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    Summary
    EudraCT Number:2021-003210-39
    Sponsor's Protocol Code Number:SXR001
    National Competent Authority:Finland - Fimea
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-08-20
    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 ConcernedFinland - Fimea
    A.2EudraCT number2021-003210-39
    A.3Full title of the trial
    A phase I/II, multicenter, randomized, double-blind, placebo within-patient controlled, first-time-in-man (FTIM) Proof of Concept (PoC) study to evaluate the safety and efficacy of topically applied SXR1096 skin cream in patients with Netherton syndrome (NS)
    I/II vaiheen satunnaistettu, kaksoissokkoutettu, toimivuuden osoittamiseksi tehtävä monikeskustutkimus, jossa arvioidaan iholle levitettävän SXR1096-emulsiovoiteen turvallisuutta ja tehokkuutta ensimmäisen kerran ihmisellä siten, että Nethertonin oireyhtymää sairastavat tutkittavat käyttävät tutkimuksen aikana sekä lumevalmistetta että vaikuttavaa ainetta sisältävää emulsiovoidetta
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase I/II, multicenter, randomized, double-blind, placebo within-patient controlled study to evaluate the safety and efficacy of SXR1096 skin cream in patients with Netherton syndrome (NS)
    I/II vaiheen satunnaistettu, kaksoissokkoutettu, toimivuuden osoittamiseksi tehtävä monikeskustutkimus, jossa arvioidaan SXR1096-emulsiovoiteen turvallisuutta ja tehokkuutta lumevalmisteeseen verrattuna Nethertonin oireyhtymää sairastavilla tutkittavilla.
    A.4.1Sponsor's protocol code numberSXR001
    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 SponsorSixera Pharma
    B.1.3.4CountrySweden
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportSixera Pharma
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSixera Pharma
    B.5.2Functional name of contact pointMaria Klockare
    B.5.3 Address:
    B.5.3.1Street AddressSkeppsbron 16
    B.5.3.2Town/ cityStockholm
    B.5.3.3Post code11130
    B.5.3.4CountrySweden
    B.5.4Telephone number+46706232505
    B.5.6E-mailklockare@sixerapharma.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.3.2Product code SXR1096
    D.3.4Pharmaceutical form Cream
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPCutaneous use
    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 placeboCutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Netherton-syndrome
    Nethertonin oireyhtymä
    E.1.1.1Medical condition in easily understood language
    Netherton-syndrome
    Nethertonin oireyhtymä
    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 10062909
    E.1.2Term Netherton's syndrome
    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
    The primary objective is to evaluate clinical safety and efficacy of the treatment with the IMP, compared to placebo, in patients with NS.
    E.2.2Secondary objectives of the trial
    Scondary objectives:
    •Skin itching during the treatment period will be assessed by VAS and a multidimensional 5- D pruritus scale (Phan et al. 2012).
    •Skin surface pH and transepidermal water loss (TEWL) will be compared at baseline and EOT.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female patients aged 18 to 65 years at the screening visit and also adolescents (12-17 years) only after initial cohort of 5 adult patients have been treated for at least three days.
    2. Patients/legal guardian endpoint must be willing to provide written informed consent.
    3. Clinical diagnosis of NS including at least 3 out of the 4 following clinical criteria;
    a. Neonatal erythroderma
    b. Bamboo hair and/or alopecia
    c. Chronic atopy specified as food allergy, asthma, rhino conjunctivitis and/or eczema for at least 2 years
    d. Ichthyosis linearis circumflexa
    4. Patients must be willing and able to understand and can comply with study requirements, apply the medication as instructed and be able to complete the study.
    5. Absent LEKTI on immunohistochemistry of skin biopsy and/or confirmed mutation in SPINK5 gene
    6. NS involvement ≥ 20% of Body Surface Area (BSA) required at both the screening and baseline visits.
    7. Investigator Global Assessment (IGA) of two areas to be treated, score ≥3, i.e. moderate or severe for each area required. Each target area approx. 9% of BSA. i.e. equal to one arm.
    8. Female of childbearing potential must either commit true abstinence when this is in line with the preferred and usual lifestyle or use an adequate and approved method of contraception throughout the study and for 4 weeks after the last study drug application. This criterion also applies to a prepubertal female subject who begins menses during the study.
    Adequate and approved methods of contraception applicable for the subject and/or her partner are defined below:
    • Progestogen-only oral hormonal contraception
    • Combination of male or female condom with cap, diaphragm, or sponge with spermicide (double barrier methods)
    • Combined (estrogen- and progestogen-containing) oral, intravaginal, or transdermal hormonal contraception
    • Injectable or implanted hormonal contraception
    • Intrauterine devices or intrauterine hormone-releasing system
    • Bilateral tubal ligation or tube insert (such as the Essure system) at least 3 months before the study
    • Vasectomy of partner at least 3 months before the study
    • Female subjects of non-childbearing potential must meet one of the following criteria:
    • Absence of menstrual bleeding for 1 year prior to screening without any other medical reason
    • Documented hysterectomy, bilateral salpingectomy, or bilateral oophorectomy at least 3 months before screening
    E.4Principal exclusion criteria
    1. Female patient who is pregnant, nursing an infant or planning a pregnancy throughout the course of the study
    2. Patient with any uncontrolled systemic disease. A potential patient in whom therapy for a systemic disease is not yet stabile for at least 3 months will not be considered for entry into the study.
    3. Patient with positive serology tests like HIV, HCV & HBsAg.
    4. Patient with presence of any skin disease that might interfere with the diagnosis or evaluation of the test medications. Cutaneous infection within 1 week before the baseline visit or, any infection requiring treatment with oral or parenteral antibiotics, antivirals, antiparasitics, or antifungals within 2 weeks before the baseline visit.
    5. Patient that has a condition or is in a situation, which in the investigator's opinion may put the patient at significant risk, may confound the study results, or may interfere significantly with the patient's participation in the study.
    6. Use of topical drugs that might alter the course of NS (e.g., topical corticosteroids and topical calcineurin inhibitors) within two weeks before baseline visit.
    7. Patient with a known sensitivity to any of the study treatments and/or their components.
    8. Patient who anticipates a need to use other topical or systemic therapy that might alter the course of NS. Emollients/creams can be used on remaining skin area but not the test areas. Use of topical prescription treatment within 2 weeks prior to initial dosing of study drug. Recent systemic treatment for NS (e.g. systemic corticosteroids, antibiotics, immunosuppressant, biologic and biosimilars treatments). A washout period of 4 weeks will be required for such patients to be eligible to participate in the trial.
    9. Patient who anticipates the need for surgery or hospitalization during the study.
    10. Concurrent involvement in any other clinical study/expanded access program with an investigational drug or device, or participation in a clinical study within 30 days prior to entering the study.
    11. Suspected or confirmed COVID-19 infection within 4 weeks before the screening or baseline visit. Unresolved COVID-19 infection. Planned vaccination for COVID-19 during screening, treatment period or before the follow-up visit.
    E.5 End points
    E.5.1Primary end point(s)
    Primary endpoints:
    • Safety assessment includes adverse events (nature and incidence), physical examination, vital signs, laboratory safety, and local tolerability.
    • Primary efficacy endpoint:
    o The change in Investigator Global Assessment (IGA) score 0-4 (see table in Appendix B) at EOT compared to baseline.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part A) To assess the safety of SXR1096 multiple doses (3 days of treatment) in adults
    Part B) To assess the safety and efficacy of 4 weeks treatment with SXR1096 in adults and adolescents (12-17 years)
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints:
    • The skin condition will be characterized primarily by Ichthyosis Area Severity Index (IASI), which integrates erythema (IASI-E) and scaling (IASI-S); this score has been recently developed and validated by applying it to patients affected with NS (Paller et al., 2017).
    • Skin itching will be assessed by VAS and a multidimensional 5-D pruritus scale (Phan et al., 2012).
    • Skin surface pH and transepidermal water loss (TEWL) will be measured on treated areas.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part A) To assess the safety of SXR1096 multiple doses (3 days of treatment) in adults
    Part B) To assess the safety and efficacy of 4 weeks treatment with SXR1096 in adults and adolescents (12-17 years)
    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) Yes
    E.7.1.1First administration to humans Yes
    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 No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    split-body (placebo within patient)
    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 trial1
    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 No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    LSLV
    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 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: 5
    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: 5
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 15
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 20
    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 Decision2021-11-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-09-08
    P. End of Trial
    P.End of Trial StatusCompleted
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