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    Summary
    EudraCT Number:2019-000752-34
    Sponsor's Protocol Code Number:DSLP-01
    National Competent Authority:Czechia - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-06-02
    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 ConcernedCzechia - SUKL
    A.2EudraCT number2019-000752-34
    A.3Full title of the trial
    A Phase 2/3, multi-center, double-blind, placebo-controlled, randomized, parallel-group, dose-response comparison of the efficacy and safety of a topical rapamycin cream for the treatment of facial angiofibromas (FA) associated with Tuberous Sclerosis Complex (TSC) in patients 6 years of age and over
    Fáze 2/3, multicentrické, dvojitě zaslepené, placebem kontrolované, randomizované klinické hodnocení u paralelních skupin porovnávající dávky léku a odezvy na účinnost a bezpečnost lokálního rapamycinového krému při léčbě faciálních angiofibromů (FA) spojených s Komplexem Tuberózní Sklerózy (TSC) u pacientů ve věku 6 a více let
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to evaluate if the study medication, Rapamycin, topical cream, is effective and safe at treating facial angiofibroma in patients 6 years of age and over.
    Klinické hodnocení hodnotící vliv hodnoceného léčiva, topický krém s rapamycinem, na účinnost a bezpečnost při léčbě faciálního angiofibromu (FA) u pacientů starších 6 let
    A.3.2Name or abbreviated title of the trial where available
    Dose-ranging efficacy and safety study of topical rapamycin cream
    A.4.1Sponsor's protocol code numberDSLP-01
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03826628
    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 SponsorDermatology Specialities Limited Partnership (DSLP)
    B.1.3.4CountryNew Zealand
    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 supportDermatology Specialities Limited Partnership (DSLP)
    B.4.2CountryNew Zealand
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDermatology Specialities Limited Partnership (DSLP)
    B.5.2Functional name of contact pointIoana Stanescu
    B.5.3 Address:
    B.5.3.1Street AddressLevel 1, Nielsen Building 129, Hurtsmere Road, Takapuna
    B.5.3.2Town/ cityAuckland
    B.5.3.3Post code0622
    B.5.3.4CountryNew Zealand
    B.5.4Telephone number006494880232
    B.5.5Fax number006494880234
    B.5.6E-mailioana@aftpharm.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/3/17/1886
    D.3 Description of the IMP
    D.3.1Product nameRapamycin cream, topical, 0.5%, 1.0% w/w
    D.3.2Product code Not applicable
    D.3.4Pharmaceutical form Cream
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPTopical use (Noncurrent)
    Cutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSIROLIMUS
    D.3.9.1CAS number 53123-88-9
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/W) percent weight/weight
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.5
    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/3/17/1886
    D.3 Description of the IMP
    D.3.1Product nameRapamycin cream, topical, 0.5%, 1.0% w/w
    D.3.2Product code Not applicable
    D.3.4Pharmaceutical form Cream
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPTopical use (Noncurrent)
    Cutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSIROLIMUS
    D.3.9.1CAS number 53123-88-9
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/W) percent weight/weight
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1.0
    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
    Facial Angiofibromas Associated with Tuberous Sclerosis Complex
    E.1.1.1Medical condition in easily understood language
    Facial Angiofibroma
    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 PT
    E.1.2Classification code 10002429
    E.1.2Term Angiofibroma
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10073678
    E.1.2Term Juvenile angiofibroma
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The Primary Efficacy Objective is to determine and compare the efficacy of two different strengths of Rapamycin cream, topical and placebo over 26 weeks of once-daily treatment for Facial Angiofibromas associated with Tuberous Sclerosis Complex (TSC).
    E.2.2Secondary objectives of the trial
    The Safety Objective of the study is to determine and compare the incidence of treatment-emergent adverse events (TEAEs) over the course of the study among the three treatment groups.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male and female patients aged > 6 years and < 65 years on the day informed consent is obtained
    2. Patients diagnosed with TSC based on the clinical diagnostic criteria of International Tuberous Sclerosis Complex Consensus Conference 2012 and presenting visible facial angiofibroma
    3. An FA severity score of 2 or 3 on the IGA scale
    4. Patients or their legal representatives capable of understanding the explanation of the clinical trial and who give written informed consent for participation
    5. Patients or their legal representatives able to maintain patient diaries following the instructions of the investigator or sub-investigator
    E.4Principal exclusion criteria
    1. Patients who cannot carry out the treatment plan or follow-up assessment
    2. Patients with serious skin lesions such as erosions or ulcers
    3. Patients with known hypersensitivity to any component of the study product
    4. Patients who have received rapamycin/sirolimus, everolimus, or temsirolimus within 3 months of enrolment
    5. Patients who received laser therapy or surgical therapy within 6 months prior to trial enrolment
    6. Patients who participated in any other clinical trial within 3 months prior to the day of enrolment
    7. Patients judged unsuitable for this clinical trial by the investigator or sub-investigator
    8. Pregnant or lactating females
    9. Sexually active females of childbearing potential not using adequate contraception and sexually active males not using adequate contraception*
    10. Patients with immune dysfunction or receiving any form of immunosuppression
    11. Patients with severe FA, with a score of 4 on the IGA scale
    12. Patients with an FA severity score of less than 2 on the IGA scale
    E.5 End points
    E.5.1Primary end point(s)
    The percentage of patients obtaining successful treatment based on a blind assessment using the IGA scale after 26 weeks treatment or at last visit if early withdrawal/discontinuation
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 26 weeks treatment
    E.5.2Secondary end point(s)
    - Time to treatment success
    - Change from baseline in IGA after 26 weeks treatment or at last visit if early withdrawal/discontinuation
    - Change from baseline in FASI after 26 weeks treatment or at last visit if early withdrawal/discontinuation
    - Subjective (patient or parent/caregiver) improvement rating from first visit (V0) after 26 weeks treatment or at last visit if early withdrawal/discontinuation
    - Objective (clinician) improvement rating from first visit (V0) after 26 weeks treatment or at last visit if early withdrawal/discontinuation
    - Categorical Improvement of Facial Angiofibroma from first visit (V0) after 26 weeks treatment
    E.5.2.1Timepoint(s) of evaluation of this end point
    After 2, 8, 14, 20 and 26 weeks of treatment
    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 No
    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) 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 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 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 EEA5
    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
    Czech Republic
    Hungary
    New Zealand
    Serbia
    Slovakia
    Spain
    United States
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months3
    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: 90
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 30
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 60
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 30
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children, Subjects with Intellectual Disability
    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 52
    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)
    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 Decision2020-09-11
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-07-12
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