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    Summary
    EudraCT Number:2017-003598-33
    Sponsor's Protocol Code Number:LJ401-HH01
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-12-22
    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 ConcernedFrance - ANSM
    A.2EudraCT number2017-003598-33
    A.3Full title of the trial
    A Phase 2, Multi-center, Randomized, Placebo Controlled, Double-Blind Study with LJPC-401 for the Treatment of Iron Overload in Adult Patients with Hereditary Hemochromatosis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A multi-center clinical study with LJPC-401 for the treatment of iron overload in adult patients with hereditary hemochromatosis
    A.4.1Sponsor's protocol code numberLJ401-HH01
    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 number+1 858 433 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 No
    D.2.5.1Orphan drug designation number
    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
    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
    Hereditary Hemochromatosis
    E.1.1.1Medical condition in easily understood language
    Iron overload caused by the body making lower than normal amounts of the hormone hepcidin
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level SOC
    E.1.2Classification code 10027433
    E.1.2Term Metabolism and nutrition disorders
    E.1.2System Organ Class 10027433 - Metabolism and nutrition 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 evaluate the effect of weekly dosing of LJPC-401 for 12 weeks on Transferrin saturation (TSAT) in adult patients with hereditary hemochromatosis
    E.2.2Secondary objectives of the trial
    To evaluate the effect of weekly dosing of LJPC-401 for 12 weeks on:
    - Phlebotomy requirements
    - Safety and tolerability in adult patients with hereditary hemochromatosis
    - Serum ferritin
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients with a clinical diagnosis of hereditary hemochromatosis.
    2. Patients who are prescribed therapeutic phlebotomy for the treatment of hereditary hemochromatosis.
    3. Patients ≥ 18 years of age.
    4. Patients with a serum ferritin level from 150 to <1,000 ng/mL.
    5. Patients with a TSAT level > 45%.
    6. 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. Refer to Protocol Section 10.1.6.4.1
    for the list of highly effective methods of contraception that can be used in this study.
    7. Female patients of child bearing potential must have a negative serum pregnancy test at Screening, and negative urine pregnancy test at Day 1 (ie, prior to initial dosing of study drug).
    8. Male patients must be surgically sterile (vasectomy), 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. Refer to Protocol Section 10.1.6.4.1 for the list of highly effective methods of contraception that can be used in this study.
    9. Patient must be willing and able to provide written informed consent.
    E.4Principal exclusion criteria
    1. Patients receiving iron chelation therapy.
    2. Patients initiating phlebotomy treatments less than 6 months prior to the first dose of study drug.
    3. 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.
    4. Pregnant or lactating women.
    5. Patients taking an immunosuppressive agent (excluding topical over-the-counter steroids, and nonsteroidal anti-inflammatory drugs) within 2 weeks prior to randomization.
    6. Patients participating in an unapproved investigational drug or investigational therapeutic device study within 30 days prior to study drug dosing, ie, there must be at least 30 days in between the last dose on a prior study and the first dose administration on this trial.
    7. Patients who are unwilling or unable to comply with the study protocol requirements.
    8. Patients with type 1 or type 2 diabetes.
    9. Patients with uncontrolled active hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) infection.
    10. Patients with Child Pugh class C cirrhosis or liver failure.
    11. Patients with severe congestive heart failure (NYHA = Class 4).
    12. Patients with clinically significant arrhythmia.
    13. Patients who have advanced renal failure with an estimated glomerular filtration rate (eGFR) < 15 mL/min/1.73m2.
    14. Patient with a history of allergic reaction to hepcidin or excipients.
    16. Patients who have planned surgery (excluding dental surgery or simple dermatologic procedures) during participation in this study.
    E.5 End points
    E.5.1Primary end point(s)
    Change in TSAT from baseline to Week 12/end of treatment (EOT)
    E.5.1.1Timepoint(s) of evaluation of this end point
    At screening, weekly from Week 1 to Week 12, and at Week 16
    E.5.2Secondary end point(s)
    • Number of phlebotomy events from Day 2 to EOT
    • Change in serum ferritin from baseline to EOT
    • Treatment-emergent adverse events (TEAEs) from Day 1 (postdose to EOS)
    • Changes in clinical laboratory evaluations including serum iron parameters from baseline to EOS
    • Changes in vital signs, electrocardiograms (ECGs), use of concomitant medications, and physical examinations from baseline to EOS
    • Immunogenicity (anti-drug antibody)/pharmacokinetics from baseline to EOS
    E.5.2.1Timepoint(s) of evaluation of this end point
    At screening, weekly from Week 1 to Week 12, and at Week 16
    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 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) 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA9
    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
    France
    Ireland
    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 years0
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months10
    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: 36
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 24
    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 40
    F.4.2.2In the whole clinical trial 60
    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 Decision2018-03-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-01-11
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-10-28
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