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    Summary
    EudraCT Number:2021-001253-32
    Sponsor's Protocol Code Number:HZNP-HZN-825-303
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-01-19
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2021-001253-32
    A.3Full title of the trial
    A Phase 2b Randomized, Double-blind, Placebo-controlled, Repeat-dose, Multicenter Trial to Evaluate the Efficacy, Safety and Tolerability of HZN-825 in Subjects with Idiopathic Pulmonary Fibrosis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Efficacy, Safety and Tolerability of HZN-825 in Subjects with Idiopathic Pulmonary Fibrosis
    A.4.1Sponsor's protocol code numberHZNP-HZN-825-303
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05032066
    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 U.S.A., 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 pointBrajesh Pandey
    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 number001 2243833959
    B.5.6E-mailbpandey@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.3.9.3Other descriptive nameSAR100842
    D.3.9.4EV Substance CodeSUB31268
    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
    Idiopathic Pulmonary Fibrosis
    E.1.1.1Medical condition in easily understood language
    A condition in which the lungs become scarred and breathing becomes increasingly difficult.
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10021240
    E.1.2Term Idiopathic pulmonary fibrosis
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part 1 (Core Phase): The primary objective is to demonstrate the efficacy of 2 dose regimens of HZN-825 versus placebo in subjects with IPF, as determined by a comparison of change in forced vital capacity (FVC) % predicted after 52 weeks of treatment.

    Part 2 (Extension Phase): The primary efficacy objective is to assess the efficacy of HZN 825 in subjects with IPF after 52 weeks of open label treatment.
    E.2.2Secondary objectives of the trial
    Core Phase:
    1.Evaluate the effect of 2 dose regimens of PR1 vs PL1 on subjects with decline in FVC% predicted ≥10% from Baseline after 52 w of treatment
    2.Evaluate the effect of 2 dose regimens of PR1 vs PL1 on the changes from Baseline in the 6MWT after 52 w of treatment
    3.Evaluate the effect of 2 dose regimens of PR1 vs PL1 on the K-BILD after 52 w of treatment
    4.Evaluate the effect of 2 dose regimens of PR1 vs PL1 on the L-IPF after 52 w of treatment.
    5.Evaluate the effect of 2 dose regimens PR1 vs PL1 on the LCQ after 52 w of treatment.
    6.Evaluate the effect of 2 dose regimens of PR1 vs PL1 on the rate of hospitalization due to respiratory distress up to 52 w of treatment.
    7.Evaluate the effect of 2 dose regimens of PR1 vs PL1 on the composite endpoint of PFS, where progression includes decline in FVC% predicted ≥10% from Baseline or death over 52 w of treatment.
    8.Assess safety and tolerability of PR1 based on AEs, SAEs and AESI
    9.Evaluate the PK of PR1 and metabolite(s)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Core Phase:
    1. Written informed consent.
    2. Male or female ≥18 years of age at Screening.
    3. Current diagnosis of IPF, as defined by American Thoracic Society (ATS)/European Respiratory Society (ERS)/Japanese Respiratory Society (JRS)/Latin American Thoracic Society (ALAT) guidelines [Raghu et al., 2022] and determined by central review; the date of initial diagnosis of IPF should be ≤7 years prior to Screening.
    4. No recent changes or planned changes to the dose or regimen for IPF therapy, defined as:
    • Receiving a stable dose of IPF-approved therapy (i.e., nintedanib or pirfenidone) for a minimum of 3 months prior to Day 1 with no plans to change the background regimen during trial participation, or
    • Not currently receiving background IPF-approved therapy at Screening (either naïve to IPF-approved therapy or previously discontinued any IPF-approved therapy at least 4 weeks prior to Day 1 or drug-specific, 5 half-lives elimination period if longer than 4 weeks), and with no current plans to restart treatment during trial participation
    • Subjects receiving any additional agent for IPF therapy must be on a stable regimen for at least 3 months prior to Day 1 with no current plans to change the treatment regimen during trial participation. Any previously discontinued therapy used to treat IPF must have been discontinued at least 4 weeks prior to Day 1 or 5 half-lives for that specific therapy must have elapsed, whichever is longer, with no plans to restart the therapy during trial participation.
    5. Lung HRCT historically performed within 6 months prior to the
    Screening Visit and according to the minimum requirements for IPF diagnosis by central review based on subject's HRCT. If an evaluable HRCT is not available within 6 months prior to Screening, an HRCT will be performed at Screening to determine eligibility, according to the same requirements as the historical HRCT. The HRCT must demonstrate a usual interstitial pneumonia or probable usual interstitial pneumonia pattern based on central review vendor interpretation. Histopathology
    in combination with HRCT results supportive of an IPF or IPF likely diagnosis according to Raghu et al., 2022 can be submitted to support subject eligibility.
    6. HRCT shows ≥10% to <50% parenchymal fibrosis (reticulation) and the extent of fibrotic changes is greater than the extent of emphysema on the most recent HRCT scan (central reviewer determined).
    7. Meets all of the following criteria during the Screening Period, as determined by central review:
    a. FVC ≥45% predicted of normal
    b. forced expiratory volume in 1 second (FEV1)/FVC ≥0.7
    c. DLCO corrected for hemoglobin is ≥25% and ≤90% predicted of
    normal
    8. Estimated minimum life expectancy of ≥30 months for non-IPF related disease, in the opinion of the Investigator.
    9. Vaccinations are up to date, according to the Investigator's
    discretion, given age, comorbidities and local availability prior to trial drug dosing.
    10. Willing and able to comply with the prescribed treatment protocol and evaluations for the duration of the trial.

    Extension Phase:
    1. Written informed consent.
    2. Completed the Double-blind Treatment Period (Week 52) of the Core Phase of the trial; subjects prematurely discontinued from trial drug in the Core Phase of the trial for reasons other than safety or tolerability may be included at the discretion of the Investigator after completing scheduled visits, including Week 52 assessments.
    3. Willing and able to comply with the prescribed treatment protocol and evaluations for the duration of the Extension Phase of the trial.
    E.4Principal exclusion criteria
    Core Phase:
    1. Any of the following cardiovascular diseases:
    a. uncontrolled, severe hypertension (≥160/100 mmHg), within 6 months of Screening
    b. myocardial infarction within 6 months of Screening
    c. unstable cardiac angina within 6 months of Screening
    2. Interstitial lung disease (ILD) associated with known primary diseases, connective tissue disorders, exposures or drugs.
    3. Known active bacterial, viral, fungal, mycobacterial or other infection, including tuberculosis or atypical mycobacterial disease (fungal infections of nail beds are allowed). The subject must be 3 months beyond any acute infection with COVID-19 if there has been a prior infection.
    4. Clinically significant pulmonary hypertension requiring chronic medical therapy.
    5. Use of any of the following therapies within 4 weeks prior to Screening, during the Screening Period or planned during the trial: prednisone at steady dose >10 mg/day or equivalent or cyclosporine. Prednisone ≤10 mg/day (or equivalent dosing of glucocorticoids) is allowed. Change in regimen or dosage of any immunosuppressant during the Screening Period through the end of trial participation will require consultation with and approval by the trial Medical Monitor.
    6. Use of rifampin within 2 weeks prior to Day 1 or planned during the trial.
    7. Malignant condition in the past 5 years (except successfully treated basal/squamous cell carcinoma of the skin or cervical cancer in situ).
    8. 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. Fertile male subjects must use a condom throughout the trial and for 4 weeks after the last dose of trial drug.
    9. Pregnant or lactating women and women who plan to become pregnant or breast feed during the trial and within 4 weeks after the last dose of trial drug.
    10. Current drug or alcohol abuse or history of either within the previous 2 years, in the opinion of the Investigator or as reported by the subject.
    11. Previous enrollment in this trial or participation in a prior HZN-825 or
    SAR100842 clinical trial.
    12. Known history of positive test for human immunodeficiency virus (HIV).
    13. Active hepatitis (any of the following at Screening):
    Hepatitis B:
    •positive hepatitis B surface antigen
    •positive for anti-hepatitis B core antibody (anti HBcAb) and a positive test for hepatitis B surface antibody (HBsAb) and presence of hepatitis B virus DNA
    •positive for HBcAb and a negative test for HBsAb and presence of hepatitis B virus DNA
    Hepatitis C:
    •positive anti hepatitis C virus (anti HCV) and positive HCV RNA.
    14. Current alcoholic liver disease, primary biliary cirrhosis or primary sclerosing cholangitis.
    15. Previous organ transplant (including allogeneic and autologous marrow transplant).
    16. International normalized ratio >2, prolonged prothrombin time >1.5 × the upper limit of normal (ULN) or partial thromboplastin time >1.5 × ULN at Screening.
    17. Alanine aminotransferase or aspartate aminotransferase >2.0 × ULN.
    18. Estimated glomerular filtration rate <30 mL/min/1.73 m2 at Screening.
    19. Total bilirubin >1.5 × ULN. Subjects with documented diagnosis of Gilbert's syndrome may be enrolled if their total bilirubin is ≤3.0 mg/dL.
    20.Moderate (Child-Pugh B) to severe (Child-Pugh C) hepatic impairment according to the Child-Pugh scoring system.
    21.Any confirmed Grade 3 or higher laboratory abnormality.
    22.Any laboratory abnormality at Screening that, in the opinion of the Investigator, would preclude the subject's participation in the trial.
    23.Exposure to an experimental drug or experimental vaccine within either 30 days, 5 half-lives of the test agent, or twice the duration of the biological effect of the test agent, whichever is the longest, prior to Day 1.
    24.Any other condition that, in the opinion of the Investigator, would preclude enrollment in the trial.

    Extension Phase:
    1.Anticipated use of another investigational agent for any condition during the course of the trial.
    2.New diagnosis of malignant condition after enrolling in Trial HZNPHZN-825-303 (except successfully treated basal/squamous cell carcinoma of the skin or cervical cancer in situ).
    3.Estimated minimum life expectancy ≤18 months, in the opinion of the Investigator.
    4.WOCBP or male subjects not agreeing to use highly effective method(s) of birth control throughout the Extension Phase and for 4 weeks after last dose of HZN-825.
    5.Pregnant or lactating women.
    6.Any other new development of the disease/condition/significant laboratory test abnormality during the course of the Core Phase of the trial, in the opinion of the Investigator, that would potentially put the subject at unacceptable
    risk.
    7.In the opinion of the Investigator, unlikely to comply with the trial protocol or has a concomitant disease or condition that could interfere with the conduct of the trial.
    E.5 End points
    E.5.1Primary end point(s)
    Core Phase: Change in FVC % predicted from Baseline to Week 52.

    Extension Phase: Change from both Baselines in FVC % predicted at Week 104.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Core Phase: From Baseline to Week 52.
    Extension Phase: From Baselines to Week 104.
    E.5.2Secondary end point(s)
    Core Phase:
    1. Proportion of subjects with decline in FVC % predicted ≥10% from Baseline at Week 52.
    2. Change from Baseline in the 6MWT results to Week 52.
    3. Change from Baseline in K-BILD scores to Week 52.
    4. Change from Baseline in L-IPF scores to Week 52.
    5. Change from Baseline in LCQ scores to Week 52.
    6. Time to first hospitalization due to respiratory distress from Baseline up to Week 52.
    7. Time to first onset of the composite endpoint of PFS from Baseline up to Week 52, where progression includes decline in FVC % predicted ≥10% or death.

    Extension Phase: Safety and Tolerability Endpoints
    1. Incidence of TEAEs and the AESI (orthostatic hypotension) in the
    Extension Phase.
    2. Concomitant medication use in the Extension Phase
    3. Change from OLE Baseline in vital signs in the Extension Phase
    4. Change from OLE Baseline in 12-lead ECG measurements in the
    Extension Phase
    5. Change from OLE Baseline in clinical safety laboratory test results in the Extension Phase
    E.5.2.1Timepoint(s) of evaluation of this end point
    Core Phase: From Baseline to Week 52.

    Extension Phase: From Baselines to Week 104
    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 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) 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 Yes
    E.8.1.7.1Other trial design description
    52-week Double-blind Treatment Period in the Core Phase and 52 weeks of open-label HZN-825 treatment
    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 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 EEA30
    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
    Australia
    Canada
    Chile
    Japan
    Korea, Republic of
    Mexico
    South Africa
    Taiwan
    United States
    France
    Germany
    Greece
    Ireland
    Italy
    Netherlands
    Poland
    Spain
    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 years3
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days13
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months11
    E.8.9.2In all countries concerned by the trial days27
    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: 40
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 95
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 50
    F.4.2.2In the whole clinical trial 135
    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. There are no plans for treatment or care after the subject has ended the participation in the trial (Core and Extension Phase).
    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 Decision2022-01-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-04-11
    P. End of Trial
    P.End of Trial StatusOngoing
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