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    Summary
    EudraCT Number:2020-005764-62
    Sponsor's Protocol Code Number:HZNP-HZN-825-301
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-04-29
    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 ConcernedFrance - ANSM
    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-mailclinicaltrials@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 No
    D.2.5.1Orphan drug designation number
    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 2 dose regimens of HZN-825 vs. placebo on
    Health Assessment Questionnaire-Disability Index after 52 weeks of
    treatment.
    2.Evaluate the effect of 2 dose regimens of HZN-825 vs. placebo on Physician Global Assessment after 52 weeks of treatment.
    3.Evaluate the effect of 2 dose regimens of HZN-825 vs. placebo on Patient Global Assessment after 52 weeks of treatment.
    4.Evaluate the effect of 2 dose regimens of HZN-825 vs. placebo on the Physical Effects subscale of the scleroderma skin patient-reported
    outcome after 52 weeks of treatment.
    5.Evaluate the effect of 2 dose 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 52 weeks of treatment.
    7. Evaluate the effect of 2 dose regimens of HZN-825 vs. placebo on ACR-CRISS 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 classificationcriteria 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 knee
    (diffuse cutaneous SSc subset byLeRoy and Medsger, 2001).
    5.At the time of enrollment, less than 36 months since the onset of the
    first SSc manifestation, other thanRaynaud's phenomenon.
    6.Based on data available through medical history and/or medical
    records, the subject should have at least 1 ofthe following:
    a.disease duration ≤18 months
    b.increase ≥3 in mRSS units compared with the last visit within the
    previous 1 month to 6 months
    c.involvement of 1 new body area with ≥2 mRSS units or 2 new body
    areas with ≥1 mRSS unit
    d.documentation of worsening skin thickening for subjects who did not
    have mRSS performed during theprevious visit
    e.presence of tendon friction rub at Screening
    7.Presence of at least 1 of the following features of elevated acute phase
    reactants at Screening:
    •high-sensitivity C-reactive protein (hsCRP) ≥0.6 mg/dL (≥6 mg/L),
    •erythrocyte sedimentation rate (ESR) ≥28 mm/hr,
    •platelet count ≥330 × 109/L (330,000/μL).
    8.Skin thickening from SSc in the forearm suitable for repeat biopsy.
    9.mRSS units ≥15 at Screening.
    10.FVC ≥45% predicted at Screening, as determined by spirometry.
    11.Willing and able to comply with the prescribed treatment protocol
    and evaluations for the duration of thetrial.
    E.4Principal exclusion criteria
    1.Positive for anti-centromere antibodies.
    2.Diagnosed with sine scleroderma or limited cutaneous SSc.
    3.Diagnosed with other autoimmune connective tissue diseases, except for fibromyalgia,scleroderma-associated myopathy and secondary Sjogren's syndrome.
    4.Scleroderma renal crisis diagnosed within 6 months of the Screening Visit.
    5.Any of the following cardiovascular diseases:
    a.uncontrolled, severe hypertension (≥160/100 mmHg) or persistent
    low blood pressure (systolic bloodpressure <90 mmHg) within 6 months of Screening,
    b.myocardial infarction within 6 months of Screening,
    c.unstable cardiac angina within 6 months 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 to 6 monthsbefore the Screening Visit.
    7.Pulmonary arterial hypertension (PAH) by right heart catheterization requiring treatment with more than1 oral PAH-approved therapy or any
    parenteral therapy. Treatment is allowed for erectile dysfunctionand/orRaynaud's phenomenon/digital ulcers.
    8.Corticosteroid use for conditions other than SSc within 4 weeks prior to Screening (topical steroids fordermatological conditions and
    inhaled/intranasal/intra-articular steroids are allowed).
    9.Use of any other non-steroid immunosuppressive agent, small biologic molecule, cytotoxic or anti-fibroticdrug within 4 weeks of Screening,
    including cyclophosphamide, azathioprine (Imuran®) or otherimmunosuppressive or cytotoxic medication.
    10.Known active bacterial, viral, fungal, mycobacterial or other infection,
    including tuberculosis or atypicalmycobacterial 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 forany condition within 90 days or 5
    half-lives, whichever is longer, prior to Screening or anticipated use during the course of the trial.
    12.Malignant condition in the past 5 years (except successfully treated basal/squamous cell carcinoma of theskin or cervical cancer in situ).
    13.Women of childbearing potential or male subjects not agreeing to use highly effective method(s) of birthcontrol throughout the trial and for 1
    month after last dose of trial drug. Male subjects must refrain fromsperm donation and females from egg/ova donation for this same time period.
    14.Pregnant or lactating women.
    15.Current drug or alcohol abuse or history of either within the previous
    2 years, in the opinion of theInvestigator 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 (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 testfor HBsAb and with presence of hepatitis B virus DNA at
    Screening; hepatitis C: positive anti-hepatitis Cvirus [anti-HCV] and
    positive RNA HCV).
    19.Current alcoholic liver disease, primary biliary cirrhosis or primary
    sclerosing cholangitis.
    20.Previous organ transplant (including allogeneic and autologous
    marrow transplant).
    21.International normalized ratio >2, prolonged prothrombin time >1.5
    × the upper limit of normal (ULN) orpartial thromboplastin time >1.5 ×
    ULN at Screening.
    22.Alanine aminotransferase or aspartate aminotransferase >2 × ULN.
    23.Estimated glomerular filtration rate <30 mL/min/1.73 m2 at
    Screening.
    24.Total bilirubin >2 × ULN. Subjects with documented diagnosis of
    Gilbert's syndrome may be enrolled iftheir total bilirubin is ≤3.0 mg/dL.
    25.Any other condition that, in the opinion of the Investigator, would
    preclude enrollment in the trial.
    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
    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) Yes
    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 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 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
    Canada
    Israel
    Japan
    Korea, Republic of
    Mexico
    Turkey
    United States
    Austria
    Belgium
    France
    Germany
    Greece
    Hungary
    Ireland
    Italy
    Poland
    Portugal
    Romania
    Spain
    Switzerland
    United Kingdom
    Argentina
    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 days
    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 state20
    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 Decision2021-06-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-06-16
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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