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    Summary
    EudraCT Number:2017-003372-31
    Sponsor's Protocol Code Number:LJ401-BT01
    National Competent Authority:Cyprus - MoH-Ph.S
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-02-23
    Trial results View 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 ConcernedCyprus - MoH-Ph.S
    A.2EudraCT number2017-003372-31
    A.3Full title of the trial
    A Multi-center, Randomized, Open-Label, Parallel-Group Study with LJPC-401 for the Treatment of Myocardial Iron Overload in Adult Patients with Transfusion-Dependent Beta Thalassemia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A multi-center clinical study to determine the effects of LJPC-401 on iron in the heart muscle in patients with beta-thalassemia who are treated with routine blood transfusions
    A.4.1Sponsor's protocol code numberLJ401-BT01
    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 SponsorLa Jolla Pharmaceutical Company
    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 supportLa Jolla Pharmaceutical Company
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationLa Jolla Pharmaceutical Company
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street Address4550 Towne Centre Ct
    B.5.3.2Town/ citySan Diego
    B.5.3.3Post code92121
    B.5.3.4CountryUnited States
    B.5.4Telephone number1 858-433-6908
    B.5.6E-maillajollaregulatoryaffairs@ljpc.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/3/15/1555
    D.3 Description of the IMP
    D.3.1Product nameLJPC-401
    D.3.2Product code LJPC-401
    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 INNHepcidin-25 (human)
    D.3.9.1CAS number 342809-17-0
    D.3.9.2Current sponsor codehepcidin-25 acetate, Synthetic human hepcidin
    D.3.9.3Other descriptive nameHepcidin-25 (human)
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Transfusion-dependent beta thalassemia with myocardial iron overload
    E.1.1.1Medical condition in easily understood language
    Increased amount of iron in the heart muscle (iron overload) as a result of blood transfusions
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level SOC
    E.1.2Classification code 10005329
    E.1.2Term Blood and lymphatic system disorders
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Efficacy on cardiac iron as measured by cardiac T2* MRI in adult patients with transfusion-dependent beta thalassemia
    E.2.2Secondary objectives of the trial
    •Safety and tolerability in adult patients with transfusion-dependent beta thalassemia
    •TSAT
    •Immunogenicity (anti-LJPC-401 antibodies)
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Pharmacokinetic (PK) Sub-study
    An optional PK substudy will be performed in a subset of subjects who provide a separate informed consent (approximately 20).
    Blood sampling for PK will be taken at first dose at Hour -24, Hour 0, and post-dose Hours 0.5, 2, 4, 8, 12 (optional), 24, 48, and 168 (pre-dose before next weekly dosing). Hour 0 and Hour 168 can be combined with the immunogenicity and PK sample collected at Weeks 27 and 28 for Group A and Weeks 1 and 2 for Group B.
    E.3Principal inclusion criteria
    1. Patients ≥ 18 years of age with transfusion-dependent beta thalassemia and must have been transfused within 1 month of randomization.
    2. Patients must have a TSAT ≥ 50% at Screening.
    3. Two separate cardiac MRIs should be greater than 3 weeks but no more than 3 months apart that both demonstrate cardiac T2* MRI of 6 to 35 (msec) with no more than a 10% difference between the first and second reading.
    4. Patients must be receiving iron chelation therapy for a minimum of 1 year and be on a stable dose of maximally tolerated iron chelation therapy for a minimum of 8 weeks prior to study and expected to remain stable during study.
    5. Female patients must be of non-childbearing potential, or using a highly effective method of contraception during participation in the study. Highly effective methods of contraception are defined as those, alone or in combination, that result in a low failure rate when used consistently and correctly. These include: surgical sterility (vasectomy, bilateral tubal ligation, or hysterectomy); hormonal intrauterine device (IUD); hormonal intrauterine system (IUS), implants, or injections; consistent use of an approved oral contraceptive pill (combined progestin/estrogen pill or progestin-only pill); combined intravaginal or transdermal method; and abstinence.
    6. Female patients of childbearing potential must have a negative serum pregnancy test at Screening, and negative urine pregnancy test at Week 1 (ie, prior to initial dosing of study drug).
    7. Male patients must be surgically sterile, or using a highly effective method of contraception during participation in the study. Highly effective methods of contraception are defined as those, alone or in combination, that result in a low failure rate when used consistently and correctly.
    8. Patient must be willing and able to provide written informed consent.
    E.4Principal exclusion criteria
    1. Any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the patient from participating in the study.
    2. Pregnant or lactating women.
    3. Patients taking an immunosuppressive agent (excluding topical over-the-counter steroids) or have a planned surgery (excluding dental surgery or simple dermatologic procedures) during participation in this study or within 2 weeks prior to randomization.
    4. Patients participating in an unapproved investigational drug or investigational therapeutic device study within 30 days prior to randomization, ie, there must be at least 30 days in between the last dose on a prior study and randomization on this trial.
    5. Patients with a concomitant disease, disability or condition, including laboratory abnormality, which may interfere with the conduct of the study, or which would, in the opinion of the investigator, pose an unacceptable risk to the patient in this study, including, but not limited to, alcohol dependency or abuse, drug dependency or abuse, or psychiatric disease.
    6. Patients who are unwilling or unable to comply with the study protocol requirements.
    7. Known hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) infection.
    8. Patients with Child Pugh class C cirrhosis or liver failure.
    9. Patients with severe congestive heart failure (New York Heart Association [NYHA] = Class 4).
    10. Use of erythropoiesis stimulating agents in the past 3 months prior to study entry.
    11. History of allergic reaction to hepcidin or excipients.
    12. Contraindication to MRI scanning.
    E.5 End points
    E.5.1Primary end point(s)
    The mean change from baseline (Screening) in intrapatient cardiac iron level as measured by T2* MRI at week 26
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 26 (comparing 26 weeks of Standard of Care (SOC) [Group A] to 26 weeks of SOC plus LJPC-401 [Group B]).
    E.5.2Secondary end point(s)
    1. Mean change from baseline (Screening) in intrapatient cardiac iron level as measured by cardiac T2* MRI at week 52
    2. Mean change from baseline (Screening) in intrapatient cardiac iron level as measured by cardiac T2* MRI at post-baseline visits other than Week 26 and Week 52.
    3. Change in time-weighted TSAT%.
    4. The proportion of patients with an increase of at least 20% compared to baseline (Screening) in the T2* level as measured by cardiac MRI.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Week 52 (comparing delayed treatment/Group A [26 weeks of SOC followed by 26 weeks of SOC plus LJPC-401] to 52 weeks of immediate treatment/Group B [SOC + LJPC 401])
    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 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) 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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Immediate treatment versus delayed treatment
    E.8.2.4Number of treatment arms in the trial2
    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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    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
    Australia
    Cyprus
    Greece
    Italy
    Lebanon
    Thailand
    Turkey
    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
    Last patient last visit
    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 months7
    E.8.9.1In the Member State concerned days30
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    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: 90
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 47
    F.4.2.2In the whole clinical trial 100
    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 Decision2018-02-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-05-02
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-08-04
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