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    Summary
    EudraCT Number:2020-002081-13
    Sponsor's Protocol Code Number:GS-2001
    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:2020-08-17
    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 number2020-002081-13
    A.3Full title of the trial
    A Phase II, randomised, multi-centre placebo-controlled, double-blind study to investigate the safety of GS-248, and efficacy on Raynaud’s phenomenon (RP) and peripheral vascular blood flow in subjects with systemic sclerosis (SSc)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to determine if the study drug (GS-248) is safe, and its effectiveness on Raynaud’s phenomenon (RP) and improving blood supply to fingers and toes in patients with systemic sclerosis (SSc).
    A.4.1Sponsor's protocol code numberGS-2001
    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 SponsorGesynta Pharma AB
    B.1.3.4CountrySweden
    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 supportGesytna Pharma AB
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGesynta Pharma AB
    B.5.2Functional name of contact pointClinical Information Point
    B.5.3 Address:
    B.5.3.1Street AddressWallingatan 24
    B.5.3.2Town/ cityStockholm
    B.5.3.3Post codeSE-111 24
    B.5.3.4CountrySweden
    B.5.6E-mailClinicalInformation@gesynta.se
    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 nameGS-248
    D.3.2Product code GS-248
    D.3.4Pharmaceutical form Capsule
    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 INNGS-248
    D.3.9.2Current sponsor codeGS-248
    D.3.9.4EV Substance CodeSUB206574
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    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 placeboCapsule
    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
    Raynaud's phenomenon (RP) in patients with Systemic sclerosis (SSc)
    E.1.1.1Medical condition in easily understood language
    Raynaud's phenomenon affects the peripheral circulation i.e. fingers and toes.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10037917
    E.1.2Term Raynauds
    E.1.2System Organ Class 100000004866
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10042953
    E.1.2Term Systemic sclerosis
    E.1.2System Organ Class 100000004859
    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 efficacy of GS-248 versus placebo on RP in subjects with SSc.
    E.2.2Secondary objectives of the trial
    To determine the efficacy of GS-248 on peripheral vascular blood flow in patients with SSc.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects must provide signed and dated written informed consent before the conduct of any study-specific procedures.
    2. Male and female subjects aged 18-75 years inclusive.
    3. SSc diagnosed according to European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR) criteria (van den Hoogen F et al. 2013).
    Subjects with signs of other autoimmune diseases (e.g. Sjögren’s syndrome, myositis, rheumatoid arthritis) could be included if SSc is the dominating phenotype.
    4. Raynaud attacks typically ≥7 times per week during the last 4 weeks prior to screening despite background medication (only allowed vasodilatory therapy is calcium channel blockers or PDE-5 inhibitors).
    5. Women of childbearing potential (WOCBP) must be using a highly effective method of contraception to avoid pregnancy throughout the study and for 4 weeks after the last dose of IMP in such manner that the risk of pregnancy is minimised.*
    6. Women must not be pregnant or breastfeeding.
    7. Male subjects to agree to use condom in combination with use of contraceptive methods with a failure rate of <1% to prevent pregnancy and drug exposure of a partner and refrain from donating sperm from the first date of dosing until 3 months after last dosing of the IMP.
    8. Ability of subjects to participate fully in all aspects of this clinical trial.
    * A woman is considered of childbearing potential, i.e. fertile, following
    menarche and until becoming post-menopausal unless permanently
    sterile. Permanent sterilisation methods include hysterectomy, bilateral
    salpingectomy and bilateral oophorectomy. A postmenopausal state is
    defined as no menses for 12 months without an alternative medical
    cause. A high follicle stimulating hormone (FSH) level in the
    postmenopausal range may be used to confirm a post-menopausal state
    in women not using hormonal contraception or hormonal replacement
    therapy. However, in the absence of 12 months of amenorrhea, a single
    FSH measurement is insufficient.
    *Methods that can achieve a failure rate of <1% per year when used
    consistently and correctly are considered as highly effective birth control
    methods. Such methods include combined (oestrogen and progestogen
    containing) hormonal contraception associated with inhibition of
    ovulation (oral, Intravaginal, and/or transdermal); progestogen-only
    hormonal contraception associated with inhibition of ovulation (oral,
    injectable, and/or implantable2); intrauterine device (IUD)2;
    intrauterine hormone-releasing system (IUS); bilateral tubal occlusion2;
    vasectomised partner; sexual abstinence.
    E.4Principal exclusion criteria
    1. SSc disease duration of greater than 120 months from first non-Raynaud manifestation
    2. Current smokers or stopped smoking or used nicotine in any form <3 months prior to Visit 1.
    3. Dose-change or initiation of vasodilating substances (calcium blockers or PDE-5 inhibitors) within 4 weeks prior to Visit 1. Subjects are not allowed to use a combination of calcium blockers and PDE-5 inhibitors from 4 weeks prior to Visit 1 and throughout the study.
    Note: The subjects could be re-screened if they fulfil this exclusion criterion.
    4. Use of iloprost or other intravenous (iv) or per os (po) prostacyclin receptor agonist within 4 weeks prior to Visit 1.
    5. Ongoing treatment with immunosuppressive therapies (other than mycophenolate) including, but not restricted to; cyclophosphamide, azathioprine, methotrexate, or cyclosporine, or use of those medications within 4 weeks prior to Visit 1.
    Note: Subjects could be included if they have been treated with a stable dose of mycophenolic acid during 4 weeks prior to study entry.
    6. Use of systemic corticosteroids within 4 weeks prior to Visit 1 and during the course of the study.
    7. Use of moderate or strong CYP3A4 inhibitors within 5 terminal half-lives or one week, whichever is longer, prior to Visit 2. Examples of a moderate or strong CYP3A4 inhibitors are diltiazem, verapamil and grapefruit juice
    8. Concurrent serious medical condition, with special attention to cardiovascular conditions, which in the opinion of the Investigator makes the subject not suitable for this study.
    9. Prolonged corrected QT interval by Fredericia (QTcF) defined as a mean QTcF >450 msec at Visit 1, or at Visit 2 (prior randomisation).
    10. Creatinine clearance <50 mL/min (determined by Cockcroft-Gault equation).
    11. Active digital ulcer (DU) within 4 weeks prior to Visit 1.
    12. Have known allergies to any components of the GS-248 formulation.
    13. Clinically meaningful laboratory abnormalities at Screening (Visit 1), as determined and documented by the Investigator.
    14. Positive test results for HBsAg, HCVAb or HIV-1 and/or -2 antibodies at Screening (Visit 1).
    15. Subjects known or suspected of not being able to comply with this trial protocol (e.g. due to alcoholism, drug dependency or psychological disorder).
    16. Subject is mentally or legally incapacitated at the time of screening or has a history of clinically significant psychiatric disorders that would impact the subject’s ability to participate in the study according to the Investigator.
    17. Malignancy within the past 5 years except for in situ removal of basal cell carcinoma and cervical intraepithelial neoplasia grade I.
    18. Planned major surgery within the duration of the study.
    19. Blood donation (or corresponding blood loss) within 12 weeks prior to Visit 1.
    20. Participation in another interventional clinical study involving IMP within 4 weeks or given an experimental drug within 5 half-lives, whichever longest, prior to Visit 1.

    Exclusion Criterion for Cold Challenge
    At Visit 2: Finger temperature below 27°C after acclimatising at an ambient temperature of 23°C (±2°C) for a period of 20 minutes.

    Randomization Criteria:
    In addition to fulfilling all inclusion and exclusion criteria, subjects must fulfil the following criteria to be randomised:
    1. ≥7 RP attacks during the last week of the run-in period as captured in the eDiary, with no more than 2 days without RP attacks.
    2. Compliance with the eDiary during the 7 most recent days prior to
    baseline (Visit 2), excluding the visit day itself, defined as having
    submitted ≥5 days of eDiary records (out of a possible 7 days) for RCS
    and RP during that period.
    E.5 End points
    E.5.1Primary end point(s)
    Primary efficacy endpoint:
    Mean change from baseline to week 4 in the number of Raynaud’s Phenomenon attacks per week.
    Primary safety endpoints:
    • Incidence of Adverse events
    • Incidence of Serious Adverse Events
    • Clinical laboratory and vital signs.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline to week 4
    E.5.2Secondary end point(s)
    Key Secondary efficacy endpoints:
    Mean change from baseline to week 4 in the Raynaud’s Condition Score (RCS)
    Mean change from baseline to week 4 in the cumulative duration of Raynaud’s Phenomenon attacks
    Mean change from baseline to week 4 in pain experienced during RP attacks
    Other secondary endpoints:
    Mean change from baseline to week 4 in peripheral blood flow prior to cold challenge and IMP administration.
    Mean change from baseline to week 4 in peripheral blood flow from pre-IMP, to post-IMP administration at Visit 2.
    Mean change in recovery of peripheral blood flow after cold challenge from pre-IMP to post-IMP administration at Visit 2
    Mean change from baseline to week 4 in recovery of peripheral blood flow after cold challenge.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy endpoints:
    Baseline to week 4
    Other endpoints:
    Week 2 and week 4
    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) 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 Yes
    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 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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 years1
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months8
    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: 65
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 15
    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 80
    F.4.2.2In the whole clinical trial 80
    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 Decision2020-08-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-11-02
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-06-15
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