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    Summary
    EudraCT Number:2022-003466-20
    Sponsor's Protocol Code Number:ARORAGE-1001
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2023-01-17
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2022-003466-20
    A.3Full title of the trial
    A Phase 1/2a Study Evaluating the Effects of ARO-RAGE Inhalation Solution in Healthy Subjects and Patients with Inflammatory Lung Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study Evaluating the Effects of ARO-RAGE in Healthy Subjects and Patients with Inflammatory Lung Disease
    A.4.1Sponsor's protocol code numberARORAGE-1001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05276570
    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 SponsorArrowhead Pharmaceuticals, 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 supportArrowhead Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPIVOTAL, S.L.U
    B.5.2Functional name of contact pointMedical Department
    B.5.3 Address:
    B.5.3.1Street AddressC/ Gobelas, 19
    B.5.3.2Town/ cityLa Florida, Madrid
    B.5.3.3Post code28023
    B.5.3.4CountrySpain
    B.5.4Telephone number3491708 12 50
    B.5.5Fax number3491708 13 01
    B.5.6E-mailjesus.gonzalez@pivotalcr.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 nameARO-RAGE Inhalation Solution
    D.3.2Product code ARO-RAGE
    D.3.4Pharmaceutical form Inhalation solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPInhalation use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNADS-015
    D.3.9.1CAS number 2756997-71-2
    D.3.9.2Current sponsor codeARO-RAGE
    D.3.9.3Other descriptive nameADS-015
    D.3.9.4EV Substance CodeSUB298007
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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 Yes
    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 placeboInhalation solution
    D.8.4Route of administration of the placeboInhalation use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Inflammatory Lung Disease
    E.1.1.1Medical condition in easily understood language
    Inflammatory Lung Disease
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10003553
    E.1.2Term Asthma
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal 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 ARO-RAGE in patients with Inflammatory Lung Disease
    E.2.2Secondary objectives of the trial
    Secondary
    •To assess the pulmonary safety of ARO-RAGE in patients using spirometry and diffusion capacity measurements
    • To assess the PK of ARO-RAGE in patients

    Exploratory
    To assess the PD effects of ARO-RAGE in patients
    To assess the efficacy of ARO-RAGE in patients
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Asthma Cohorts C1 to C3: Male and nonpregnant, nonlactating volunteers aged 18 to 60 years (19 to 60 years of age, where applicable according to local regulation). Subjects must have a clinical diagnosis of asthma. Asthma severity must be mild-to-moderate, with severity measured by the type/quantity of medication required to keep the subject’s asthma under control (as detailed in the protocol). Asthma subjects must have a Screening prebronchodilator ppFEV1 ≥70% and blood eosinophil count ≥200 cells/μL. Asthma subjects must be able to produce an acceptable induced sputum sample at Screening. Asthma subjects with a recent asthma exacerbation, or ALT or AST >2×ULN, will be excluded.

    “High FeNO” Asthma Cohorts D1 and D3: Male and nonpregnant, nonlactating volunteers aged 18 to 65 years (19 to 65 years of age, where applicable according to local regulation) having a clinical diagnosis of asthma. Asthma must be treated with a stable daily maintenance controller regimen that includes inhaled corticosteroids (ICS). Asthma may not be treated with biologic therapies. Subjects must have a Screening prebronchodilator ppFEV1 ≥40%. In addition, subjects must have FeNO ≥35 ppb at all Screening visits. Asthma subjects with a recent asthma exacerbation, or ALT or AST >2×ULN, will be excluded.

    Please refer to the study Protocol to see all the inclusion criteria
    E.4Principal exclusion criteria
    Exclusion Criteria for asthma Cohorts C1 to C3
    1. Acute lower respiratory infection or asthma exacerbation within 30 days prior to first dose
    2. Acute upper respiratory infection within 7 days prior to first dose
    3. Positive COVID-19 test during Screening window. Any record of a positive test during the Screening window, whether protocol-mandated antigen test or any NAAT or antigen test obtained outside of this study, serves as a potential exclusion criterion (see below).
    a. In the event of a positive COVID-19 test during Screening, and if the PI deems the subject clinically appropriate to proceed to study drug dosing and no other exclusion criteria are met (eg, #1 or #4), the PI may elect to proceed to randomize the subject. In such a case, the first dose may not be given until >7 days after both clinical resolution of infection and conversion of antigen test from positive to negative (if such results are available), whichever is later. If necessary, the Screening period may be extended to >28 days, but in no case will the Screening period be extended to >45 days.
    4. Chronic or acute infection that is clinically significant or requires treatment with systemic antibiotics, antivirals, antifungals, or antiparasitics within 30 days prior to first dose.
    a. Please note that a positive sputum bacterial culture from Study Day 1 does not meet this exclusion criterion
    5. Use of systemic corticosteroid therapy within 90 days prior to first dose
    6. Use of immunosuppressive medication (eg, methotrexate, cyclosporine, azathioprine, etc.) within 90 days prior to first dose. See Section 8.2.5.4 for more details.
    7. Use of biologic therapies for asthma (ie, anti-IgE, anti-IL5/IL5R, anti-IL4R, anti-TSLP) within 16 weeks prior to first dose
    8. Prior history of bronchial thermoplasty treatment
    9. Diagnosis of vocal cord dysfunction, reactive airways dysfunction syndrome, panic attacks with hyperventilation, or other mimics of asthma
    10. Any concomitant pulmonary disease that, in the opinion of the Investigator and/or Medical Monitor, will interfere with the evaluation of the study drug or interpretation ofpatient safety or study results (including, but not limited to: COPD, interstitial lung disease, cystic fibrosis, allergic bronchopulmonary aspergillosis, bronchiectasis, tuberculosis, etc). Any concomitant pulmonary disease must be discussed with the Medical Monitor during Screening.
    11. Any history of organ transplant
    12. Human immunodeficiency virus infection, as shown by presence of anti-HIV antibodies (seropositive)
    13. Seropositive for HBV or HCV (positive result for anti-HCV antibodies must be confirmed with positive HCV RNA test for exclusion)
    14. Uncontrolled hypertension (SBP >150 mmHg or DBP >100 mmHg) at Screening
    15. A history of Torsades de Pointes, ventricular rhythm disturbances (eg, ventricular tachycardia or fibrillation), pathologic sinus bradycardia (<50 bpm with symptoms), heart block (excluding first-degree block, being PR prolongation only), congenital long QT syndrome, new ST segment elevation or depression, or new Q wave on ECG. Participants with a history of atrial arrhythmias should be discussed with the Medical Monitor.
    16. A family history of congenital long QT syndrome or unexplained sudden cardiac death
    17. Use of medications known to prolong the QTc interval within 30 days prior to first dose (eg, azithromycin)
    18. Use of theophylline within 30 days prior to first dose
    19. Symptomatic heart failure (per NYHA guidelines), unstable angina, myocardial infarction, severe cardiovascular disease (ejection fraction <20%), TIA, or CVA within 24 weeks prior to first dose
    20. History of malignancy within the past 2 years except for adequately treated basal cell carcinoma, squamous cell skin cancer, superficial bladder tumors, or in situ cervical cancer. Participants with other curatively-treated malignancies who have no evidence of metastatic disease and >2-year disease-free interval may be entered following approval by the Medical Monitor.
    21. History of major surgery within 12 weeks prior to first dose
    22. Unwilling to limit alcohol consumption to within moderate limits for the duration of the study, as follows: not more than 14 units per week for women and 21 units per week for men (1 unit=150 mL of wine, 360 mL of beer, or 45 mL of 40% alcohol)
    23. Use of illicit drugs (such as cocaine or PCP) within 1 year prior to Screening or positive urine drug screen at Screening (a urine drug screen deemed positive due to prescription medications or for benzodiazepines, opioids, or THC/marijuana is acceptable and inclusion is at the discretion of the PI). Subjects who smoke or vape prescription THC/marijuana should be discussed with the Medical Monitor.

    Please refer to the study protocol Exclusion Criteria Section to see complete information
    E.5 End points
    E.5.1Primary end point(s)
    The incidence and frequency of treatment-emergent adverse events (TEAEs) over time through end of study (EOS)
    E.5.1.1Timepoint(s) of evaluation of this end point
    All visits
    E.5.2Secondary end point(s)
    • Change from baseline over time through EOS in FEV1 as a safety assessment
    • Change from baseline over time through EOS in FVC as a safety assessment
    • Change from baseline over time through EOS in DLCO as a safety assessment
    • Plasma PK parameters of ARO-RAGE after each dose of study drug
    E.5.2.1Timepoint(s) of evaluation of this end point
    Cohorts C
    • Change in FEV1: at all visits
    • Change in FVC: at all visits
    • Change in DLCO: at visits 2, 6, 11 and 15
    • Plasma PK parameters of ARO-RAGE: at visits 2, 3, 7 and 8
    Cohorts D
    • Change in FEV1: at all visits
    • Change in FVC: at all visits
    • Change in DLCO: at visits 3,4,5 and 9
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans Yes
    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 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA7
    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
    New Zealand
    Australia
    Korea, Republic of
    Thailand
    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
    LPLV
    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 months15
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months28
    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: 69
    F.1.3Elderly (>=65 years) No
    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 44
    F.4.2.2In the whole clinical trial 149
    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 Decision2023-03-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-01-18
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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