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    Summary
    EudraCT Number:2020-000134-17
    Sponsor's Protocol Code Number:MT-7117-G02
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-08-18
    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 number2020-000134-17
    A.3Full title of the trial
    A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects with Diffuse Cutaneous Systemic Sclerosis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2, Multicenter, Randomised, Double-Blind, Placebo-Controlled-Parallel-Group study to determine how safe, effective and tolerable MT-7117 is in subjects with Diffuse Cutaneous Systemic Sclerosis
    A.4.1Sponsor's protocol code numberMT-7117-G02
    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 SponsorMitsubishi Tanabe Pharma Development America, Inc. (MTDA)
    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 supportMitsubishi Tanabe Pharma Development America, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMitsubishi Tanabe Pharma Development America, Inc. (MTDA)
    B.5.2Functional name of contact pointAnne Weller
    B.5.3 Address:
    B.5.3.1Street Address525 Washington Blvd, Suite 400
    B.5.3.2Town/ cityJersey City
    B.5.3.3Post codeNJ 07310
    B.5.3.4CountryUnited States
    B.5.4Telephone number +44 7968528013
    B.5.5Fax number +44 2070655050
    B.5.6E-mailaweller@mt-pharma-eu.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 nameDersimelagon
    D.3.2Product code MT-7117
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDersimelagon
    D.3.9.2Current sponsor codeMT-7117
    D.3.9.3Other descriptive nameMT-7117
    D.3.9.4EV Substance CodeSUB181965
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Diffuse Cutaneous Systemic Sclerosis
    E.1.1.1Medical condition in easily understood language
    Diffuse Cutaneous Systemic Sclerosis
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10012977
    E.1.2Term Diffuse systemic sclerosis
    E.1.2System Organ Class 10028395 - Musculoskeletal and connective tissue 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 efficacy of MT-7117 treatment in subjects with diffuse cutaneous systemic sclerosis (dcSSc) using the American College of Rheumatology Composite Response Index in Diffuse Systemic Sclerosis (ACR CRISS) at Week 52.
    E.2.2Secondary objectives of the trial
    •To evaluate the efficacy of MT-7117 treatment for up to 52 weeks using patient-reported outcomes (PROs) as measured by the Health Assessment Questionnaire Disability Index (HAQ-DI) and Patient Global Assessment.
    •To evaluate the efficacy of MT-7117 treatment for up to 52 weeks on pulmonary function as measured by percent predicted forced vital capacity (%pFVC).
    •To evaluate the efficacy of MT-7117 treatment for up to 52 weeks using the Physician Global Assessment.
    •To evaluate the efficacy of MT-7117 treatment for up to 52 weeks using the modified Rodnan Skin Score (mRSS).
    •To evaluate ACR CRISS at Weeks 16, 26, and 39.
    •To evaluate ACR CRISS Score improvement proportion up to 52 weeks.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects who meet all the following criteria will be considered eligible to participate in the study:
    1. Must provide signed and dated informed consent form (ICF) to participate in the study. Subjects must be able to (in the judgment of the Investigator) understand the nature of the study and all risks involved with participation in the study. Subjects must be willing to cooperate and comply with all protocol restrictions and procedures including study visits.
    Male or female age ≥ 18 years inclusive at screening with documented diagnosis of systemic sclerosis (SSc), as defined using the 2013 ACR/European League Against Rheumatism (EULAR) criteria (Appendix 1).
    3. Has diffuse cutaneous form of SSc according to Leroy and Medsger's criteria(Appendix 1).
    4. Disease duration ≤ 5 years from the first non-Raynaud's phenomenon manifestation.
    5. Has an modified Rodnan Skin Score (mRSS) of 15 to 45 units at screening and have clinical skin involvement proximal and distal to the elbows, knees, or both or any truncal involvement, with or without face involvement.
    6. If disease duration is > 24 months defined as time from the first non-Raynaud phenomenon manifestation, subject must fulfil at least 1 of the criteria listed below that are indicatives of active disease at screening:
    a. A documentation of new skin involvement that occurred within the past 9 months, , or
    b. Increase in mRSS ≥ 3 units within the past 9 months, or
    c. Presence of tendon friction rubs (TFRs) or,
    d. C- reactive protein ≥ 6 mg/L, or
    e. Erythrocyte sedimentation rate ≥ 28 mm/hr, or
    f. Platelet count ≥ 330 x 10^9/L (330,000/microliter).
    7. Willing to follow restrictions regarding concomitant medications that are described in Appendix 2.
    8. Female subjects who are non-lactating and have a negative urine pregnancy test at baseline visit prior to receiving the first dose of study drug.
    9. Female subjects of childbearing potential and male subjects with partner of child-bearing potential currently using/willing to use two effective methods of contraception including barrier method as described in Appendix 3.
    E.4Principal exclusion criteria
    1. Has a history or presence of rheumatic autoimmune diseases other than dcSSc unless the dominant features of the current disease are from dcSSc, as determined by the Investigator after the discussion with the Medical Monitor
    2. Has a pulmonary disease with FVC ≤ 50% of predicted at time of screening
    3. Has a diagnosis of clinically significant resting pulmonary hypertension (if exceeding estimated right ventricular systolic pressure of > 40 mmHg estimated by transthoracic echocardiography [unless the right heart catheterization is normal within the last 6 months] or mean pulmonary artery pressure > 30 mmHg as measured by right heart catheterization) and requires treatment with more than one oral medication
    4. Has a cardiac abnormality such as left ventricular failure with ejection fraction < 45%,significant arrhythmia, congestive heart failure (New York Heart Association Class II-IV), unstable angina, uncontrolled hypertension, or symptomatic pericardial effusion at screening
    5. Has a history of myocardial infarction in the last 26 weeks prior to screening
    6. Has a history of renal crisis within the past 52 weeks prior to screening
    7. Has a documented history of chronic kidney disease (stage 4-5, an
    estimated glomerular filtration rate [eGFR] < 30 mL/min at screening)
    8. Presence or history of hepatobiliary disease at screening, determined
    as clinically significant by the Investigator after the discussion with the
    Sponsor Medical Monitor
    9.Aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP) ≥ 2.0 × upper limit of normal (ULN), or total bilirubin>1.5 × ULN at screening
    10. Has a history or presence of clinically significant disease not related to SSc [neurologic, renal, endocrine, gastrointestinal cardiovascular, hepatic, dermatologic, hematological, musculoskeletal, genitourinary, thromboembolic, advanced arteriosclerosis, hyperthyroidism, moderate to severe hypertension, immunologic disease, pulmonary (e.g., uncontrolled asthma, emphysema, chronic obstructive pulmonary disease) or any other disorder] as determined by the Investigator at screening
    11. Has a history or presence of serious or clinically significant (as judged by the Investigator) psychiatric disorder including but not limited to, anxiety disorder, depression, and bipolar disorder that may make a subject unlikely or unable to complete the study or comply with study procedures and requirements, impact the subject’s ability to participate in the study and/or interfere with the study evaluation and/or safety of the subject
    12. Has any clinically significant disease or laboratory abnormality judged to be clinically significant by the Investigator and which may interfere with the study evaluation and/or safety of the subject at screening. Laboratory abnormalities include but not limited to any of the followings: Hemoglobin < 9 g/dL; WBC < 3,000/mm3 (< 3 x 10^9/L); platelets < 100,000/mm3 (<100 x 10^9/L)
    13. Has a history of positive hepatitis B surface antigen, hepatitis C antibody, except for documented cure for the hepatitis B virus (HBV), defined as sustained, undetectable HBsAg and HBV DNA in serum and adequately treated hepatitis C virus (HCV) with documentation of sustained virologic response defined as undetectable HCV RNA at least 12 weeks after the EOT
    14. Has a history of positive human immunodeficiency virus (HIV)
    15. Has a history of melanoma, familial melanoma (defined as having 2 or more first-degree relatives, such as parent, sibling, and/or child), or presence of melanoma and/or lesions suspicious for melanoma at screening
    16. Presence of squamous cell carcinoma, basal cell carcinoma, or other malignant skin lesions. Any suspicious lesions or nevi (Melanocytic Lesions) will be evaluated. If the suspicious lesion or nevi (Melanocytic Lesions) cannot be resolved through biopsy or excision, the subject will be excluded from the study
    17. Has history of any other malignancy(ies) in the last 5 years with the exception of cervical carcinoma in situ
    18. Has a history or planning to receive cell-depleting therapy and or bone marrow transplantation during study treatment period
    19. Has a history of ultraviolet (UV) phototherapy within 6 weeks prior to screening or planning to receive UV phototherapy during study treatment period
    20. Treatment of SSc disease with medication outlined in the protocol
    21. Treatment with afamelanotide or other MC1R agonist within 12 weeks before screening visit 1
    22. Treatment with any drugs or supplements which, in the opinion of the Investigator, may interfere with the objectives of the study or safety of the subject
    23. Has previously exposed to MT-7117 (this does not include placebo treated subjects)

    A full list of the exclusion criteria can be found in section 9.4 of the protocol
    E.5 End points
    E.5.1Primary end point(s)
    The American College of Rheumatology Composite Response Index in Diffuse Systemic Sclerosis (ACR CRISS) composite score (0-1)
    E.5.1.1Timepoint(s) of evaluation of this end point
    at week 52
    E.5.2Secondary end point(s)
    Secondary endpoint
    •Change in HAQ-DI from baseline at Weeks 16, 26, 39, and 52.
    •Change in Patient Global Assessment from baseline at Weeks 16, 26, 39, and 52.
    •Change in percent predicted forced vital capacity (%pFVC) from baseline at Weeks 16, 26, 39, and 52.
    •Change in Physician Global Assessment from baseline at Weeks 16, 26, 39, and 52.
    •Change in mRSS from baseline at Weeks 16, 26, 39, and 52.
    •ACR CRISS Score at Weeks 16, 26, and 39.
    •ACR CRISS Score improvement at Weeks 16, 26, 39 and 52: Proportion of subjects with 25% improvement in mRSS, HAQ-DI, Patient Global Assessment, Physician Global Assessment, or ≥ 5% improvement in FVC for at least 3 of the 5 ACR CRISS measures.
    •ACR CRISS score responder (CRISS ≥ 0.6) at Weeks 16, 26, 39, and 52.


    Safety Endpoint(s)
    •Treatment-emergent adverse events ([TEAEs] including serious adverse events [SAEs], adverse events leading to withdrawal, and adverse events of special interest [AESIs]).
    •Physical examination.
    •Vital signs (blood pressure, pulse rate, respiratory rate, and body temperature).
    •Clinical laboratory examinations (hematology, coagulation, biochemistry, and urinalysis), including liver function markers (ALT, AST, gamma glutamyl transpeptidase [GGT], ALP, direct and total bilirubin).
    •12-lead electrocardiogram (ECG).
    •Nevi (Melanocytic Lesions) appearance (assessed by a dermatologist or other qualified site staff). Any nevi (Melanocytic Lesions) undergoing change of clinical concern during active treatment will be biopsied for follow-up and evaluated by a central pathology laboratory.

    Pharmacokinetics Endpoint
    •Assessment of plasma PK concentrations of MT-7117 measured at scheduled visits
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary Endpoint:
    •Change in HAQ-DI, Patient Global Assessment, %pFVC, mRSS : From baseline at weeks 16, 26, 39 and 52
    • ACR CRISS score: at weeks 16, 26 and 39
    • CRISS Score improvement : at weeks 16, 26, 39 and 52

    Safety Endpoint
    - At study visits

    Pharmacokinetics Endpoint
    - plasma PK concentrations of MT-7117: at study visits
    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 Yes
    E.6.13.1Other scope of the trial description
    tolerability
    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 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 concerned9
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA22
    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
    Canada
    United Kingdom
    United States
    Belgium
    Germany
    Italy
    Poland
    Spain
    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 years2
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days1
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days29
    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: 63
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 13
    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 state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 52
    F.4.2.2In the whole clinical trial 76
    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)
    N/A
    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 Decision2021-09-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-06-24
    P. End of Trial
    P.End of Trial StatusOngoing
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