15. UPCR between 3.5g/g and 12.0g/g.
    16. eGFR > 45 mL/min/1.73m2 (using CKD-EPI equation for adults and Bedside Schwartz equation for children).
    17. If taking concomitant ACEi and/or ARB treatment, it should remain at a stable dose for a minimum of 28 days prior to enrollment and during the course of the study.
    E.4Principal exclusion criteria
    1. Uncontrolled diabetes mellitus as evidenced by an HbA1c ≥ 11%.
    For Germany: HbA1c ≥ 8.5%.
    2. Uncontrolled hypertension
    a. Adults: (SBP ≥ 180mmHg and/or DBP ≥ 100mmHg).
    For Germany: (SBP ≥ 140mmHg and/or DBP ≥ 100mmHg).
    b. Children: ≥ 95th percentile or ≥ 130/80 mm Hg, whichever is lower, as defined in Appendix 13.8.
    3. Moderate or severe hepatic impairment as per Child Pugh score (See Section 9.5.4.7), except if (a) decreased serum albumin is directly related to the renal disease (resulting in a Child Pugh score of 7), and (b) no other Child-Pugh Score parameters are increased and (c) patient has no liver pathology in medical history.
    4. Presence of any active (i.e., with symptoms) and/or uncontrolled infection (including COVID).
    5. Presence of Human immunodeficiency virus (HIV).
    6. BMI > 40. For Germany: BMI > 35 (Obesity Class II).
    7. History of malignancy other than treated basal cell or squamous cell skin cancer within the past 5 years.
    8. History of alcohol abuse in the last 5 years or currently drinks in excess of 21 and 14 units per week for males and females, respectively.
    9. Received an investigational agent within 30 days or 5 half-lives prior to screening (whichever is longer).
    10. History of non-compliance such that patient is unlikely to be compliant with study visits, procedures or drug administration.
    11. Patient has had an organ transplant, is currently on an organ transplant waiting list or there is a reasonable possibility that the patient will have an organ transplant in the 6 months after screening.
    12. Participation in an interventional trial within the previous 3 months prior to screening or concurrent participation in a research trial.
    13. Patient is not suitable to participate in the study for any reason (including, but not limited to co-morbidities, history of non-compliance with study visits, procedures, or drug administration) in the opinion of the investigator.
    14. Females of childbearing potential (those who are not surgically sterilized or post-menopausal for at least 1 year) are excluded from participation in the study unless they agree to use highly effective contraception as described in Section 13.3.
    15. Females that are lactating.
    16. History of hypersensitivity to study drug and/or any of its excipients.
    17. Patients with hereditary galactose intolerance, total lactase deficiency or glucose-galactose malabsorption.
    18. Required concomitant use of bardoxolone, rituximab, cyclo-phosphamide, abatacept, or sparsentan
    AS Exclusion Criteria (in addition):
    19. Kidney disease apart from AS, e.g., diabetic nephropathy or lupus nephritis.
    20. Use of Bardoxolone or sparsentan treatment in the 30 days prior to screening. SGLT2 inhibitors are allowed if the patient is on a stable dose for at least 3 months prior to screening.
    FSGS Exclusion Criteria (in addition):
    21. Patient has collapsing variant of FSGS on renal biopsy.
    22. Patient has FSGS secondary to another condition (e.g., obesity, cardiovascular, infectious, or autoimmune disorder).
    23. Use of Rituximab, cyclophosphamide or abatacept treatment in the 120 days prior to screening. If taking other chronic immune-modulatory medications that are small molecules, the dosage must be stable for 4 weeks prior to screening.
    24. If previous Rituximab treatment is greater than 120 days from screening, CD20 cell count should be within normal limits.
    25. If previous other antibody treatment on a stable dose is greater than 120 days from screening, the investigator must deem administration of study drug to be safe.
    26. Use of sparsentan in the 30 days prior to screening. SGLT2 inhibitors are allowed if the patient is on a stable dose for at least 3 months prior to screening.
    E.5 End points
    E.5.1Primary end point(s)
    Safety and tolerability endpoints will be assessed by occurrence of adverse events (AE), changes in physical examinations, vital signs, ECGs and clinical laboratory parameters. Efficacy endpoint is change from baseline in UPCR after 12 weeks of treatment.
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 weeks
    E.5.2Secondary end point(s)
    No secondary endpoints.
    E.5.2.1Timepoint(s) of evaluation of this end point
    No 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 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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    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 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 concerned2
    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
    United Kingdom
    United States
    Belgium
    France
    Germany
    Netherlands
    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
    LVLS
    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 Yes
    F.1.1Number of subjects for this age range: 15
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 15
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 32
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 50
    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)
    None
    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 Decision2022-07-25
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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