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    EudraCT Number:2023-000283-62
    Sponsor's Protocol Code Number:KB103-001
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2023-03-21
    Trial results
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    A. Protocol Information
    A.2EudraCT number2023-000283-62
    A.3Full title of the trial
    A Phase I/II Study of KB103, a Non-Integrating, Replication-Incompetent HSV Vector Expressing the Human Collagen VII
    Protein, for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase I/II Study of KB103, a Non-Integrating, Replication-Incompetent HSV Vector Expressing the Human Collagen VII
    Protein, for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)
    A.3.2Name or abbreviated title of the trial where available
    A Phase I/II Study of KB103, a Topical HSV1-COL7, on DEB Patients ( GEM-1 )
    A.4.1Sponsor's protocol code numberKB103-001
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/464/2022
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorKrystal Biotech
    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 supportKristal Biotech
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationKrystal Biotech
    B.5.2Functional name of contact pointClinical Infomation
    B.5.3 Address:
    B.5.3.1Street Address2100 Wharton St
    B.5.3.2Town/ cityPittsburgh
    B.5.3.3Post code15203
    B.5.3.4CountryUnited States
    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/3/18/2012
    D.3 Description of the IMP
    D.3.1Product nameTopical beremagene geperpavec HSV1-COL7A1 vector
    D.3.2Product code KB103
    D.3.4Pharmaceutical form Gel
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPTopical
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D. cell therapy medicinal product No
    D. therapy medical product Yes
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 Yes
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    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 placeboGel
    D.8.4Route of administration of the placeboTopical
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Dystrophic Epidermolysis Bullosa
    E.1.1.1Medical condition in easily understood language
    Dystrophic epidermolysis bullosa (DEB) is a group of heritable skin diseases characterized by skin fragility, blister formation, milia, and scarring.
    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 10014989
    E.1.2Term Epidermolysis bullosa
    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 objectives were the evaluation of safety, through incidence of adverse events associated with the administration of KB103, as well as the demonstration of molecular correction of the disease by establishing the presence of functional COL7 expression and anchoring fibrils (AF) formation post administration of KB103.
    E.2.2Secondary objectives of the trial
    To assess the proportion of wounds with complete wound closure (≥90% reduction from baseline wound surface area) at Week 8, 10, and 12, the duration of wound closure, and the time to wound closure of kB103 treated wounds as
    compared with placebo treated wounds.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Clinical diagnosis of the recessive form of dystrophic epidermolysis bullosa (RDEB).
    • Age
    a. Phase 1: 18 years old or older,
    b. Phase 2a: 5 years old or older,
    c. Phase 2b: 2 years old or older,
    d. Phase 2c: 2 years old or older.
    • Willing and able to give consent/assent
    • Confirmation of RDEB diagnosis by genetic testing, IF, and IEM
    • LH24 antibody negative (non-collagenous [NC] 2domain [NC2-]) and NC1 domain [NC1+]). (This criterion is applicable to the first 2 adults on the study (Phase 1). Subsequent subjects can be NC1+ or NC1-)
    • Confirmed RDEB COL7A1 mutations in subject
    • Wound that meets the wound size/surface area entry criteria:
    a. Phase 1: Two wounds up to 10 cm2; 1 randomized to B-VEC and 1 randomized to placebo
    b. Phase 2a and 2b: At least 3 wounds up to 20 cm2; 2 wounds randomized to B-VEC and 1 randomized to placebo
    c. Phase 2c: At least 2 wounds up to 50 cm2; at least 1 randomized to B-VEC and 1 randomized to placebo
    • Subjects, who are, in the opinion of the investigator, able to understand the study, cooperate with the study procedures, andare willing to return to the clinic for all the required follow-up visits
    E.4Principal exclusion criteria
    • Medical instability limiting ability to travel to the investigative center
    • The presence of medical illness expected to complicate participation and/or compromise the safety of this technique, such as active infection with human immunodeficiency virus (HIV), hepatitis B (as determined by hepatitis B surface antigen screening), or hepatitis C (as determined by detection of hepatitis C antibodies, or positive result of hepatitis C polymerase chain reaction [PCR] analysis)
    • Serum antibodies to COL7 demonstrated on enzyme-linked immunosorbent assay (ELISA), indirect immunofluorescence microscopy, Western blot, or cell-mediated immunity to enzyme-lined ImmunoSpot® (subjects with negative results within 12 months of screening are eligible)
    • Active infection in the area that will undergo administration
    • Evidence of systemic infection
    • Known allergy to any of the constituents of the product
    • Current evidence or a history of squamous cell carcinoma in the area that will undergo treatment
    • Active drug or alcohol addiction
    • Hypersensitivity to local anesthesia (lidocaine/prilocaine cream)
    • Receipt of chemical or biological study product for the specific treatment of RDEB in the past 3 months
    • Specific wounds that have previously been administered investigational gene or cell therapy
    • Subjects who have taken systemic antibiotics within 7 days
    E.5 End points
    E.5.1Primary end point(s)
    1. Number of Subjects Reported at Least One Adverse Event, Safety Population
    2. Number of Adverse Events Reported, Safety Population
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. baseline to 12 weeks post-baseline
    2. baseline to 12 weeks post-baseline
    E.5.2Secondary end point(s)
    1. Complete Wound Closure Responder, ITT Population
    2. Time to Wound Closure Analysis, ITT Population
    3. Duration of Wound Closure, ITT Population
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. from baseline to Weeks 8, 10, and 12
    2. baseline to complete wound closure
    3. from the complete closure to the first reopening of the same wound
    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 No
    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. 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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E. trial design description
    intra-subject design
    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 Will this trial be conducted at a single site globally? Yes
    E.8.4 Will this trial be conducted at multiple sites globally? No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months12
    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) Yes
    F. of subjects for this age range: 1
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 7
    F.1.3Elderly (>=65 years) No
    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. of subjects incapable of giving consent
    For subjects younger than 18 years old, the Investigator was responsible for obtaining consent from both parents or legal guardians.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 14
    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)
    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 Stanford Dermatology Clinic
    G.4.3.4Network Country United States
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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