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    Summary
    EudraCT Number:2021-001083-16
    Sponsor's Protocol Code Number:DCR-PHXC-203
    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:2021-10-18
    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 number2021-001083-16
    A.3Full title of the trial
    A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Nedosiran in Pediatric Patients from Birth to 5 Years of Age with Primary Hyperoxaluria and Relatively Intact Renal Function
    Studio multicentrico di fase 2 in aperto per valutare la sicurezza, la farmacocinetica e l'efficacia di Nedosiran in pazienti pediatrici dalla nascita fino ai 5 anni di età con Iperossaluria Primaria e funzione renale relativamente intatta
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Nedosiran in Pediatric Patients from Birth to 5 Years of Age with PH1, PH2, or PH3 and Relatively Intact Renal Function
    Nedosiran in pazienti pediatrici dalla nascita fino ai 5 anni di età con PH1, PH2 o PH3 e funzione renale relativamente intatta
    A.3.2Name or abbreviated title of the trial where available
    PHYOX8
    PHYOX8
    A.4.1Sponsor's protocol code numberDCR-PHXC-203
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/071/2021
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorDicerna 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 supportDicerna Pharmaceuticals Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDicerna Pharmaceuticals Inc
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address75 Hayden Avenue
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number0016176126275
    B.5.6E-mailahenderson@dicerna.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/3/18/2052
    D.3 Description of the IMP
    D.3.1Product nameNedosiran
    D.3.2Product code [DCR-PHXC]
    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 INNDCR-L1360
    D.3.9.2Current sponsor codeDCR-PHXC
    D.3.9.4EV Substance CodeSUB190536
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number170
    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 Hyperoxaluria
    Iperossaluria primaria
    E.1.1.1Medical condition in easily understood language
    Primary hyperoxaluria is where the liver overproduces oxalate. As a result calciumoxalate crystals form in the urinary system and lead to the formation of kidney stones and impaired kidney function.
    L'iperossaluria primaria è dove il fegato produce eccesso di ossalato. Nel sistema urinario si formano cristalli di ossalato di calcio e e quindi calcoli renali e ridotta funzionalità renale.
    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
    To characterize the safety of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinine
    Caratterizzare la sicurezza di nedosiran in neonati, lattanti e bambini piccoli con PH e funzione renale relativamente intatta sulla base di eGFR e creatinina sierica.
    E.2.2Secondary objectives of the trial
    1. To assess the efficacy of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinine
    2. To characterize the PK of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinine
    3. To assess the efficacy of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinine
    4. To evaluate the effect of nedosiran on eGFR in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinine
    1. Valutare l'efficacia di nedosiran in neonati, lattanti e bambini piccoli con PH e funzione renale relativamente intatta sulla base della eGFR e della creatinina sierica.
    2. Caratterizzare la PK di nedosiran in neonati, lattanti e bambini piccoli con PH e funzione renale relativamente intatta sulla base della eGFR e della creatinina sierica.
    3. Valutare l'efficacia di nedosiran in neonati, lattanti e bambini piccoli con PH e funzione renale relativamente intatta sulla base della eGFR e della creatinina sierica.
    4. Valutare l'effetto di nedosiran sulla eGFR in neonati, lattanti e bambini piccoli con PH e funzione renale relativamente intatta sulla base della eGFR e della creatinina sierica.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Estimated glomerular filtration rate (eGFR) at Screening = 30 mL/min normalized to 1.73 m2 body surface area (BSA).
    2. Average spot Uox-to-creatinine ratio at Screening above 2 times the 95th percentile for age based on Matos et al, 1999:
    > 0.44 mol/mol in participants < 6 months
    > 0.34 mol/mol in participants from 6 months to <12 months
    > 0.26 mol/mol in participants 12 months to < 2 years
    > 0.20 mol/mol in participants from 2 to < 3 years and
    > 0.16 mol/mol in participants from 3 to 5 years
    1. Velocità di filtrazione glomerulare stimata (eGFR) allo screening =30 ml/min normalizzata a 1,73 m2 della superficie corporea (BSA).
    2. Rapporto medio Uox/creatinina a campione allo screening 2 volte superiore al 95° percentile per l'età basato su Matos et al, 1999:
    >0,44 mol/mol in partecipanti <6 mesi di età
    >0,34 mol/mol in partecipanti da 6 mesi a <12 mesi di età
    >0,26 mol/mol in partecipanti da 12 mesi a <2 anni di età
    >0,20 mol/mol in partecipanti da 2 anni a <3 anni di età e
    >0,16 mol/mol in partecipanti da 3 anni ai 5 anni di età
    E.4Principal exclusion criteria
    1. Renal or hepatic transplantation (prior or planned within the study period)
    2. Plasma oxalate (Pox) > 30 µmol/L at Screening
    3.Documented evidence of clinical manifestations of severe systemic oxalosis (including preexisting retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)
    1. Trapianto renale o epatico (effettuato in precedenza o previsto nel corso del periodo dello studio)
    2. Ossalato plasmatico (Pox) >30 µmol/l allo screening
    3. Prove documentate di manifestazioni cliniche di ossalosi sistemica grave (tra cui calcificazioni retiniche, cardiache o cutanee pre-esistenti oppure anamnesi di dolore osseo grave, fratture patologiche o deformazioni ossee)
    E.5 End points
    E.5.1Primary end point(s)
    - The incidence and nature of TEAEs and SAEs
    - Change from Baseline in 12-lead ECG, physical examination findings, vital sign assessments, and clinical laboratory tests
    - Incidenza e natura degli eventi avversi emergenti dal trattamento (EAET) e degli eventi avversi gravi (EAG)
    - Variazione rispetto al basale dell’elettrocardiogramma (ECG) a 12 derivazioni, risultati dell'esame obiettivo, segni vitali e test di laboratorio clinici
    E.5.1.1Timepoint(s) of evaluation of this end point
    Descriptive statistics will be provided for absolute values and changes from baseline in physical examination findings, vital sign measurements, ECGs, and clinical laboratory results at each timepoint
    Verranno fornite statistiche descrittive per i valori assoluti e le variazioni rispetto al basale nei risultati dell'esame obiettivo, misurazioni dei segni vitali, ECG e risultati di laboratorio clinico a ciascun timepoint
    E.5.2Secondary end point(s)
    1. Percent and absolute change from Baseline to Month 6 in spot urinary oxalate-to-creatinine ratio
    2. Plasma PK parameters for nedosiran and/or its metabolites, including Cmax, AUCt and AUC8 (if estimable)
    3. Percentage of participants with spot urinary oxalate-to-creatinine ratio = the ULN and = 1.5 x ULN at any time point through Month 6
    4. Change from Baseline in eGFR at Month 6 (only in participants = 12 Months of age at Screening)
    1. Variazione assoluta e percentuale nel rapporto ossalato/creatinina urinaria a campione, dal basale al Mese 6.
    2. I parametri farmacocinetici (PK) plasmatici per nedosiran e/o i suoi metaboliti, compresi Cmax, AUCt, e AUC8 (se calcolabile)
    3. Percentuale di partecipanti con rapporto ossalato-creatinina urinaria =ULN e =1,5 x ULN in qualsiasi momento fino al Mese 6.
    4. Variazione rispetto al basale nella eGFR al Mese 6 (solo per partecipanti di età =12 mesi al momento dello screening).
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. The percent and absolute change in average spot urinary oxalate-to-creatinine ratio from Baseline to Day 180 will be summarized
    2. Pharmacokinetic analyses will be described in the SAP finalized before database lock. The population PK analysis will be presented separately from the main CSR
    3. The percentage of participants with spot urinary oxalate-to-creatinine ratio = the ULN and = 1.5 x ULN at any time point through Month 6 will be summarized.
    4. The percent change from baseline in eGFR at Month 6 (only in participants = 12 Months of age at Screening) will be summarized.
    1. Verranno riassunte la variazione percentuale e assoluta del rapporto medio tra ossalato urinario e creatinina spot dal basale al giorno 180
    2. Le analisi farmacocinetiche saranno descritte nel SAP finalizzato prima del blocco del database. L'analisi PK di popolazione sarà presentata separatamente dalla CSR principale
    3. Verrà riassunta la percentuale di partecipanti con rapporto spot tra ossalato urinario e creatinina = ULN e = 1,5 x ULN in qualsiasi momento fino al mese 6.
    4. Verrà riassunta la variazione percentuale rispetto al basale in eGFR al mese 6 (solo nei partecipanti = 12 mesi di età allo screening).
    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 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) 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 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 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.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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 EEA9
    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
    Canada
    Japan
    Lebanon
    Morocco
    Turkey
    United States
    France
    Germany
    Italy
    Poland
    Spain
    United Kingdom
    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
    A participant is considered to have completed the study if he or she has completed all phases of the study, including the last visit or the last scheduled procedure shown in the Schedule of Activities (SoA), Section 1.3. The end of the study is defined as completion of the last visit or procedure shown in the SoA in the trial globally.
    Si considera che un partecipante abbia completato lo studio se ha completato tutte le fasi dello studio, inclusa l'ultima visita o l'ultima procedura programmata mostrata nel Programma delle attività (SoA), sezione 1.3. La fine dello studio è definita come il completamento dell'ultima visita o procedura indicata nella SoA nello studio a livello globale.
    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 days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    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 Yes
    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: 13
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    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 No
    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
    Pediatric patients from birth till 5 years old.
    Pazienti pediatrici dalla nascita ai 5 anni di età
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 7
    F.4.2.2In the whole clinical trial 15
    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)
    Participants successfully completing study DCR-PHXC-203 may be screened for entry into the roll-over study (DCR-PHXC-301) at the Day 180, EOS visit.
    I pazienti che avranno completato con successo lo studio DCR-PHXC-203 potranno essere screenati per entrare nello studio roll-over DCR-PHXC-3014 at giorno 180, visita EoS
    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 Decision2021-10-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-10-27
    P. End of Trial
    P.End of Trial StatusOngoing
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