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    Summary
    EudraCT Number:2020-003308-14
    Sponsor's Protocol Code Number:Nef-301OLE
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-05-24
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2020-003308-14
    A.3Full title of the trial
    An Open-Label Extension (OLE) Study to Evaluate the Efficacy and Safety of Nefecon Treatment in Patients With IgA Nephropathy Who Have Completed Study Nef-301
    Studio di estensione in aperto (OLE) volto a valutare l’efficacia e la sicurezza del trattamento con Nefecon in pazienti con nefropatia da IgA i quali hanno completato lo studio Nef-301
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study in which Efficacy and Safety of Nefecon is Evaluated in Patients With IgA Nepropathy Who Have Completed Study Nef-301
    Studio nel quale l'efficacia e la sicurezza di Nefecon sono valutati in pazienti con nefropatia da IgA i quali hanno completato lo studio Nef-301
    A.3.2Name or abbreviated title of the trial where available
    -
    -
    A.4.1Sponsor's protocol code numberNef-301OLE
    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 SponsorCALLIDITAS THERAPEUTICS AB
    B.1.3.4CountrySweden
    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 supportCalliditas Therapeutics AB
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCalliditas Therapeutics AB
    B.5.2Functional name of contact pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street AddressKungsbron 1
    B.5.3.2Town/ cityStockholm
    B.5.3.3Post code11122
    B.5.3.4CountrySweden
    B.5.4Telephone number+46763949872
    B.5.6E-mailfredrik.juhlin@calliditas.se
    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 numberEMA/OP/139/16; EMA/COMP/513518/2016
    D.3 Description of the IMP
    D.3.1Product nameNefecon
    D.3.2Product code [Nefecon]
    D.3.4Pharmaceutical form Modified-release 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 INNBUDESONIDE
    D.3.9.1CAS number 51333-22-3
    D.3.9.2Current sponsor code-
    D.3.9.4EV Substance CodeSUB05955MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number4
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 IgA nephropathy patients at risk of developing end stage renal disease
    Pazienti con nefropatia IgA primaria a rischio di sviluppare una malattia renale allo stadio terminale
    E.1.1.1Medical condition in easily understood language
    Patients with kidney diseases that have damage to the kidneys associated with Immunoglobulin A
    Pazienti con malattie renali che hanno danni ai reni associato a immunoglobulina A
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10069341
    E.1.2Term Berger's disease
    E.1.2System Organ Class 100000004857
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    •To assess the effect of 9 months of retreatment with Nefecon on urine protein to creatinine ratio (UPCR) and estimated glomerular filtration rate (eGFR) in patients who completed Study Nef 301 with Nefecon treatment; and
    •To assess the effect of 9 months of treatment with Nefecon on UPCR and eGFR in patients who completed Study Nef-301 with placebo treatment.
    •Valutare l'effetto di 9 mesi di ritrattamento con Nefecon sul rapporto proteine urinarie/creatinina (UPCR) e sulla velocità di filtrazione glomerulare stimata (eGFR) nei pazienti che hanno completato lo studio Nef 301 con il trattamento con Nefecon; e
    •Valutare l'effetto di 9 mesi di trattamento con Nefecon su UPCR e eGFR in pazienti che hanno completato lo studio Nef-301 con il trattamento con placebo.
    E.2.2Secondary objectives of the trial
    •To assess the safety and tolerability of 9 months of retreatment with Nefecon in patients who completed Study Nef-301 with Nefecon treatment;
    •To assess the safety and tolerability of 9 months of treatment with Nefecon in patients who completed Study Nef-301 with placebo treatment;
    •To assess the effect of 9 months of retreatment with Nefecon on additional aspects of renal function in patients who completed Study Nef-301 with Nefecon treatment; and
    •To assess the effect of 9 months of treatment with Nefecon on additional aspects of renal function in patients who completed Study Nef-301 with placebo treatment.
    •Valutare la sicurezza e la tollerabilità di 9 mesi di ritrattamento con Nefecon in pazienti che hanno completato lo studio Nef-301 con il trattamento con Nefecon;
    •Valutare la sicurezza e la rollerabilità di 9 mesi di trattamento con Nefecon in pazienti che hanno completato lo studio Nef-301 con il trattamento con il placebo;
    •Valutare gli effetti di 9 mesi di ritrattamento con Nefecon su ulteriori aspetti della funzione renale nei pazienti che hanno completato lo studio Nef-301 con il trattamento con Nefecon; e
    •Valutare gli effetti di 9 mesi di trattamento con Nefecon su ulteriori aspetti della funzione renale nei pazienti che hanno completato lo studio Nef-301 con il trattamento con placebo.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Completed Study Nef-301, defined as Part A (9-month study drug treatment [Nefecon 16 mg/day or placebo] and 3-month follow-up) and Part B (12-month follow-up);
    -Completed Visit 17b in Study Nef-301 within 3 months before Study Visit 3;
    -On a stable dose of RAS inhibitor therapy (ACEIs and/or ARBs) at the maximum allowed dose or maximum tolerated dose according to the 2012 KDIGO guidelines21 (see Appendix C). A stable dose is defined as a dose within 25% of the dose at Visit 17a or 17b in Study Nef 301; and
    -Proteinuria based on 2 consecutive measurements (24-hour urine sampling) after informed consent, separated by at least 2 weeks and calculated by the central laboratory. Both samples of the same parameter must show either of the following: oProteinuria >/=1 g/day (>/=1000 mg/day) in 2 consecutive measurements; or oUPCR >/=0.8 g/gram (>/=90 mg/mmol) in 2 consecutive measurements; and
    -eGFR >/=30 mL/min per 1.73 m2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula confirmed by the central laboratory at Study Visit 1 or Study Visit 3.
    -Studio Nef-301 completato, definito come Parte A (trattamento farmacologico in studio di 9 mesi [Nefecon 16 mg/die o placebo] e follow-up di 3 mesi) e Parte B (follow-up di 12 mesi);
    -Visita 17b completata nello studio Nef-301 entro 3 mesi prima della Visita 3;
    -Su una dose stabile di terapia con inibitori della RAS (ACEIs e/o ARBs) alla dose massima consentita o alla dose massima tollerata in base alle linee guida 21 KDIGO del 2012 (vedere Appendice C). Una dose stabile è definita come una dose entro il 25% della dose alla Visita 17a o 17b nello studio Nef-301; e
    -Proteinuria basata su 2 misurazioni consecutive (prelievo di urina nelle 24 ore) dopo il consenso informato, separate da almeno 2 settimane e calcolate dal laboratorio centrale. Entrambi i campioni dello stesso parametro devono mostrare uno dei seguenti: oProteinuria >/=1 g/die (>/=1000 mg/die) in 2 misurazioni consecutive; o oUPCR >/=0.8 g/gram (>/=90 mg/mmol) in 2 misurazioni consecutive; e
    -eGFR >/=30 mL/min per 1.73 m2 utilizzando la formula Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) confermata dal laboratorio centrale alla Visita 1 o Visita 3 dello studio.
    E.4Principal exclusion criteria
    -Had a dose reduction to Nefecon 8 mg/day in Study Nef-301;
    -Systemic diseases that may cause mesangial IgA deposition including, but not limited to, Henoch Schönlein purpura, systemic lupus erythematosus, dermatitis herpetiformis, and ankylosing spondylitis;
    -Patients with nephrotic syndrome (i.e., proteinuria >3.5 g/day and with serum albumin <3.0 g/dL, with or without edema);
    -Patients with unacceptable blood pressure control defined as a blood pressure consistently above national guidelines for proteinuric renal disease, as assessed by the Investigator. Patients with >/=140 mmHg systolic blood pressure or >/=90 mmHg diastolic blood pressure are not eligible. At least 1 blood pressure measurement at either Study Visit 1 or Study Visit 3 should be within these limits (based on up to 3 measurements, measured 1 minute apart, after resting in the supine position for at least 5 minutes);
    -Patients who have received rescue therapy with systemic immunosuppressants, including GCSs, during Study Nef-301;
    -Patients who have been treated with any systemic GCSs within the 3 months before screening;
    -Patients who have been treated with any systemic GCSs within the 12 months before screening except for a maximum of 3 periods of 2 weeks with the equivalent of 0.5 mg/kg/day prednisolone or less for non-IgAN indications;
    -Somministrazione di una dose ridotta a 8 mg/die di Nefecon nello studio Nef-301;
    -Malattie sistematiche che possono causare il deposito mesangiali inclusi, ma non limitati a, porpora Henoch Schönlein, lupus eritematoso sistematico, dermatite erpetiforme e spondilite anchilosante;
    -Pazienti con sindrome nefrosica (ad esempio proteinuria>3.5 g/die e con albumina sierica <3.0 g/dL, con o senza edema);
    -Pazienti con un controllo della pressione sanguigna inaccettabile definita come pressione sanguigna costantemente al di sopra delle linee guida nazionali per la malattia renale proteinurica, come valutato dallo Sperimentatore. I pazienti con pression sanguigna sistolica >/=140 mmHg o pressione sanguigna diastolica >/= 90 mmHg non sono eleggibili. Almeno 1 misurazione della pressione sanguigna alla Visita 1 o alla Visita 3 dello studio dovrebbe rientrare in questi limiti (sulla base di un massimo di 3 misurazioni, misurate a 1 minuto di distanza, dopo essere stati a riposo in posizione supina per almeno 5 minuti);
    -Pazienti che hanno ricevuto una terapia di emergenza con immunosoppressori sistematici, inclusi GCSs, durante lo studio Nef-301;
    -Pazienti che sono stati trattati con eventuali GCSs sistematici nei 3 mesi precedenti lo screening;
    -Pazienti che sono stati trattati con eventuali GCSs sistematici nei 12 mesi precedenti lo screening ad eccezione di un massimo di 3 periodi di 2 settimane con l'equivalente di 0.5 mg/kg/die di prednisolone o meno per indicazioni non-IgAN;
    E.5 End points
    E.5.1Primary end point(s)
    •Ratio of eGFR at 9 months compared to baseline, calculated using the CKD-EPI formula; and
    •Ratio of UPCR at 9 months compared to baseline.
    •Rapporto di eGFR al nono mese rispetto al basale, calcolato utilizzando la formula CKD-EPI; e
    •Rapporto di UPCR al nono mese rispetto al basale.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Included in E.5.1
    Incluso in E.5.1
    E.5.2Secondary end point(s)
    •Ratio of urine albumin to creatinine ratio (UACR) at 9 months compared to baseline;
    •Short Form 36 quality of life assessment at 12 months compared to baseline;
    •Proportion of patients with microhematuria at 9 months compared to baseline;
    •Proportion of patients receiving rescue treatment and time to receiving rescue treatment;
    •Proportion of patients on dialysis, undergoing kidney transplantation, or with eGFR <15 mL/min per 1.73 m2; and
    •Cortisol suppression at 9 and 12 months, measured as urinary cortisol excretion over 24 hours compared to baseline.
    •Rapporto tra albumina urinaria e creatina (UACR) al 9° mese rispetto al basale;
    •Valutazione della qualità della vita Short Form 36 al 12° mese rispetto al basale;
    •Proporzione di pazienti con microematuria al 9° mese rispetto al basale;
    •Proporzione di pazienti che ricevono terapie di emergenza e tempi per ricevere trattamenti di emergenza;
    •Proporzione di pazienti in dialisi, sottoposti a trapianto di rene o con eGFR<15mL/min per 1.73 m2; e
    •Soppressione del cortisolo al mese 9 e 12, misurato come escrezione urinaria di cortisolo nell'arco di 24 ore rispetto al basale.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Included in E.5.2
    Incluso in E.5.2
    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 No
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 Yes
    E.8.1.7.1Other trial design description
    Open
    Open
    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.8.2.4Number of treatment arms in the trial1
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA66
    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 Information not present in EudraCT
    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
    Belarus
    Canada
    Korea, Republic of
    Taiwan
    Turkey
    United States
    Belgium
    Finland
    France
    Germany
    Greece
    Italy
    Poland
    Spain
    Sweden
    United Kingdom
    Czechia
    Argentina
    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
    Last visit of the last subject
    Last visit of the last subject
    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 months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 227
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 23
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 125
    F.4.2.2In the whole clinical trial 250
    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
    Nessuno
    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 Decision2020-11-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-10-06
    P. End of Trial
    P.End of Trial StatusOngoing
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