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    Summary
    EudraCT Number:2020-005764-62
    Sponsor's Protocol Code Number:HZNP-HZN-825-301
    National Competent Authority:Portugal - INFARMED
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-08-19
    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 ConcernedPortugal - INFARMED
    A.2EudraCT number2020-005764-62
    A.3Full title of the trial
    A Randomized, Double-blind, Placebo-controlled, Repeat-dose, Multicenter Trial to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of HZN-825 in Patients with Diffuse Cutaneous Systemic Sclerosis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of HZN-825 in Patients with Diffuse Cutaneous Systemic Sclerosis
    A.4.1Sponsor's protocol code numberHZNP-HZN-825-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04781543
    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 SponsorHorizon Therapeutics Ireland DAC
    B.1.3.4CountryIreland
    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 supportHorizon Therapeutics USA, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHorizon Therapeutics U.S.A., Inc.
    B.5.2Functional name of contact pointFarah Ali
    B.5.3 Address:
    B.5.3.1Street Address1 Horizon Way
    B.5.3.2Town/ cityDeerfield, IL
    B.5.3.3Post code60015
    B.5.3.4CountryUnited States
    B.5.4Telephone number0012243833050
    B.5.6E-mailfali@horizontherapeutics.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/3/13/1108
    D.3 Description of the IMP
    D.3.2Product code HZN-825
    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 INNHZN-825
    D.3.9.2Current sponsor codeHZN-825
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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 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
    Diffuse Cutaneous Systemic Sclerosis
    E.1.1.1Medical condition in easily understood language
    Disease characterized by skin hardening (fibrosis) and problems in many organs of the body.
    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 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
    The primary objective is to demonstrate the efficacy of 1 or 2 dose regimens of HZN-825 versus placebo in subjects with diffuse cutaneous SSc, as determined by a comparison of change in forced vital capacity (FVC) % predicted after 52 weeks of treatment
    E.2.2Secondary objectives of the trial
    1.Evaluate the effect of 2dose regimens of HZN-825 vs.placebo on Health Assessment Questionnaire-Disability Index after 52weeks of treatment.
    2.Evaluate the effect of 2 dose regimens of HZN-825 vs. placebo on Physician Global Assessment (MDGA) after 52 weeks of treatment.
    3.Evaluate the effect of 2 dose regimens of HZN-825 vs. placebo on Patient Global Assessment (PTGA) after 52 weeks of treatment.
    4.Evaluate the effect of 2dose regimens of HZN-825 vs.placebo on the
    PhysicalEffects subscale of the scleroderma skin patientreportedoutcome after 52weeks of
    treatment.
    5.Evaluate the effect of 2dose regimens of HZN-825 vs.placebo on the Physical Limitations subscale of the SSPRO-18 after 52 weeks of treatment
    6.Evaluate the effect of 2 dose regimens of HZN-825 vs.placebo on the modified Rodnan skin score,after 52weeks of treatment
    7.Evaluate the effect of 2 dose regimens of HZN-825 vs.placebo on ACRCRISS after 52 weeks of treatment.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Written informed consent.
    2.Male or female between the ages of 18 and 75 years, inclusive, at
    Screening.
    3.Meets the 2013 American College of Rheumatology/European League Against Rheumatism classification criteria for SSc with a total score of ≥ 9
    (Van den Hoogen et al., 2013).
    4.Classified as having skin involvement proximal to the elbow and/or
    knee (diffuse cutaneous SSc subset by LeRoy and Medsger, 2001).
    5.At the time of enrollment, less than 36 months since the onset of the first SSc manifestation, other than Raynaud's phenomenon.
    6.Skin in the forearm suitable for repeat biopsy (only applicable to the
    first 110 subjects for whom biopsy will be performed).
    7.mRSS units ≥15 at Screening.
    8.FVC ≥45% predicted at Screening, as determined by spirometry.
    9.Willing and able to comply with the prescribed treatment protocol
    and evaluations for the duration of the trial.
    E.4Principal exclusion criteria
    1.Positive for anti-centromere antibodies.
    2.Diagnosed w/sine scleroderma or limited cutaneous SSc.
    3.Diagnosed w/other autoimmune connective tissue diseases ,except for
    fibromyalgia, scleroderma-associated myopathy & secondary
    Sjogren's syndrome.
    4.Scleroderma renal crisis diagnosed within6months of the Screening
    Visit.
    5.Any of the following cardiovascular diseases: a. uncontrolled, severe
    hypertension(≥160/100mmHg)or persistent low blood pressure
    (systolic blood pressure<90 mmHg)within6months of Screening, b.
    myocardial infarction within6months of Screening, c. unstable cardiac
    angina within6months of Screening.
    6.DLCO<40%predicted(corrected for hemoglobin).If severe acute
    respiratory syndrome coronavirus2 (SARS-CoV-2) exposure is of clinical
    concern for any subject, consider using a DLCO up to6months before the
    Screening Visit.
    7.Pulmonary arterial hypertension(PAH)by right heart catheterization
    requiring treatment w/more than1oral PAH-approved therapy or any
    parenteral therapy. Treatment is allowed for erectile dysfunction and/or
    Raynaud's phenomenon/digital ulcers.
    8.Corticosteroid use for conditions other than SSc within4weeks prior to
    Screening(topical steroids for dermatological conditions&
    inhaled/intranasal/intra-articular steroids are allowed).
    9.Use of any other non-steroid immunosuppressive agent, small biologic
    molecule, cytotoxic or antifibrotic drug within4weeks prior to Screening,
    including cyclophosphamide, azathioprine(Imuran®)or other
    immunosuppressive or cytotoxic medication. Exceptions include
    mycophenolate mofetil (CellCept®),mycophenolic acid
    (Myfortic®),methotrexate and low-dose prednisone, as follows: use of
    CellCept ≤3g/day, Myfortic ≤2.14g/day, methotrexate ≤15 mg/week
    and prednisone ≤10 mg/day (or equivalent dosing of glucocorticoids) is
    allowed. See Table 9.1 for full details. Subjects taking CellCept, Myfortic
    or methotrexate must have been doing so for≥6months and the dose
    must have been stable for ≥16 weeks prior to the Day 1 Visit.
    Prednisone must have been at a stable dose for ≥8 weeks prior to the
    Day1Visit. It is acceptable to be on background low-dose prednisone
    &anti-malarial drug along with CellCept, Myfortic or methotrexate.
    Rituximab must not have been used within 6 months of the
    Day1Visit.Subjects must not be withdrawn from any standard-of-care
    treatment that is considered necessary for the clinical management of
    the subject in order to fulfill the trial eligibility requirements.
    10.Known active bacterial, viral, fungal, mycobacterial or other infection,
    including tuberculosis or atypical mycobacterial disease(fungal
    infections of nail beds are allowed).
    11.Use of a United States Food and Drug Administration-approved agent for SSc or an investigational agent for any condition within 90days or
    5half-lives,whichever is longer, prior to Screening or anticipated use
    during the course of the trial.
    12.Malignant condition in the past5years(except successfully treated
    basal/squamous cell carcinoma of the skin or cervical cancer in situ).
    13.Women of childbearing potential (WOCBP) or male subjects not
    agreeing to use highly effective method(s)of birth control throughout
    the trial and for 4 weeks after last dose of trial drug. Male subjects must
    refrain from sperm donation and females from egg/ova donation for this
    same time period. Women are considered of childbearing potential if
    they are not postmenopausal and not surgically sterile(documented
    bilateral salpingectomy, bilateral oophorectomy, or hysterectomy).A
    postmenopausal state is defined as no menses for 12months without an
    alternative medical cause. A high FSH level in the postmenopausal range
    may be used to confirm a postmenopausal state in women not using
    hormonal contraception or hormonal replacement therapy. However,in
    the absence of 12months of amenorrhea, a single FSH measurement is
    insufficient. Fertile male subjects must use a condom throughout the
    trial and for4weeks after the last dose of trial drug.A man is considered
    fertile after puberty unless permanently sterile by bilateral
    orchidectomy.
    15.Current drug or alcohol abuse or history of either within the
    previous2years,in the opinion of the Investigator or as reported by the
    subject.
    16.Previous enrollment in this trial or participation in a prior HZN-825 or
    SAR100842 clinical trial.
    17.Known history of positive test for human immunodeficiency virus.
    18.Active hepatitis(and of the following at Screening): (hepatitis B:
    positive hepatitis B surface antigen and positive anti-hepatitis B core
    antibody[anti-HBcAb] and negative hepatitis B surface antibody
    [HBsAb]or positive for HBcAb with a positive test for HBsAb and with
    presence of hepatitis B virus DNA ; hepatitis C: positive anti-hepatitis C
    virus [anti-HCV] and positive HCV RNA.
    19.Current alcoholic liver disease, primary biliary cirrhosis or primary
    sclerosing cholangitis or moderate(Child-Pugh B)to severe(Child-Pugh
    C) hepatic impairment by Child-Pugh scoring system.
    E.5 End points
    E.5.1Primary end point(s)
    Change in FVC % predicted from Baseline to Week 52
    E.5.1.1Timepoint(s) of evaluation of this end point
    From Baseline to Week 52
    E.5.2Secondary end point(s)
    1.Change from Baseline in HAQ-DI at Week 52.
    2.Change from Baseline in MDGA at Week 52.
    3.Change from Baseline in PTGA at Week 52.
    4.Change from Baseline in the Physical Effects subscale of the SSPRO-18 at Week 52.
    5.Change from Baseline in the Physical Limitations subscale of the SSPRO-18 at Week 52.
    6.Proportion of subjects with an mRSS decrease of ≥5 points and 25% from Baseline at Week 52.
    7.Responder rate (defined as ACR-CRISS [predicted probability] of at least 0.6) at Week 52.
    8.Proportion of subjects with an improvement in ≥3 of 5 core measures from Baseline: ≥20% in mRSS, ≥20%in HAQ-DI, ≥20% in PTGA, ≥20% in MDGA and ≥5% for FVC % predicted at Week 52(ACR-CRISS-20).
    E.5.2.1Timepoint(s) of evaluation of this end point
    From Baseline to Week 52
    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 No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability and dose-ranging
    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 trial3
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA76
    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
    Argentina
    Switzerland
    Austria
    Belgium
    Canada
    France
    Germany
    Greece
    Israel
    Italy
    Japan
    Korea, Republic of
    Mexico
    Netherlands
    Poland
    Portugal
    Romania
    Spain
    United Kingdom
    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 years2
    E.8.9.1In the Member State concerned months7
    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 months7
    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: 285
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 190
    F.4.2.2In the whole clinical trial 300
    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)
    All subjects who complete the Double-blind Treatment Period will be eligible to enter into a 52-week extension trial.
    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 Decision2021-08-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-08-24
    P. End of Trial
    P.End of Trial StatusOngoing
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