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    Summary
    EudraCT Number:2018-003272-12
    Sponsor's Protocol Code Number:18-ICH-001
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-03-14
    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 ConcernedUK - MHRA
    A.2EudraCT number2018-003272-12
    A.3Full title of the trial
    A Phase 2 Randomized, Multi-center, Double-blind, Vehicle-controlled, 12-Week, Safety, Efficacy, and Systemic Exposure Study followed by a 12-Week Open-label Extension of Trifarotene (CD5789) Cream HE1 in Adults and Adolescents with Autosomal Recessive Ichthyosis with Lamellar Scale
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study of Trifarotene Cream in Adults and Adolescents with With Lamellar Ichthyosis
    A.3.2Name or abbreviated title of the trial where available
    ASafetyEfficacyandSystemicExposureStudyofCD5789CreaminAdultsandAdolescentsWithLamellarIchthyosis
    A.4.1Sponsor's protocol code number18-ICH-001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03738800
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/325/2019
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorMayne Pharma LLC
    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 supportMayne Pharma
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMayne Pharma LLC
    B.5.2Functional name of contact pointPhoevos Hughes
    B.5.3 Address:
    B.5.3.1Street Address3301 Benson Drive, Suite 401
    B.5.3.2Town/ cityRaleigh
    B.5.3.3Post codeNC 27609
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1919 573 7948
    B.5.6E-mailPhoevos.Hughes@maynepharma.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/3/20/2264
    D.3 Description of the IMP
    D.3.1Product nameTrifarotene (CD5789) cream HE1
    D.3.2Product code CD5789
    D.3.4Pharmaceutical form Cream
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPTopical use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTRIFAROTENE
    D.3.9.1CAS number 895542-09-3
    D.3.9.2Current sponsor codeCD5789
    D.3.9.3Other descriptive nameTrifarotene (CD5789) cream HE1
    D.3.9.4EV Substance CodeSUB184480
    D.3.10 Strength
    D.3.10.1Concentration unit % percent
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.01
    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.IMP: 2
    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/3/20/2264
    D.3 Description of the IMP
    D.3.1Product nameTrifarotene (CD5789) cream HE1
    D.3.2Product code CD5789
    D.3.4Pharmaceutical form Cream
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPTopical use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTRIFAROTENE
    D.3.9.1CAS number 895542-09-3
    D.3.9.2Current sponsor codeCD5789
    D.3.9.3Other descriptive nameTrifarotene (CD5789) cream HE1
    D.3.9.4EV Substance CodeSUB184480
    D.3.10 Strength
    D.3.10.1Concentration unit % percent
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.02
    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 placeboCream
    D.8.4Route of administration of the placeboTopical use (Noncurrent)
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Lamellar ichthyosis (LI)
    Autosomal Recessive Ichthyosis with Lamellar Scale
    E.1.1.1Medical condition in easily understood language
    Lamellar ichthyosis (LI)
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10023686
    E.1.2Term Lamellar ichthyosis
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To compare the safety and efficacy of 2 concentrations of trifarotene cream HE1 versus vehicle in adults and adolescents with moderate to severe autosomal recessive ichthyosis with lamellar scale, also known as lamellar ichthyosis (LI) after 12 weeks of treatment.
    E.2.2Secondary objectives of the trial
    • To assess systemic exposure to trifarotene and its major metabolites after topical application of the investigational product (IP) on up to 90% body surface area (BSA) twice weekly.
    • To assess safety for up to 24 weeks of dosing with open-label trifarotene cream HE1 200 μg/g.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    PK substudy will be conducted on Days 1 and 30 at sites with the capability to conduct it. Participation in the PK substudy will be optional and will include at least 30 subjects, 15 adults and 15 adolescents. Subjects who participate in the PK substudy will come from both study cohorts and will undergo serial blood sampling predose and at 1, 2, 4, 8, 12, and 24 hours postdose on Day 1 and Day 30. For the subjects in the PK substudy, postdose ECGs will be performed at each serial blood draw on Day 1 and Day 30.
    E.3Principal inclusion criteria
    1. For Cohort A: subject is ≥18 years old; for Cohort B: subject is ≥12 years old.
    2. Subject has known diagnosis of LI.
    3. Subject has moderate to severe (IGA 3-4) LI on the IGA of LI severity
    4. Subject has signed an ICF at Screening before any investigational procedures.
    Subjects <18 years of age (or Age of Majority) must sign an assent form in conjunction with an ICF signed by the parent/legal representative.
    5. Subject who is participating in photography has signed a photography ICF.
    6. Subject who is participating in the optional PK substudy has signed a PK ICF.
    7. Subject is not of childbearing potential, i.e., a female who has not yet begun menstruating or who is postmenopausal (absence of menstrual bleeding for 1 year oophorectomy),
    OR
    Subject is a woman of childbearing potential (WOCBP) or a male subject with sexual partners capable of reproduction who agrees to use 2 effective forms of contraception during the study and for at least 1 month after the last study drug application. The 2 authorized forms of contraception are condom used with 1 of the following methods of contraception:
    • bilateral tubal ligation
    • combined oral contraceptives (estrogens and progesterone) or implanted or injectable contraceptives with a stable dose for at least 1 month before Baseline; hormonal contraceptives must inhibit ovulation
    • hormonal intrauterine device (IUD) inserted at least 1 month before Baseline
    OR
    Agrees to abstain from heterosexual intercourse during study participation and for 1 month after the last application of study drug and to use a highly effective contraceptive as backup if he or she becomes sexually active during the study. Abstinence is only acceptable if this is the subject’s usual lifestyle. Periodic abstinence (calendar, symptothermal, postovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhoea method are not acceptable methods of contraception.
    AND
    Male subjects may not donate sperm during the study and for at least 1 month after the last study drug application.
    Note: Subjects who are premenstrual at Screening but begin menses during the study should follow the pregnancy testing schedule for WOCBP and must abstain from sexual intercourse while in the study and for at least 1 month after the last study drug application.
    8. Women of child-bearing potential must be nonlactating and have negative pregnancy test results at Screening (serum) and on Day 1 before study drug administration (urine).
    9. Subject is reliable and capable of adhering to the protocol and visit schedule, in the investigator’s judgment, and has signed informed consent/assent, as applicable.
    10. Subject is taking no more than 3500 IU/day Vitamin A (e.g., as in a multivitamin).
    E.4Principal exclusion criteria
    1. Subject has any variant of ichthyosis other than LI or another disorder of keratinization including syndromic ichthyoses.
    2. Subject has current moderate or severe stinging/burning at Screening.
    3. Subject has an ongoing cutaneous infection or any other significant concomitant skin disease (other than the LI) which, in the investigator’s opinion, may interfere with the study assessments.
    4. Subject with a known lipid disorder (hypertriglyceridemia >200 mg/dL, hypercholesterolemia >250 mg/dL) unless well controlled by stable doses of lipid-lowering agents for at least 6 months.
    5. Subject was previously treated with trifarotene/CD5789, in an acne or ichthyosis study.
    6. Subject has any other significant concomitant disease, or poorly controlled medical condition other than LI that in the investigator’s opinion may put him or her at risk if he or she takes part in the study, and/or that may interfere with the study assessments.
    7. Subject has a medical condition that potentially alters bone metabolism (e.g., osteoporosis, thyroid dysfunction, Cushing syndrome, Crohn’s disease, or ulcerative colitis).
    8. Subject is being treated for major depression disorder and/or has a history of major depression or suicide attempt requiring hospitalization, medications, and close psychiatric surveillance to prevent suicide attempts.
    9. Subject with positive serology for hepatitis B surface antigen, hepatitis C, or are known to be HIV positive or to have AIDS at Screening.
    10. Subject with any of the following laboratory values at Screening:
    a. Aspartate aminotransferase or alanine aminotransferase >1.5 × upper limit of normal defined by the laboratory
    b. Total bilirubin >1.1 mg/dL or, in case of Gilbert’s syndrome, total bilirubin >3 mg/dL
    c. Hemoglobin <12.5 g/dL for men and <11.5 g/dL for women
    d. Platelets <150 × 109/L or >400 × 109/L.
    11. Subject has any clinically other significant abnormal laboratory value (hematology,chemistry, or urinalysis) at Screening that, in the investigator’s opinion, may put the subject at risk if he or she takes part in the study, and/or that may interfere with the study assessments.
    12.Subject has had recent systemic malignancy (e.g., within 5 years) with exception of nonmelanoma skin cancer or cervical intraepithelial neoplasia of Grade 1 who are >6 months post-treatment
    13. Subject has a history of long QT syndrome or clinically significant electrocardiogram (ECG) abnormalities, including clinically significant conduction disorders or significant arrhythmias, QTcF interval >450 ms, PR interval is not between 120 and 220 ms (inclusive), HR >100 bpm or <50 bpm, QRS interval >110 ms, or QT intervals that cannot be consistently analyzed.
    14. Subject has a known allergy or sensitivity to any of the components of the investigational products.
    15. Subject has been exposed to excessive ultraviolet (UV) radiations on the treated zones within 1 month before Baseline visit or who is planning intensive UV exposure during the study (e.g., occupational exposure to the sun, sunbathing, phototherapy, etc.).
    16. Subject is inherently sensitive to sunlight.
    17.Subject is unable or unwilling to stop use of topical or systemic retinoids
    18. Subject is presumed to be abusing drugs or alcohol at Screening or Baseline Visits based on medical history or current clinical symptoms.
    19. Subject is participating in another interventional clinical trial.
    20. Subject is institutionalized
    21.Subject is in any way related to the sponsor, investigator, or site personnel
    E.5 End points
    E.5.1Primary end point(s)
    The proportion of subjects in each treatment group who experience successful resolution of LI where “success” is defined as clear/almost clear on treated areas and at least a 2-grade change from Baseline at Week 12/end-of-treatment (EOT) in the Double-blind Period on the 5-point IGA full body scale
    E.5.1.1Timepoint(s) of evaluation of this end point
    The primary time point is Day 90 for double blind and day 194 for open label part of the study
    E.5.2Secondary end point(s)
    • The difference in mean scores between the active trifarotene cream HE1 and vehicle groups in the following assessment indices from Baseline through Week 12:
    -5-point Visual Index for Ichthyosis Severity (VIIS) for scaling from Baseline through Week 12 (overall 16 points for scaling, i.e. 0-4 points for 4 body areas: chest/abdomen, back, arms and legs)
    -Individual score for roughness (Scale: 0–4) overall
    -Palm/sole Assessment (Scale: 0–4)
    -Quality of life per Dermatology Life Quality Index (DLQI) and children’s DLQI (cDLQI)
    • The difference in proportion of subjects with presence of fissures on palms/soles (presence/absence, number of fissures, and pain associated with fissures on a 0 3 scale) at Week 12 between the active trifarotene cream HE1 and vehicle groups
    Exploratory endpoints:
    • The difference in mean ectropion (Ectropion Severity Score [ESS] of 0–8) scores between the active trifarotene cream HE1 and vehicle groups from Baseline through Week 12
    • The difference in quality of life per EQ-5D-5L and EQ-5D-Y score between the active trifarotene cream HE1 and vehicle groups from Baseline through Week 12
    Safety endpoints:
    • Reported serious adverse events (SAEs), treatment-emergent AEs (TEAEs), and changes in clinical laboratory tests, vital signs, physical examinations, and 12-lead ECGs
    • Local tolerability (Scale: 0–3 [none, mild, moderate, severe], determined by the investigator) for each body area (chest/abdomen, back, legs, arms, and face/neck).
    Pharmacokinetic endpoints: Plasma concentrations of CD5789 and its major metabolites will be measured.
    E.5.2.1Timepoint(s) of evaluation of this end point
    secondary and exploratory efficacy endpoints as well as PK endpoints will be presented for all study visits (Day 1,14,30,60,90 and 104,120,150,180,194). Safety endpoints will be monitored throughout 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 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 Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Double-blind 12-Week study followed by 12-Week Open-label extension study
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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
    Canada
    France
    Germany
    Israel
    Spain
    Ukraine
    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
    LPLV
    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 months2
    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 months2
    E.8.9.2In all countries concerned by the trial days30
    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: 40
    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: 40
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 70
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 56
    F.4.2.2In the whole clinical trial 120
    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)
    after the subject has ended the participation in the trial he will receive the standard treatment of his condition
    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 DST DIGITAL SCIENCE TECHNOLOGIES
    G.4.3.4Network Country United States Minor Outlying Islands
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-08-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-10-14
    P. End of Trial
    P.End of Trial StatusOngoing
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