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    Summary
    EudraCT Number:2017-004139-35
    Sponsor's Protocol Code Number:MNK14112096
    National Competent Authority:Bulgarian Drug Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-06-28
    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 ConcernedBulgarian Drug Agency
    A.2EudraCT number2017-004139-35
    A.3Full title of the trial
    A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical Trial in Male Subjects with Muscular Disease
    A.4.1Sponsor's protocol code numberMNK14112096
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03400852
    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 SponsorMallinckrodt ARD 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 supportMallinckrodt ARD Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMallinckrodt ARD Inc.
    B.5.2Functional name of contact pointClinical Trial Lead
    B.5.3 Address:
    B.5.3.1Street Address1425 Route 206
    B.5.3.2Town/ cityBedminster
    B.5.3.3Post codeNJ 07921
    B.5.3.4CountryUnited States
    B.5.4Telephone number+19089979390
    B.5.6E-mailLizelyn.Foley@mnk.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 nameCosyntropin acetate
    D.3.2Product code MNK-1411
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation Yes
    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 INNTETRACOSACTIDE HEXAACETATE
    D.3.9.1CAS number 22633-88-1
    D.3.9.3Other descriptive nameCosyntropin acetate
    D.3.9.4EV Substance CodeSUB35804
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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 placeboSuspension 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
    Duchenne Muscular Distrophy
    E.1.1.1Medical condition in easily understood language
    Muscular Disease
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10013801
    E.1.2Term Duchenne muscular dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic 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 determine the effect of MNK-1411 on motor function in subjects with DMD.
    E.2.2Secondary objectives of the trial
    1-To assess the effect of MNK-1411 on motor skills and strength.
    2-To determine the safety and tolerability of MNK-1411 in subjects with DMD.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects must meet all of the following criteria for inclusion in the study at the Screening Visit and at the Baseline Visit (except as noted):
    1. Each subject’s parent or legal guardian must be adequately informed and understand the nature and risks of the study and must be able to provide a signature and date on the ICF. In addition, each subject who is capable of understanding it must sign an assent form. The
    consent and assent (where applicable) for this study must be signed before any protocol required procedures are performed.
    2. Subjects must be male and between 4 and 8 years of age (inclusive) at the Screening Visit.
    3. Subjects must have a documented diagnosis of DMD confirmed by complete dystrophin deficiency (by immunofluorescence and/or immunoblot), or identifiable mutation in the DMD gene where reading frame can be predicated as “out of frame”, or complete
    dystrophin gene sequencing consistent with DMD; AND in the opinion of the Investigator, a typical clinical profile consistent with DMD.
    4. Subjects taking approved treatments for DMD (by a Health Authority) that target dystrophin gene mutations (eg, eteplirsen or ataluren) may be enrolled in the study if they have been on a stable dose for 30 days prior to the first dose of study drug, and plan to
    remain on that dose throughout the study
    5. Subjects should have serum potassium within the reference range at screening. Subjects who have potassium levels below the lower limit of normal may be given potassium supplements and then retested within the 28 day screening period.
    6. Subjects and/or subjects’ parent or legal guardian must be able to communicate effectively with study personnel.
    7. Subjects and subjects’ parent or legal guardian must be able and willing to follow all protocol requirements and study restrictions.
    8. Subjects and subjects’ parent or legal guardian must be able and willing to return for all study visits.
    E.4Principal exclusion criteria
    Subjects are ineligible for study participation if they meet any of the following criteria at the Screening Visit and at the Baseline Visit (except as note):
    1. Subject is from a vulnerable population (excluding pediatric populations), as defined by the US CFR Title 45, Part 46, Section 46.111(b) and other local and national regulations, including but not limited to, employees (temporary, part-time, full-time, etc) or family members of the research staff conducting the study, or of the sponsor, or of the clinical research organization, or of the IRB/IEC.
    2. Subject has had previous systemic treatment with corticosteroids within 2 months prior to the Screening Visit. Exception: In subjects who were down-titrated to a physiological dose of corticosteroids (ie, 3 mg/m2 of prednisone or deflazacort) a maximum of 1 month of no greater than a physiological dose followed by 1 month completely off corticosteroids prior to the Screening Visit will be acceptable for study entry. Transient previous use of corticosteroids will be evaluated on a case-by-case basis by the sponsor or designee. The use of topical or intra-articular corticosteroids is permitted during the study.
    3. Subject has been treated with immunosuppressants, or mineralocorticoids (including, but not limited to, spironolactone, eplerone, and carrenone) in the 3 months prior to the Screening Visit.
    4. Subject has taken any investigation medication within 30 days or 5 half lifes (whichever is longer) prior to the first dose of study drug.
    5. Subject is unwilling to receive, or is intolerant of, SC injections.
    6. Subject has symptomatic cardiomyopathy in the opinion of the investigator.
    7. Subject is unable to complete the 10 meter Walk/Run test at the Screening and/or Baseline Visit.
    8. Subject has a history of any drug allergy, hypersensitivity, or intolerance to cosyntropin, H.P. Acthar® Gel or other ACTH products.
    9. Subject has a history or evidence of active infection or febrile illness within 7 days prior to the Screening Visit or subject has a clinically significant infection requiring administration of intravenous antibiotics or hospitalization in the 4 weeks prior to the Screening Visit or between the Screening Visit and the first dose of study drug.
    10. Subject has any known contraindication(s) to the class of ACTH products including, but not limited to:
    Asthma.
    Acute psychoses.
    Any primary adrenocortical insufficiency or adrenal cortical hyperfunction.
    Cushing’s Syndrome.
    Any current congestive heart failure (defined as New York Heart Association Functional Class III to IV).
    Peptic ulcer (within 24 weeks prior to the Screening Visit).
    Recent major surgery (within 24 weeks prior to the Screening Visit).
    11. Subject has Type 1 or Type 2 diabetes mellitus.
    12. Subject has a history of chronic active hepatitis including acute or chronic hepatitis B, or acute or chronic hepatitis C.
    13. Subject has a history of tuberculosis (TB) infection, any signs/symptoms of TB, or any close contact with an individual with an active TB infection.
    14. Subject has known immune compromised status (not related to disease/condition under study), including but not limited to, individuals who have undergone organ transplantation or who are known to be positive for the human immunodeficiency virus.
    15. Subject has any solid tumor or hematologic malignancy currently diagnosed or undergoing therapy, or has received therapy for any solid tumor or hematological malignancy in the 5 years prior to the Screening Visit; with the exception of treated and cured basal cell
    carcinoma or treated and cured squamous cell carcinoma of the skin.
    16. Subject has any of the following laboratory abnormalities at the Screening Visit:
    Hemoglobin ≤ 8.0 g/dL.
    Platelets ≤ 50,000 cells/μL.
    Absolute neutrophil count ≤ 1000 cells/μL.
    Glycosylated hemoglobin (HbA1c) > 6.5%.
    Positive Hepatitis B surface antigen (HBsAg) or Hepatitis B core antibody (HBcAb), or positive Hepatitis C virus antibody (HCV). (If HCV is positive at screening, HCV polymerase chain reaction (PCR) will be automatically analyzed. Subjects with a positive HCV must have HCV PCR < 25 IU/mL at the Screening Visit to be eligible).
    Positive or indeterminate interferon gamma release assay (IGRA) for TB.
    17. Subject has any other clinically significant disease, disorder or laboratory abnormality which, in the opinion of the investigator (by its nature or by being inadequately controlled), might put the patient at risk due to participation in the study, or may influence the results of the study or the subject’s ability to complete the study.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in 10 meter walk/run time
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 24
    E.5.2Secondary end point(s)
    1-Change from baseline in quantitative muscle testing, the 4 Stair Climb, Time to Stand, and the North Star Ambulatory Assessment (NSAA)
    2-Summary of general safety profile, including AEs, vital signs, immunogenicity, and laboratory assessments over the entire study.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-Week 24
    2-Over the entire study
    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 No
    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 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 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 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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA25
    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
    Belgium
    Bulgaria
    Canada
    Chile
    Germany
    Israel
    Italy
    Netherlands
    Serbia
    Spain
    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 Follow-up visit:
    -Subjects who complete the study and do not enter the open label extension will have their Follow-up Visit at approximately Week 28.
    -Subjects who complete the open label extension will have their Follow-up Visit at approximately Week 52.
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    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 Yes
    F.1.1Number of subjects for this age range: 132
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 132
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 100
    F.4.2.2In the whole clinical trial 132
    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 Decision2018-08-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-08-06
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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