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    Summary
    EudraCT Number:2019-004226-16
    Sponsor's Protocol Code Number:MT-7117-G01
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-09-15
    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 ConcernedGermany - BfArM
    A.2EudraCT number2019-004226-16
    A.3Full title of the trial
    A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Adults and Adolescents with Erythropoietic Protoporphyria or X-Linked Protoporphyria
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase 3 trial to determine how safe, tolerable and effective MT-7117 is in adults and adolescents with Erythropoietic Protoporphyria or x-linked Protoporphyria
    A.3.2Name or abbreviated title of the trial where available
    RESPITE
    A.4.1Sponsor's protocol code numberMT-7117-G01
    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 (MTDA), 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 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 (MTDA), Inc.
    B.5.2Functional name of contact pointGeneral Information
    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+447968528013
    B.5.5Fax number+442070655050
    B.5.6E-mailaweller@mt-pharma-us.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
    Erythropoietic Protoporphyria or X-Linked Protoporphyria
    E.1.1.1Medical condition in easily understood language
    Erythropoietic Protoporphyria or X-Linked Protoporphyria
    E.1.1.2Therapeutic area Diseases [C] - Symptoms and general pathology [C23]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 24.0
    E.1.2Level LLT
    E.1.2Classification code 10015289
    E.1.2Term Erythropoietic protoporphyria
    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
    To investigate the efficacy of MT-7117 on time to onset and severity of first prodromal symptoms (burning, tingling, itching, or stinging) associated with sunlight exposure in adults and adolescents with Erythropoietic Protoporphyria (EPP)or X-Linked Protoporphyria (XLP).
    E.2.2Secondary objectives of the trial
    - To investigate the effect on patient-reported QOL adult and adolescent with EPP or XLP.
    - To investigate the effect on the percentage of responders based on the within-subject meaningful threshold for time to first prodromal symptom in adults and adolescents with EPP or XLP.
    - To investigate the effect on number and severity of sunlight-induced pain events (prodrome and phototoxic reactions) in adults and adolescents with EPP or XLP.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects provided written informed consent to participate. For minor subjects, both minor assent and parental consent will be provided.
    2. Male and female subjects with a confirmed diagnosis of Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) based on medical history, aged 12 years to 75 years, inclusive, at Screening.
    3. Subjects have a body weight of ≥30 kg.
    4. Subjects are willing and able to travel to the study sites for all scheduled visits.
    5. In the Investigator’s opinion, subject is able to understand the nature of the study and any risks involved in participation, and willing to cooperate and comply with the protocol restrictions and requirements (including travel).
    6. Female subjects who are non-lactating and have a negative urine pregnancy test at baseline prior to receiving the first dose of IMP.
    7. Female subjects of childbearing potential and male subjects with partner of child-bearing potential currently using/willing to use 2 effective methods of contraception including barrier method as described in Section 8.7.1.
    E.4Principal exclusion criteria
    1. History or presence of photodermatoses other than EPP or XLP.
    2. Subjects who are unwilling or unable to go outside during daylight hours most days (e.g., between 1 hour post sunrise and 1 hour pre-sunset) during the study.
    3. Presence of clinically significant hepatobiliary disease based on Liver function test (LFT) values at Screening.
    4. Subjects with AST, ALT, ALP ≥3.0 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN at Screening.
    5. Subjects with or having a history (in the last 2 years) of excessive alcohol, intake in the opinion of the Investigator.
    6. History of melanoma.
    7. Presence of melanoma and/or lesions suspicious for melanoma at Screening.
    8. History of familial melanoma (defined as having 2 or more first-degree relatives, such as parents, sibling and/or child).
    9. Presence of squamous cell carcinoma, basal cell carcinoma, or other malignant skin lesions. Any suspicious lesions or nevi will be evaluated. If the suspicious lesion or nevi cannot be resolved through biopsy or excision, the subject will be excluded from the study.
    10. History or presence of psychiatric disease judged to be clinically significant by the Investigator and which may interfere with the study evaluation and/or safety of the subjects.
    11. Presence of clinically significant acute or chronic renal disease based
    upon the subject's medical records including hemodialysis; an estimated
    glomerular filtration rate (eGFR) <60 ml/min as calculated by the
    Chronic Kidney Disease-Epidemiology Collaboration (CKD-EPI) creatinine
    equation (2009) for adults and by the Schwartz creatinine equation for
    adolescents (2009) . Modification of Diet in Renal Disease (MDRD) can
    be used for adults per local recommendations.
    12. Presence of any clinically significant disease or laboratory abnormality which, in the opinion of the Investigator, can interfere with the study objectives and/or safety of the subjects.
    13. Female subjects who are pregnant, lactating, or intending to become pregnant during the study.
    14. Treatment with phototherapy within 3 months before Randomization (Visit 2).
    15. Treatment with afamelanotide within 3 months before Randomization (Visit 2).
    16. Treatment with cimetidine within 4 weeks before Randomization (Visit 2).
    17. Treatment with antioxidant agents within 4 weeks before Randomization (Visit 2), at doses which, in the opinion of the Investigator, may affect study endpoints (including but not limited to beta-carotene, cysteine, pyridoxine).
    18. Chronic treatment with any scheduled analgesic agents including, but not limited to, opioids and opioid derivatives such as morphine, hydrocodone, oxycodone, Fentanyl or their combination with other unscheduled analgesics or non-steroidal anti-inflammatory drug (Percocet and Vicodin-like prescription drugs) within 4 weeks before Randomization (Visit 2). Acute use of scheduled narcotics greater than 3 months prior to randomization, Over the counter (OTCs), such as Non-steroidal anti-inflammatory drug (NSAIDs) or aspirin for analgesia, or prior temporary use of scheduled agents within 3 months of screening are not excluded.
    19. Treatment with any drugs or supplements which, in the opinion of the Investigator, can interfere with the objectives of the study or safety of the subjects.
    20. Previous exposure to MT-7117 (this does not include placebo treated subjects).
    21. Previous treatment with any investigational agent within 12 weeks before Screening OR 5 half-lives of the investigational product (whichever is longer).
    22. Using the following drugs (including but not limited to) within 1 week of Randomization (Visit 2):
    a. Drugs known to be predominantly metabolized by cytochrome P450 (CYP)3A4 with a narrow therapeutic index for which elevated plasma concentrations are associated with clinical safety concern or significant medical events.
    b. Drugs that are known substrates of P-glycoprotein (P-gp), breast cancer resistance protein (BCRP), organic anion transporing polypeptide (OATP)1B1, or OATP1B3 for which elevated plasma concentrations are associated with significant medical events.
    23. Patients who are legally institutionalized, or patients under judicial protection.
    24. Patients with an immediate family member (i.e., spouse, parent/legal guardian, sibling or a child) being a member of study site staff or being a member of the sponsor’s study team.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in average daily sunlight exposure time (minutes) to first prodromal symptom (burning, tingling, itching, or stinging) associated with sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset at Week 26 (Visit 7).
    E.5.1.1Timepoint(s) of evaluation of this end point
    To calculate the average daily duration, a 14-day window on or before a time-point (Week 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22, 24, and 26) will be used. For baseline, a 14-day window before Day 1 will be used. A 14-day window will be applied to similar situations for other efficacy endpoints related to sunlight exposure diary.
    E.5.2Secondary end point(s)
    1. Patient Global Impression of Change (PGIC) at Week 26.
    2. Total number of sunlight-induced pain events defined as prodrome symptoms (burning, tingling, itching, or stinging) with pain rating of 1-10 on the Likert scale during the 26-week double-blind treatment period.
    3. Change from baseline for total score in the domain of pain intensity in the Patient-reported Outcomes Measurement Informaiton (PROMIS)-57 at Week 26.
    4. The percentage of subjects who are responders based on average daily sunlight exposure time to first prodromal symptom associated with sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset defined by within-subject meaningful change of 66 minutes increase from baseline to Week 26.
    5. Change from baseline for total score in the domain of physical function in the PROMIS-57 at Week 26.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1.Patient Global impression of change (PGIC), change from baseline for total score in domainof pain intensity, change from baseline for total score of physical function: at week 26

    2. Total number of prodrome symptoms and percentage of subjects who are responders based on average daily sunlight exposure: from baseline to week 26.
    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 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) 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 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 trial3
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA14
    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
    Canada
    Japan
    United States
    Germany
    Italy
    Norway
    Spain
    Sweden
    United Kingdom
    E.8.7Trial has a data monitoring committee No
    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 months2
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months2
    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: 20
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 37
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 138
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 9
    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 Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Adolescents
    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 60
    F.4.2.2In the whole clinical trial 184
    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)
    Standard of Care
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation American Porphyria Foundation
    G.4.3.4Network Country United States
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-01-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-04-20
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-07-26
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