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    Summary
    EudraCT Number:2021-001083-16
    Sponsor's Protocol Code Number:DCR-PHXC-203
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-23
    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 ConcernedSpain - AEMPS
    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
    Estudio de fase II abierto y multicéntrico para evaluar la seguridad, farmacocinética y eficacia de Nedosiran en pacientes pediátricos desde el nacimiento hasta los 5 años de edad con hiperoxaluria primaria y función renal relativamente intacta
    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 en pacientes pediátricos desde el nacimiento hasta los 5 años de edad con HP1, HP2 o HP3 y función renal relativamente intacta
    A.3.2Name or abbreviated title of the trial where available
    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 number0001617612 6275
    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.3.9.3Other descriptive nameNedosiran
    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 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
    Primary Hyperoxaluria
    Hiperoxaluria Primaria
    E.1.1.1Medical condition in easily understood language
    Primary Hyperoxaluria is where the liver overproduces oxalate. As a result calcium-oxalate crystals form in the urinary system and lead to the formation of kidney stones and impaired kidney function.
    Hiperoxaluria primaria se produce cuando el hígado sobreproduce oxalato. Como resultado los cristales de oxalato de calcio en el sistema urinario forman piedras en el riñon dañando la función renal
    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 assess the safety of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatinine
    Caracterizar la seguridad de Nedosiran en neonatos, lactantes y niños con HP y función renal relativamente intacta con base en la TFGe y la creatinina sérica
    E.2.2Secondary objectives of the trial
    1. To characterize the efficacy of nedosiran in neonates, infants, and young children with PH and relatively intact renal function based upon eGFR and serum creatin
    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 creatinineine
    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
    Evaluar la eficacia de Nedosiran en neonatos, lactantes y niños con HP y función renal relativamente intacta con base en la TFGe y la creatinina sérica
    Caracterizar la farmacinética de Nedosiran en neonatos, lactantes y niños con HP y función renal relativamente intacta con base en la TFGe y la creatinina sérica
    Evaluar la eficacia de Nedosiran en neonatos, lactantes y niños con HP y función renal relativamente intacta con base en la TFGe y la creatinina sérica
    Evaluar el efecto de Nedosiran en la TFGe en neonatos, lactantes y niños con HP y función renal relativamente intacta con base en la TFGe y la creatinina sérica
    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. Tasa de filtración glomerular estimada (TFGe) en la selección ≥ 30 mlL/min normalizada a 1,73 m2 de area de superficie corporal (ASC).
    2. Proporcion media de oxalato-creatinina de las muestras puntuales de orina en la selección 2 veces por encima del percentil 95 para la edad según Matos et al, 1999:
    > 0,44 mol / mol en participantes <6 meses
    > 0,34 mol / mol en participantes de 6 meses a <12 meses
    > 0,26 mol / mol en participantes de 12 meses a <2 años
    > 0,20 mol / mol en participantes de 2 a <3 años y
    > 0,16 mol / mol en participantes de 3 a 5 años
    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. Trasplante renal o hepático (previo o planificado dentro del período de estudio)
    2. Oxalato plasmático (Pox)> 30 µmol / L en la selección
    3.Evidencia documentada de manifestaciones clínicas de oxalosis sistémica grave (incluidas calcificaciones retinianas, cardíacas o cutáneas preexistentes, o antecedentes de dolor óseo severo, fracturas patológicas o deformaciones óseas)
    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
    La incidencia y las características de los acontecimientos adversos surgidos durante el tratamiento (AAST) y los acontecimientos adversos graves (AAG)
    Cambio desde el inicio en el ECG de 12 derivaciones, los hallazgos en las exploraciones físicas, las evaluaciones de las constantes vitales y los análisis clínicos de laboratorio
    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.
    Se proporcionarán estadísticas descriptivas para los valores absolutos y los cambios desde el inicio en los hallazgos del examen físico, mediciones de signos vitales, ECG y resultados de laboratorio clínico en cada punto temporal
    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 AUC∞ (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. Cambio porcentual y absoluto desde el inicio hasta el mes 6 en la proporción oxalato-creatinina de las muestras puntuales de orina
    2. Parámetros farmacocinéticos plasmáticos de Nedosiran y/o sus metabolitos, incluido la Cmáx, el área bajo la curva (AUC, en inglés) y el AUC∞ (si puede estimarse)
    3. Porcentaje de participantes con una proporción oxalato-cratinina de las muestras puntuales de orina ≤LSN y ≤1,5 x LSN en cualquier punto temporal hasta el mes 6
    4. Cambio desde el inicio en la TFGe en el mes 6 (solo en participantes ≥12 meses de edad en la selección)
    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. Se resumira el cambio porcentual y absoluto en la proporcion media de oxalato-creatinina de las muestras puntuales de orina hasta el dia 180
    2. Los analisis farmacocineticos seran descritos en el SAP finalizado antes dell cierre de la base de datos. El analisis farmacocineticao poblacional se presentara por separado al Informe final de resultados principal
    3. Se resumira el porcentaje de participantes con una proporción oxalato-cratinina de las muestras puntuales de orina ≤LSN y ≤1,5 x LSN en cualquier punto temporal hasta el mes 6
    4. Se resumira el cambio desde el inicio en la TFGe en el mes 6 (solo en participantes ≥12 meses de edad en la selección)
    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 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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 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
    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.
    Se considerara que un participante ha completado el estudio si el o ella ha completado todas las fases del estudio, incluida la última visita o el último procedimiento programado que se muestra en el calendario de actividades (SoA), Sección 1.3. El final del estudio se define como la finalización de la última visita o procedimiento que se muestra en el SoA del ensayo a nivel mundial.
    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) 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 to 5 years old
    Pacientes pediátricos desde el nacimiento hasta 5 años
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    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.
    Los participantes que completen satisfactoriamente el estudio DCR-PHXC-203 podrán ser seleccionados para participar en el estudio de transferencia (DCR-PHXC-301) en el dia 180, visita de fin de estudio
    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 Decision2021-08-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-08-03
    P. End of Trial
    P.End of Trial StatusOngoing
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