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    The EU Clinical Trials Register currently displays   43876   clinical trials with a EudraCT protocol, of which   7294   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:2021-001083-16
    Sponsor's Protocol Code Number:DCR-PHXC-203
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-02
    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 ConcernedGermany - BfArM
    A.2EudraCT number2021-001083-16
    A.3Full title of the trial
    A Phase 2 Open-Label Multicenter Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Nedosiran in Pediatric Patients from Birth to 11 Years of Age with Primary Hyperoxaluria and Relatively Intact Renal Function
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Nedosiran in Pediatric Patients from Birth to 11 Years of Age with PH1, PH2, or PH3 and Relatively Intact Renal Function
    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/007/2023
    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 pointCatherine Sullivan
    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 number+19195932121
    B.5.5Fax number+16176126298
    B.5.6E-mailcsullivan@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
    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.
    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 efficacy of nedosiran in neonates, infants, and children with PH and relatively intact renal function based upon eGFR and serum creatinine
    E.2.2Secondary objectives of the trial
    1. To characterize the safety of nedosiran in neonates, infants, and 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 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 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 children with PH and relatively intact renal function based upon eGFR and serum creatinine
    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. Documented diagnosis of PH1, PH2, or PH3 confirmed by genotyping (historically available genotype information is acceptable for study eligibility)
    3. 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
    > 0.14 mol/mol in participants from 5 to < 7 years
    > 0.12 mol/mol in participants from 7 to 11 years
    4. Estimated glomerular filtration rate (eGFR) at Screening ≥ 30 mL/min normalized to 1.73 m2 body surface area (BSA). For infants aged less than 12 months, serum creatinine below the 97.5th percentile of a healthy population (Boer et al., 2010)
    5. Participants must have been on a stable treatment regimen for PH for 3 months prior to Day 1 and parent(s)/legal guardian should be willing to ensure participant remains on the same stable treatment regimen during the study. Dose adjustments for interval weight gain are acceptable.
    6.Male or female
    Male participants:
    A male participant with a female partner of childbearing potential must agree to use contraception, as detailed in Section 10.5.2, during the treatment period and for at least 12 weeks after the last dose of study intervention and refrain from donating sperm during this period.
    Female participants:
    A female participant is eligible to participate if she is not pregnant (see Section 10.5.1), not breastfeeding, and at least 1 of the following conditions applies:
    Not a woman of childbearing potential (WOCBP) as defined in Section 10.5.1 OR A WOCBP who agrees to follow the contraceptive guidance in Section 10.5.2 during the treatment period and for at least 12 weeks after the last dose of study intervention. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
    Informed Consent and Assent:
    7. Participant's parent or legal guardian is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
    a.For children younger than 12 years of age, assent will be based on local regulation. If assent is required, participant must be able to provide written assent for participation.
    Other
    8. A legal guardian or primary caregiver must be available to help the study-site personnel ensure follow up; accompany the participant to the study site on each assessment day according to the SoA (e.g., able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures); consistently and consecutively be available to provide information on the participant using the rating scales during the scheduled study visits; accurately and reliably dispense study intervention as directed.
    9. Affiliated with or is a beneficiary of a health insurance system (if applicable per national regulations)
    E.4Principal exclusion criteria
    1. Prior renal or hepatic transplantation; or planned transplantation within the study period
    2. Currently receiving dialysis or anticipating requirement for dialysis during the study period
    3.Plasma oxalate (Pox) > 30 μmol/L at Screening
    4.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)
    5.Presence of any condition or comorbidities that would interfere with study compliance or data interpretation or potentially impact participant’s safety including, but not restricted to:
    a.Severe intercurrent illness
    b.Known causes of active liver disease/injury or transaminase elevation (e.g., alcoholic liver disease, nonalcoholic fatty liver disease/steatohepatitis [NAFLD/NASH])
    c.History of serious mental illness that includes, but is not limited to, schizophrenia, bipolar disorder, or severe depression requiring hospitalization or pharmacological intervention
    d.Clinically relevant history or presence of cardiovascular, respiratory, gastrointestinal, hematological, lymphatic, neurological,
    musculoskeletal, genitourinary, immunological diseases, including dermatological including rash, severe eczema or dermatitis, or
    connective tissue diseases or disorders
    6.Use of an RNAi drug within the last 6 months
    7.History of 1 or more of the following reactions to an oligonucleotidebased therapy:
    a.Severe thrombocytopenia (platelet count ≤ 100,000/μL)
    b.Hepatotoxicity, defined as ALT or AST > 3 times the upper ULN and total bilirubin > 2 × ULN or INR > 1.5
    c.Severe flu-like symptoms leading to discontinuation of therapy
    d.Localized skin reaction from the injection (graded severe) leading to discontinuation of therapy
    e.Coagulopathy/clinically significant prolongation of clotting time
    8.Participation in any clinical study in which they received an IMP within 4 months or 5 times the half-life of the drug (whichever is longer) before Screening
    a.For IMPs with the potential to reduce urine and/or plasma oxalate concentrations, these concentrations must have returned to historical baseline levels prior to Screening
    9.Liver function test (LFT) abnormalities: ALT and/or AST > 1.5 × ULN for age and gender
    10.Known hypersensitivity to nedosiran, or any of its ingredients
    11.Participants who are suitable for, and have access to, another approved PH1 treatment
    12.Inability or unwillingness to comply with the specified study procedures, including the lifestyle considerations detailed in Section 5.3.
    E.5 End points
    E.5.1Primary end point(s)
    Percent and absolute change from Baseline to Month 6 in spot urinary oxalate-to-creatinine ratio in PH1, PH2, or PH3 participant subgroups
    E.5.1.1Timepoint(s) of evaluation of this end point
    The percent and absolute change in average spot urinary oxalate-tocreatinine ratio from Baseline to Day 180 will be summarized
    E.5.2Secondary end point(s)
    1. 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
    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 or ≤ 1.5 x ULN at any time point through Month 6 in PH1, PH2, or PH3 participant subgroups
    4. Change from Baseline in eGFR at Month 6 (only in participants ≥ 12 Months of age at Screening) in PH1, PH2, or PH3 participant subgroups
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. 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
    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.
    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 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 EEA5
    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
    Germany
    Italy
    Japan
    Lebanon
    Poland
    Spain
    Turkey
    United Kingdom
    United States
    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.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    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: 25
    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: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 20
    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 11 years old.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 25
    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.
    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 Decision2021-09-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-10-04
    P. End of Trial
    P.End of Trial StatusOngoing
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