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    Summary
    EudraCT Number:2020-002826-97
    Sponsor's Protocol Code Number:DCR-PHXC-204
    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:2020-12-08
    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 number2020-002826-97
    A.3Full title of the trial
    A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study for patients with Primary Hyperoxaluria and Severe Renal Impairment to evaluate the efficacy, safety and tolerability of DCR-PHXC
    A.3.2Name or abbreviated title of the trial where available
    PHYOX 7: Safety & Efficacy of DCR-PHXC in patients with PH1/2 and ESRD
    A.4.1Sponsor's protocol code numberDCR-PHXC-204
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04580420
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/426/2019
    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 pointTom Bowman, MD
    B.5.3 Address:
    B.5.3.1Street Address75 Hayden Ave.
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number0018572766954
    B.5.6E-mailTMBW@novonordisk.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.3.10 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 DCR-PHXC in lowering Pox in participants with PH1 or PH2 and severe renal impairment, with or without hemodialysis or peritoneal dialysis
    E.2.2Secondary objectives of the trial
    1. To assess the efficacy of DCR-PHXC in lowering Pox in participants with PH1 or PH2 and severe renal impairment, with or without hemodialysis or peritoneal dialysis
    2. To characterize the safety of DCR-PHXC in participants with PH1 or PH2 and severe renal impairment, with or without hemodialysis or peritoneal dialysis
    3. To assess the multiple dose PK of DCR PHXC and/or its metabolites in participants with PH1 or PH2 and severe renal impairment, with or without hemodialysis or peritoneal dialysis
    4. To assess the effect of DCR-PHXC on the hemodialysis or peritoneal dialysis clearance of participants with PH1 or PH2 undergoing hemodialysis or peritoneal dialysis
    5. To evaluate the effect of DCR PHXC on stone burden in participants with PH1 or PH2 and severe renal impairment, with or without hemodialysis or peritoneal dialysis
    6. To evaluate the effect of DCR PHXC on nephrocalcinosis score
    7. To evaluate the effect of DCR PHXC on cardiac oxalosis
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    In order to be eligible to participate in this study, an individual must meet all of the following criteria:
    Age
    1. Four age groups of participants will be enrolled, in sequence:
    a. adults and adolescents (aged ≥ 12 years)
    b. children 6 to 11 years of age
    c. children 2 to 5 years of age and
    d. infants and newborns from birth to < 2 years of age.
    Type of Participant and Disease Characteristics
    2. Documented diagnosis of PH1 or PH2, confirmed by genotyping (historically available genotype information is acceptable for study eligibility)
    3. Estimated GFR at Screening < 30 mL/min normalized to 1.73 m2 BSA. See Section 8.2.6.2 for equations. For infants aged less than 12 months, serum creatinine above the 97.5th percentile of a healthy population (Boer et al., 2010)
    4. Mean of 2 plasma oxalate values > 20 µmol/L during Screening
    5. For participants receiving hemodialysis or peritoneal dialysis, total duration of hemodialysis or peritoneal dialysis must be less than or equal to 18 months and hemodialysis or peritoneal dialysis regimen must have been stable for at least 2 weeks prior to Screening.
    Weight
    6. Body weight of:
    a. adults and adolescents aged ≥ 12 years: ≥ 31.0 kg
    b. children 6 to 11 years of age: ≥ 12 kg
    c. children 2 to 5 years of age: to be determined
    d. infants and newborns from birth to < 2 years of age: to be determined
    Sex
    7. Male or female
    Male participants:
    A male participant with a female partner of childbearing potential must agree to use contraception, as detailed in Section 10.4.2, 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 10.4.3), not breastfeeding, and at least one of the following conditions applies:
    Not a woman of childbearing potential (WOCBP) as defined in Section 10.4.1.1.
    OR
    A WOCBP who agrees to follow the contraceptive guidance in Section 10.4.2.2 for the 4 weeks prior to randomization, during the treatment period, and for at least 12 weeks after the last dose of study intervention and agrees to refrain from harvesting/freezing eggs during this period.
    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/Assent
    8. Participant (and/or participant’s parent or legal guardian if participant is a minor [defined as patient < 18 years of age, or younger than the age of majority according to local regulations]) is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
    a. Adolescents (12 to < 18 years of age, or older than 12 years but younger than the age of majority according to local regulations) must be able to provide written assent for participation.
    b. For children younger than 12 years of age, assent will be based on local regulations.
    Other
    9. Affiliated with or is a beneficiary of a health insurance system (if applicable per national regulations)
    E.4Principal exclusion criteria
    An individual who meets any of the following criteria will be excluded from participation in this study:
    Medical Conditions
    1. Prior hepatic transplantation; or scheduled transplantation within 6 months of Day 1. Renal transplantation planned in the 6 months from Day 1. Prior renal transplantation is allowed.
    2. 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)
    3. Presence of any condition or comorbidities that would interfere with study compliance or data interpretation or potentially impact patient safety including, but not restricted to:
    a. Severe intercurrent illness
    b. Known causes of active liver disease/injury (e.g., alcoholic liver disease, nonalcoholic fatty liver disease/steatohepatitis)
    c. Non-PH related conditions contributing to renal insufficiency
    d. Physician concerns about intake of drugs of abuse or excessive alcohol intake, or history of excessive alcohol intake in the 2 years prior to enrollment (defined as ≥ 21 units of alcohol per week in men and ≥ 14 units of alcohol per week in women; where a “unit” of alcohol is equivalent to a 12-ounce beer, 4-ounce glass of wine, or 1 ounce shot of hard liquor)
    Prior/Concomitant Therapy
    4. Use of an RNAi drug, other than DCR-PHXC, within the last 6 months
    5. History of one or more of the following reactions to an oligonucleotide-based therapy:
    a. Severe thrombocytopenia (platelet count ≤ 100,000/µL)
    b. Hepatotoxicity, defined as alanine transaminase (ALT) or aspartate transaminase (AST) > 3 times the upper limit of normal (ULN) and total bilirubin > 2 × ULN or international normalized ratio (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
    Prior/Concurrent Clinical Study Experience
    6. Participation in any clinical study in which they received an investigational medicinal product (IMP) other than DCR-PHXC within 4 months or 5 times the half-life of the drug (whichever is longer) before Screening.
    Diagnostic Assessments
    7. Liver function test abnormalities: ALT and/or AST >1.5 × ULN for age and gender
    8. Positive anti-double-stranded deoxyribonucleic acid (anti-dsDNA) antibody test at Screening
    Other Exclusions
    9. Known hypersensitivity to DCR-PHXC or any of its ingredients
    10. 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)
    Absolute and percent change in Pox from Baseline to Day 180 in PH1 or PH2 participant subgroups
    E.5.1.1Timepoint(s) of evaluation of this end point
    Efficacy analyses will be performed on the MITT Population and the Evaluable Population. Sensitivity analysis and subgroup analysis will be explored for the primary endpoint. The primary endpoint evaluation will be based on the absolute and percent change in Pox from Baseline to Day 180 summarized by PH1 or PH2 participant subgroups.
    E.5.2Secondary end point(s)
    1. Absolute and percent change in Pox from Baseline to maximum reduction through Day 180 in PH1 or PH2 participant subgroups
    2. Incidence and severity of TEAEs and SAEs, and the change from Baseline in 12-lead ECG, physical examination findings, vital signs, and clinical laboratory tests
    3. Plasma PK parameters for DCR-PHXC and/or its metabolites (e.g., Cmax and AUC, based on NCA or POP PK analysis)
    4. The change in the dialysis clearance from Baseline to Month 12 and quarterly thereafter in the long-term extension in PH1 or PH2 participant subgroups
    5. Percent change in the summed surface area and number of kidney stones identified via ultrasound from Baseline to Day 180 in those in whom paired ultrasounds are available in PH1 or PH2 participant subgroups
    6. Change in nephrocalcinosis scores over time in PH1 or PH2 participant subgroups
    7. Change in cardiac oxalosis as determined by changes in cardiac strain over time in PH1 or PH2 participant subgroups
    E.5.2.1Timepoint(s) of evaluation of this end point
    The secondary endpoints evaluation will be summarized by PH1 or PH2 participant subgroups:
    - The absolute and percent change in Pox from Baseline to maximum reduction through Day 180
    - The change in the dialysis clearance from Baseline to Month 12 and quarterly thereafter in the long-term extension
    - The percent change in the summed surface area and number of kidney stones identified via ultrasound from Baseline to Day 180 in those in whom paired ultrasounds are available.
    - The change in nephrocalcinosis scores over time.
    - The change in cardiac oxalosis as determined by changes in cardiac strain over time.
    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 Yes
    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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 concerned2
    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
    Lebanon
    Morocco
    United Arab Emirates
    United States
    France
    Germany
    Italy
    Spain
    United Kingdom
    Romania
    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 the last scheduled visit (Month 42) as shown in Table 2 of the protocol.
    The end of the study overall is defined by the locking of the clinical database.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months8
    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: 21
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 2
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 3
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 7
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 17
    F.4.2.2In the whole clinical trial 30
    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 the Day 180 visit will enter the extended follow-up period and continue to receive open-label DCR-PHXC for an additional 3 years, or until DCR PHXC is commercially available to this patient population, whichever comes first.
    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 Decision2020-12-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-04-20
    P. End of Trial
    P.End of Trial StatusOngoing
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