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    Summary
    EudraCT Number:2017-003534-89
    Sponsor's Protocol Code Number:DCR-PHXC-101
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-10-04
    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 ConcernedFrance - ANSM
    A.2EudraCT number2017-003534-89
    A.3Full title of the trial
    A Placebo-Controlled, Single-Blind, Single-Center Phase 1 Study in Normal Healthy Volunteers and Open-Label Multi Center Study in Patients with Primary Hyperoxaluria to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of DCR PHXC Solution for Injection (subcutaneous use)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    2-part study of DCR-PHXC in healthy volunteers and in primary hyperoxaluria patients
    A.3.2Name or abbreviated title of the trial where available
    2-part study of DCR-PHXC in NHV and PH patients
    A.4.1Sponsor's protocol code numberDCR-PHXC-101
    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 pointRalf Rosskamp
    B.5.3 Address:
    B.5.3.1Street Address87 Cambridge Park Drive
    B.5.3.2Town/ cityCambridge,
    B.5.3.3Post codeMA 02140
    B.5.3.4CountryUnited States
    B.5.4Telephone number617 612 6275
    B.5.6E-mailrrosskamp@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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameDCR-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-PHXC
    D.3.9.2Current sponsor codeDCR-PHXC
    D.3.9.4EV Substance CodeAS1
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    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
    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
    The primary objective for part B of the study is to evaluate the safety and tolerability of single doses of DCR PHXC in Primary Hyperoxaluria patients.
    E.2.2Secondary objectives of the trial
    The secondary objective of part B of the study is:
    • To characterize the pharmacokinetics parameters (PK) of single doses of DCR-PHXC. This means, for example, looking to see how fast the drug is absorbed and seeing how much of the drug reaches the blood stream.

    • To evaluate what effect the drug has on the body (pharmacodynamic) after single doses of DCR PHXC are given to patients with PH. To measure this we will look at biochemical markers including, but not limited to, changes in plasma oxalate and glycolate , and urine oxalate and glycolate concentrations

    The exploratory objectives are:
    • To evaluate the pharmacodynamic (PD) effects of single doses of DCR PHXC in patients with PH aged 12 to 17 and adults. Looking at the changes in plasma glyoxalate.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    PH patients must meet all of the following criteria to be eligible for participation in this study.
    1. Patient and/or patient's parent or guardian if the patient is a minor (defined as patient < 18 years of age, or younger than the age of majority, according to local regulations),
    a. Understands the full nature and purpose of the study, including possible risks and side effects.
    b. Is willing and able to comply with all study procedures including collection of 24-hr urine samples.
    c. Provides informed consent. 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. For children younger than 12 years of age, assent will be based on local regulations.

    2. Male or female, at least 6 years of age at the time of obtaining informed consent.

    3. Patient must have a minimum body weight of 25 Kg.

    4. Documented diagnosis of PH1 or PH2, confirmed by genotyping (historically available genotype information is acceptable for study eligibility).
    5. 24-hr urine oxalate excretion ≥0.7 mmol for patients 18 years and older, or ≥0.7 mmol per 1.73 m2 body surface area (BSA) for patients less than 18 years of age, on at least one of the two assessments conducted in the screening period, with less than 30% variation between both oxalate measurements.

    6. eGFR ≥30 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 6 to < 18 years old (Levey et al., 1999; Schwartz et al., 2009; National Kidney Foundation, 2002).

    7. Males, female patients of childbearing potential and female partners of male patients of childbearing potential must be willing to use a highly effective and approved contraceptive method(s) from the date of informed consent until 12 weeks after the last dose of IMP. A highly effective method of contraception is defined as fulfilling at least one of the following:
    a. Strict abstinence: When this is in line with the preferred and usual lifestyle of the patient. [Periodic abstinence (e.g., calendar, ovulation, symptom-thermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.]
    b. Surgically sterile (having undergone one of the following surgical procedures: hysterectomy, bilateral tubal ligation, bilateral oophorectomy, or bilateral salpingectomy) and at least 6 weeks post-sterilization.
    c. Combined hormonal oral contraceptive (estrogen and progesterone), implanted, or injectable contraceptive on a stable dose for at least 1 month prior to the screening visit plus a barrier method. Combined hormonal contraception is considered a highly effective method of contraception only if it is associated with inhibition of ovulation. If associated with inhibition of ovulation, progesterone-only hormonal contraception is also considered a highly effective method of contraception.
    d. Intrauterine devices plus condoms. Hormonal IUD inserted at least 1 month prior to the screening visit.
    e. Double-barrier methods [e.g., Condom and Occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/suppository]. Of note, a female condom and a male condom, or two male condoms, should not be used together as friction between the two can result in either product failing.
    f. Vasectomized partner (at least 6 months post-procedure) prior to the screening visit.
    8. Postmenopausal females: defined as 12 months with no menses prior to screening and a serum FSH >26 IU/L at screening.
    9. For WOCP: a negative pregnancy test at screening and Day 0
    10. Patients with PH1 receiving pyridoxine at stable doses at least 4 weeks prior to study entry must be willing to remain on the same stable dose during the study. In the unlikely event that a patient with PH2 is receiving pyridoxine, this should be discontinued at least 4 weeks prior to study entry
    E.4Principal exclusion criteria
    PH patients meeting any of the following criteria will be excluded from this study:
    1. Prior renal and/or hepatic transplantation.
    2. Currently receiving dialysis.
    3. Documented evidence of clinical manifestations of systemic oxalosis.
    4. Participation in any clinical study where they received an investigational medical product within 4 months before enrollment. For IMPs with the potential to reduce Uox and/or plasma oxalate, these concentrations must have returned to historical baseline levels. a. If patient participated in an earlier cohort in this study (DCR-PHXC-101), a minimum of 8 weeks must have elapsed prior to re-enrollment and urinary oxalate excretion must have returned to ≥80% of baseline.

    5. Presence of any medical condition or co-morbidities that would interfere with study compliance or data interpretation or potentially impact patient safety including, but not restricted to: a. severe intercurrent illness
    b. routine vaccination within 30 days prior to dosing and through EOS visit
    c. known causes of active liver disease/ injury or transaminase elevation (e.g., alcoholic liver disease, Nonalcoholic fatty liver disease/ steatohepatitis (NAFLD/NASH)
    d. physician concerns about excess alcohol consumption
    e. routine or chronic use of more than 3 grams of acetaminophen daily.

    6. History of alcohol consumption exceeding more than 21 units in males, 14 units in females, per week as determined by the Investigator. See Section 5 for details.
    7. Women who are pregnant, lactating, or planning to attempt to become pregnant during this study or within 90 days after last dosing of IMP.
    8. Liver function test (LFT) abnormalities: ALT and/or AST >1.5 times ULN for age and gender.
    9. History of one or more of the following reactions to an oligonucleotide-based therapy a. Severe thrombocytopenia
    b. Hepatotoxicity
    c. Severe flu-like symptoms leading to discontinuation of therapy
    d. Localized skin reaction from the injection (Grade 3 or higher) leading to discontinuation of therapy.
    e. Coagulopathy/ clinically important prolongation of clotting time

    E.5 End points
    E.5.1Primary end point(s)
    Safety analysis will be based on the Safety Analysis Sets, which will include all volunteers and patients who received a full or partial dose of DCR PHXC. This will be the primary analysis population for safety evaluation.

    PK analysis will be based on the PK analysis sets (PK Set), which will include all volunteers and patients who received a full dose of DCR PHXC and have sufficient data for at least 1 post-dose PK assessment.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety will be measured at multiple intervals

    PK blood measured at the following: Pre-dose, Day 1, and at 5, 15 and 30 minutes and 1, 2, 4, 6, 8, and 12 hours after the injection, and at Days 2 (24 hours), 3 (48 hours), 8 (168 hours), 15 (336 hours), 22 (504 hours) and 42 (1008 hours) and 57 (1344 hours).

    PK urine: 2ml aliquots to be taken from pooled urine 0-6hrs, 6-12hrs and 12-24 hours.

    PK blood for aged 6-11 patient group at the following: Pre-dose, Day 1, and at 30 minutes and 2, and 8 hrs after the injection, and at Days 2 (24 hrs), 15 (336 hrs), and 57 (1344 hrs).
    E.5.2Secondary end point(s)
    Not Applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not Applicable
    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 No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 EEA6
    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
    United States
    E.8.7Trial has a data monitoring committee Yes
    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 years0
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months9
    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: 6
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 35
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 35
    F.4.2.2In the whole clinical trial 41
    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 to stick with normal treatment such as a diet with reduced oxalate intake etc.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-02-22
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-11-19
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