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    Summary
    EudraCT Number:2021-003680-10
    Sponsor's Protocol Code Number:AROAPOC3-3001
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-03-11
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2021-003680-10
    A.3Full title of the trial
    A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults with Familial Chylomicronemia Syndrome
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults with Familial Chylomicronemia Syndrome
    A.4.1Sponsor's protocol code numberAROAPOC3-3001
    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 organisationArrowhead Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointJesse Ho
    B.5.3 Address:
    B.5.3.1Street Address177 East Colorado Boulevard, Suite 700
    B.5.3.2Town/ cityPasadena, CA
    B.5.3.3Post code91105
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16265670691
    B.5.6E-mailjho@arrowheadpharma.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/3/21/2459
    D.3 Description of the IMP
    D.3.2Product code ARO-APOC3
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTBD
    D.3.9.1CAS number 2379776-40-4
    D.3.9.2Current sponsor codeADS-005
    D.3.9.3Other descriptive nameSynthetic double-stranded siRNA oligonucleotide directed against apolipoprotein C-III mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues
    D.3.9.4EV Substance CodeSUB208166
    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 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 placeboSolution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Familial Chylomicronemia Syndrome
    E.1.1.1Medical condition in easily understood language
    Rare genetic disease
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 24.1
    E.1.2Level LLT
    E.1.2Classification code 10085051
    E.1.2Term Familial chylomicronemia syndrome
    E.1.2System Organ Class 100000004850
    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 of the study is to evaluate the efficacy and safety of ARO-APOC3 in adults with FCS
    E.2.2Secondary objectives of the trial
    Not Applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Males or nonpregnant (who do not plan to become pregnant), nonlactating females ≥18 years of age
    2. Able and willing to provide written informed consent prior to the performance of any study specific procedures
    3. Fasting TG ≥10 mmol/L (≥880 mg/dL) at screening, that is refractory to standard lipid lowering therapy (sample drawn after at least the minimum time on stable lipid lowering regimen described in the protocol). Two repeat tests are allowed to qualify.
    4. A diagnosis of FCS based on a documented history of fasting TG levels in excess of 1000 mg/dL on repeated testing (for at least 3 prior occasions), and at least one of the following:
    a. A supportive genetic test (from a source-verifiable medical record or based on screening genotype). Supportive genetic testing includes but is not limited to homozygous, compound heterozygous, or double heterozygote for loss of function or otherwise inactivating mutations in genes affecting lipoprotein lipase activity including LPL, APOC2, APOA5, GPIHBP1, GPD1, or LMF1; or evidence of low LPL activity (<20% of normal) based on source verifiable documentation; or
    b. Documented history of recurrent episodes of acute pancreatitis, not caused by alcohol or cholelithiasis; or
    c. Documented history of recurrent hospitalizations for severe abdominal pain without other explainable cause; or
    d. Documented history of childhood pancreatitis; or
    e. Family history of hypertriglyceridemia-induced pancreatitis
    5. Willing to follow dietary counseling as per PI judgment based on local standard of care, consistent with an intake of ≤ 20 g of fat per day during the study
    6. If on medications for management of type 2 diabetes, or other medications specified in the protocol, the dosing regimen must be stable before collection of qualifying lipid parameter at screening.
    7. Participants with a medical history of clinical atherosclerotic cardiovascular disease (ASCVD) or those with elevated 10-year ASCVD risk (eg, ≥7.5% per American Heart Association / American College of Cardiology risk calculator) must be on appropriate lipid-lowering therapy as per local standard of care (ie, including moderate to high intensity statin, as indicated) prior to collection of qualifying TG levels.
    8. Participants of childbearing potential must agree to use a highly effective form of contraception in addition to a male condom, during the study and for at least 24 weeks after the last dose of IP. Women of childbearing potential on a hormonal contraceptive must be stable on the medication for >2 menstrual cycles prior to Day 1. Men must not donate sperm during the study and for at least 24 weeks after the last dose of IP.
    E.4Principal exclusion criteria
    1. Current use or use within the last 365 days from Day 1 of any hepatocyte-targeted siRNA or antisense oligonucleotide molecule
    2. Diabetes mellitus with any of the following:
    a. Newly diagnosed within 12 weeks of screening
    b. HbA1c ≥9.0% (or >75 mmol/mol International Federation of Clinical Chemistry [IFCC] units) at screening
    3. Active pancreatitis within 12 weeks before Day 1
    4. History of acute coronary syndrome events (myocardial infarction or unstable angina) or procedures (coronary revascularization, angioplasty, or stenting) within 24 weeks of Day 1
    5. History of major surgeries within 12 weeks of Day 1 (including cardiac and vascular surgeries, eg, coronary artery bypass graft)
    6. Any of the following laboratory values at screening:
    a. ALT or AST ≥3×ULN at screening
    b. Total bilirubin ≥1.5 ULN (if the participant has a prior diagnosis and documentation of Gilbert’s syndrome, then total bilirubin must be ≤3 mg/dL at screening)
    c. Estimated glomerular filtration rate <30 mL/min/1.73 m2 at screening, using the Chronic Kidney Disease-Epidemiology Collaboration (CKD-EPI) creatinine equation (Levey 2009)
    d. Spot urine protein/spot urine creatinine ratio greater than 3 grams per day
    e. Clinically significant abnormality in prothrombin time, partial thromboplastin time, or INR
    7. Uncontrolled hypertension (blood pressure >160/100 mmHg at screening); if untreated, participant may be rescreened once hypertension is treated and controlled
    8. Use of any of the following:
    a. Systemic use of corticosteroids or anabolic steroids within 4 weeks prior to Day 1 or planned use during the study
    b. GLP-1 receptor agonists
    c. Plasma apheresis within 4 weeks prior to Day 1 or planned during the study
    d. Blood donation of 50 to 499 mL within 4 weeks of collection of qualifying lipid parameter collection or of >499 mL within 8 weeks of qualifying lipid parameter collection
    9. On treatment with HIV antiretroviral therapy
    10. Seropositive (hepatitis B surface antigen [HBsAg] +) for hepatitis B virus (HBV) or hepatitis C virus (HCV) (HCV seropositivity requires positive test for antibodies confirmed with positive test for HCV RNA)
    11. New York Heart Association (NYHA) Class II, III, or IV heart failure or last known ejection fraction of <30%
    12. Clinical evidence of primary hypothyroidism (screening TSH > ULN and free T4<LLN), primary subclinical hypothyroidism (screening TSH > ULN and free T4 WNL), or secondary hypothyroidism (screening TSH < LLN and free T4< LLN)
    13. History of stroke, transient ischemic attack, or peripheral artery disease within 24 weeks of first dose
    14. History of bleeding diathesis or coagulopathy
    15. Current diagnosis of nephrotic syndrome
    16. 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 men (1 unit approximately corresponds to 80 mL of wine, 200 mL of beer, or 25 mL of 40% alcohol)
    17. History of malignancy within the last 2 years prior to the date of consent requiring systemic treatment except for adequately treated basal cell carcinoma, squamous cell skin cancer, superficial bladder tumors, or in situ cervical cancer. Currently receiving systemic cancer treatment(s) or, in the PI’s opinion, at risk of relapse for recent cancer.
    18. Use of an investigational agent or device within 30 days or within 5 half-lives, based on plasma PK (whichever is longer) prior to Day 1 or current participation in an interventional investigational study. Participants previously exposed to ARO APOC3 or ARO-ANG3 will require a washout period of at least 1 year from last dose.
    19. Any concomitant medical or psychiatric condition or social situation or any other situation that, in the PI’s judgment, would make it difficult to comply with protocol requirements or put the participant at additional safety risk.
    20. Clinical evidence of Cushing’s syndrome
    E.5 End points
    E.5.1Primary end point(s)
    Percent change from baseline at Month 10 in fasting TG
    E.5.1.1Timepoint(s) of evaluation of this end point
    Month 10
    E.5.2Secondary end point(s)
    Key secondary endpoints:
    1. Percent change from baseline at Month 10 and 12 (averaged) in fasting TG
    2. Percent change from baseline at Month 10 in fasting APOC3
    3. Percent change from baseline at Month 12 in fasting APOC3
    Secondary endpoints:
    4. Percent change from baseline at Month 10 in non-high density lipoprotein cholesterol (non-HDL-C), and HDL-C
    5. Percent change from baseline at Month 12 in fasting TG, non-HDL-C, and HDL-C
    6. Proportion of participants achieving TG of <500 mg/dL at Month 10
    7. Proportion of participants achieving TG of <500 mg/dL at Month 12
    8. Change and percent change from baseline at each scheduled assessment in fasting TG up to Month 12
    9. Participant incidence of TEAEs
    10. Incidence of positively adjudicated events of acute pancreatitis
    E.5.2.1Timepoint(s) of evaluation of this end point
    Key secondary endpoints:
    1. month 10 and 12 (averaged)
    2. month 10
    3. month 12
    Secondary end points:
    4 and 6 month 10
    5 and 7 month 12
    8. each scheduled assessment up to month 12
    9 and 10- throughout both periods
    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 No
    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 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) No
    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 Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Randomized double-blind period followed by an open-label extension period
    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 trial4
    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 EEA16
    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
    Israel
    Japan
    Mexico
    New Zealand
    Oman
    Singapore
    United States
    Turkey
    Serbia
    Austria
    Belgium
    France
    Germany
    Ireland
    Poland
    Spain
    Korea, Republic of
    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 years1
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months7
    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: 54
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 18
    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 13
    F.4.2.2In the whole clinical trial 72
    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)
    After participation in the trial, patients will be treated per the principal investigator's discretion and local standard of care.
    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 Decision2022-04-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-06-21
    P. End of Trial
    P.End of Trial StatusOngoing
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