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    Summary
    EudraCT Number:2022-000676-19
    Sponsor's Protocol Code Number:DS1211-A-U201
    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:2022-06-23
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2022-000676-19
    A.3Full title of the trial
    A Phase 2, 12-Week, Randomized, Double-Blind, Placebo-Controlled Study of DS-1211b in Individuals with Pseudoxanthoma Elasticum
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of DS-1211b in Individuals With PseudoXanthoma Elasticum
    A.4.1Sponsor's protocol code numberDS1211-A-U201
    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 SponsorClinical Director, Daiichi Sankyo, 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 supportDaiichi Sankyo, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDaiichi Sankyo Inc.
    B.5.2Functional name of contact pointClinical Director
    B.5.3 Address:
    B.5.3.1Street Address211 Mt. Airy Road
    B.5.3.2Town/ cityBasking Ridge, NJ
    B.5.3.3Post code07920
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1908992 6400
    B.5.6E-mailCTRinfo@dsi.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 nameDS-1211b
    D.3.2Product code DS-1211b
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNot Applicable
    D.3.9.1CAS number Not Applicab
    D.3.9.2Current sponsor codeDS-1211b
    D.3.9.3Other descriptive nameDS-1211b
    D.3.9.4EV Substance CodeSUB259017
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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 Yes
    D.3.11.13.1Other medicinal product typeSmall-molecule inhibitor of TNAP
    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 placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Pseudoxanthoma Elasticum (PXE)
    E.1.1.1Medical condition in easily understood language
    Pseudoxanthoma Elasticum (PXE)
    E.1.1.2Therapeutic area Not possible to specify
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10037150
    E.1.2Term Pseudoxanthoma elasticum
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic 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 and tolerability of DS-1211b compared with placebo in subjects with PXE

    To assess the dose response by assessing the treatment changes in PD endpoints
    E.2.2Secondary objectives of the trial
    To characterize the PK of DS-1211a (the free form of DS-1211b in plasma) in subjects with PXE
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Signed and dated informed consent form prior to the start of any study-specific qualification procedures
    • Male or female participants aged 18 to 75 years at screening
    • Have an established diagnosis of PXE, with typical ocular and dermatological clinical features such as ocular and skin findings
    • Fully vaccinated for coronavirus disease 2019 (COVID-19) per current Center for Disease Control and Prevention guidelines
    • Women of childbearing potential must have a negative serum pregnancy test at screening and must be willing to use an effective method of birth control upon entering study screening, during the treatment period, and up until the time of the follow-up visit
    • Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, other study procedures, and study restrictions
    E.4Principal exclusion criteria
    • Have a history of bone fracture in the past 6 months
    • Have a history of active metabolic bone disease, excluding osteopenia or osteoporosis without fragility fracture
    • Have a history of calcium pyrophosphate deposit disease, such as chondrocalcinosis, pseudogout, and pyrophosphate arthropathy
    • Have a history of hypophosphatasia
    • Have a history of untreated hyperparathyroidism
    • Participated in another interventional research study in the past 60 days.
    • Currently participating or have participated within the last 12 months in PXE trials or in clinical trials relating to bone mineralization
    • Used bisphosphonate in the preceding 12 months or had plans to use bisphosphonate during the study.
    • Used strong inhibitors or inducers of Cytochrome P450 isozymes, 2C19, 3A4, or 3A5
    • Received Vitamin B6 supplementation >5 mg/day in the month prior to screening and during the study
    • Initiated or changed dose of Vitamin D in the preceding month prior to screening
    • Have an alkaline phosphatase <lower limit of normal (LLN) range
    • Have alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2 × upper limit of normal (ULN) range; total bilirubin >1 × ULN
    • Have PLP >ULN
    • Have hemoglobin <10.0 g/dL
    • Have a QTcF interval duration >450 ms at screening
    • Have moderate to severe renal insufficiency (Creatinine clearance <60 mL/min by Cockcroft-Gault method)
    • Are pregnant or breast-feeding women
    • Are female participants unwilling to use contraceptive methods
    • Have a history of sulfonamide allergy
    • Have any elective surgery planned during the study period
    • Have any other significant condition (medical, psychiatric, social, or medication) that, in the judgment of the Investigator, would prevent full participation or would be inappropriate for the study
    E.5 End points
    E.5.1Primary end point(s)
    1. Number of Participants with Treatment-emergent Adverse Events (TEAEs) in Participants Receiving DS-1211b
    2. Change from Baseline in Pharmacodynamic Parameter Alkaline Phosphatase (ALP) Levels
    3. Change from Baseline in Pharmacodynamic Parameter Inorganic Pyrophosphate (PPi) Levels
    4. Change from Baseline in Pharmacodynamic Parameter Pyridoxal 5'-phosphate (PLP) Levels
    E.5.1.1Timepoint(s) of evaluation of this end point
    Timepoint for primary end point 1:
    From the time the subject signs the study informed consent form (ICF) and up to Day 98 (14 days after the last dose of study drugs)
    Timepoint for primary end point 2:
    Screening (Days -1 to -30) and at Days 1, 15, 43, 84, 86-88, and 98 post-dose of 12-week treatment period
    Timepoint for primary end point 3:
    Screening (Days -1 to -30) and at Days 1, 15, 43, 84
    Timepoint for primary end point 4:
    Screening (Days -1 to -30) and at Days 1, 15, 43, 84, 86-88, and 98 post-dose of 12-week treatment period
    E.5.2Secondary end point(s)
    5. Pharmacokinetic Parameter Maximum Concentration (Cmax)
    6. Pharmacokinetic Parameter Time to Maximum Concentration (Tmax)
    7. Pharmacokinetic Parameter Trough Plasma Concentration (Ctrough)
    8. Pharmacokinetic Parameter Area Under the Plasma Concentration-time Curve (AUC)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Timepoint for secondary end point 5:
    Days, 1, 15, 43, and 84
    Timepoint for secondary end point 6:
    Days, 1, 15, 43, and 84
    Timepoint for secondary end point 7:
    Days, 1, 15, 43, and 84
    Timepoint for secondary end point 8:
    Days, 1, 15, 43, and 84
    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 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) 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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 trial4
    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 No
    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
    Netherlands
    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 years0
    E.8.9.1In the Member State concerned months3
    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 months3
    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 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 63
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Elderly
    F.4 Planned number of subjects to be included
    F.4.1In the member state16
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 16
    F.4.2.2In the whole clinical trial 64
    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)
    None
    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 Decision2022-06-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-07-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-11-21
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