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    Summary
    EudraCT Number:2015-003142-51
    Sponsor's Protocol Code Number:DCR-PH1-101
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-11-17
    Trial results View 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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2015-003142-51
    A.3Full title of the trial
    A Phase 1 Study of DCR-PH1 in Patients with Primary Hyperoxaluria Type 1 (PH1)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Early Phase Study of DCR-PH1 in Patients with an Inherited Disorder Resulting in Overproduction of Oxalate
    A.4.1Sponsor's protocol code numberDCR-PH1-101
    A.5.4Other Identifiers
    Name:P-IND NumberNumber:122598
    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 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 Operations
    B.5.3 Address:
    B.5.3.1Street Address87 Cambridgepark Drive
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02140
    B.5.3.4CountryUnited States
    B.5.4Telephone number0016176126253
    B.5.5Fax number0016176126298
    B.5.6E-mailbfielman@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/3/15/1528
    D.3 Description of the IMP
    D.3.1Product nameDCR-PH1
    D.3.4Pharmaceutical form Concentrate for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot Yet Assigned
    D.3.9.1CAS number NA
    D.3.9.2Current sponsor codeDCR-1171X
    D.3.9.3Other descriptive nameDCR-1171X
    D.3.9.4EV Substance CodeSUB178429
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2.0
    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 Type 1
    E.1.1.1Medical condition in easily understood language
    Inherited Disorder Resulting in Overproduction of Oxalate
    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 18.1
    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 determine the safety and tolerability of DCR-PH1 administered via IV infusion to patients with PH1.
    E.2.2Secondary objectives of the trial
    To study the pharmacokinetics of DCR-PH1

    To study the pharmacodynamic effects of DCR-PH1 including, but not limited to changes in plasma and urine oxalate and glycolate levels
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female, at least 12 years of age at the time of obtaining informed consent.
    2. Diagnosis of PH1, confirmed by genotyping for homozygosity or compound heterozygosity in the AGXT gene (historically available genotype information is acceptable for study eligibility).
    3. 24-hour urine oxalate excretion ≥ 0.7 mmol per 1.73 m2 BSA.
    4. eGFR ≥ 40 mL/min normalized to 1.73 m2 BSA calculated using the Modification of Diet in Renal Disease (MDRD) formula in adults (age ≥ 18 years), or the formula by Schwartz in patients 12 to < 18 years old [Levey et al, 1999; Schwartz et al, 1976; National Kidney Foundation, 2002].
    5. Resting pulse oximetry reading of ≥ 92% and resting systolic blood pressure ≥ 100 mm Hg.
    6. All patients must have at least three 24-hour urine oxalate measurements done by a central laboratory prior to receiving study drug.
    a. Patients who did not participate in PHYOS observational study will be required to have at least three 24-hour urine oxalate measurements during the screening period.
    b. Patients who participated in PHYOS but had less than three 24-hour urine oxalate measurements done by a central laboratory, should have the remaining 24-hour urine oxalate assessments during the screening period.
    7. Adults must be able to understand and give written informed consent for participation in this study, including all evaluations and procedures as specified by this protocol. Minors (patients <18 years of age, or younger than the age of majority, according to local regulations) must have a parent or guardian who is able to understand and give written informed consent for participation in this study, including all evaluations and procedures as specified by this protocol. 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.
    E.4Principal exclusion criteria
    1. Prior renal and/or hepatic transplantation.
    2. History of clinical signs and symptoms of systemic oxalosis other than nephrolithiasis or nephrocalcinosis.
    3. Participation in any clinical study involving administration of any investigational drug within the 30 days before enrollment (NOTE: participation in PHYOS observational study is allowed).
    4. Pregnancy or lactation at the time of screening or enrollment.
    5. Sexually active females of childbearing potential (FOCBP) who are not using a highly effective contraception method and men with a fertile FOCBP partner who are unable or unwilling to use a highly effective contraception method (see Section 8.4).
    6. Patients with a known history of human immunodeficiency virus (HIV), or active infection with hepatitis B virus or hepatitis C virus.
    7. Moderate or severe hepatic impairment (Child-Pugh class B or C)
    8. Liver function test abnormalities: alanine transaminase (ALT) and/or aspartate transaminase (AST) > 2 times upper limit of normal (ULN).
    9. Active alcohol or substance abuse within 60 days prior to enrollment.
    10. Prolonged QTc ≥ 450 msec for males or QTc ≥ 470 msec for females, at baseline as assessed by either Bazett’s or Fredericia’s correction methods.
    11. Clinically significant prior cardio-respiratory disease, such as myocardial infarction within 6 months due to obstructive coronary artery disease, third degree atrioventricular block or uncontrolled rhythm disturbances, active cardiomyopathy (i.e., symptomatic left ventricular dysfunction), or chronic heart failure (CHF) New York Heart Association (NYHA) Class ≥III, or documented moderate or more severe chronic obstructive pulmonary disease (COPD) (ie forced expiratory volume in during the first second [FEV1] < 80% predicted).
    12. Major surgery or interventional procedure within 30 days prior to study entry, or patients with an inadequate recovery from any surgical or interventional procedure.
    13. History of severe reaction to a liposomal product or a known hypersensitivity to lipid products. For Part B (MAD portion), patients with a Grade 3 or higher adverse reaction during Part A (SAD portion) will not be eligible to participate.
    14. Unable to collect 24-hour urine samples or follow other study procedures.
    15. Any disorder or alteration in mental status that would preclude understanding of the informed consent process and/or completion of the study-related evaluations.
    16. Any significant illness, organ system dysfunction, or other condition that, in the opinion of the Investigator, would interfere with the patient’s ability to comply with the protocol requirements, including the ability to attend all visits and undergo all assessments.
    E.5 End points
    E.5.1Primary end point(s)
    Safety and tolerability as determined by number of subjects with adverse events
    E.5.1.1Timepoint(s) of evaluation of this end point
    SAD: Adverse events to be collected from Day 1 through EOS or Day 29 (±1 day).
    MAD: Adverse events to be collected from Day 1 through EOS or Day 85 (±1 day).
    E.5.2Secondary end point(s)
    1) Determination of pharmacokinetics parameters

    2) Change in plasma levels from baseline (BL) to each time point of oxalate and glycolate

    3) Change in urine levels from baseline (BL) of oxalate, oxalate to creatinine ratio and glycolate
    E.5.2.1Timepoint(s) of evaluation of this end point
    1) Plasma samples to be collected:
    SAD: Before DCR-PH1 administration on Day 1, and at ca. 60 min after the start of infusion, at the end of infusion; at 30, 120, 240, 360 and 480 min after end of infusion; at Days 2, 3, 8 (±1 day), 15 (±1 d), 22 (±1 d), 29 (±1 d)
    MAD: Before DCR-PH1 administration and at ca. 60 min after the start of infusion, at the end of infusion; at 30, 120, 240, 360 and 480 min after end of infusion on Day 1; on Days 2, 3, 8 (±1 day), 22 (±1 d), 29 (±1 d), 57 (±1 d)

    2) Plasma samples to be collected:
    SAD: Days 1 (BL), 15 (±1 day), 22 (±1 d), 29 (±1 d)
    MAD: Days 1 (BL), 22(±1 day), 29 (±1 d), 57(±1 d), 85 (±1 d)

    3) Urine samples to be collected:
    SAD: Scr (BL), days 8(±1 day), 15(±1 d), 22 (±1 d)
    MAD: Scr(BL), days 22(±1 day), 29(±1 d), 57(±1 d), 78(±1 d)
    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 Yes
    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) Yes
    E.7.1.1First administration to humans Yes
    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) 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 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
    France
    Germany
    Israel
    Netherlands
    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
    LVLS
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    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.1.1Number 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) 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) Yes
    F.1.1.6.1Number of subjects for this age range: 21
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 21
    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 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 state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 34
    F.4.2.2In the whole clinical trial 42
    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)
    Patients will be treated according to the normal standard of care. Patients participating in Part B will be allowed to participate in an open label long term extension study, provided they have tolerated the study drug and meet extension study eligibility criteria.
    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 Decision2015-11-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-07-21
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-10-14
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