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    Summary
    EudraCT Number:2018-001009-98
    Sponsor's Protocol Code Number:allo-APZ2-EB-II-01
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-05-03
    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 - PEI
    A.2EudraCT number2018-001009-98
    A.3Full title of the trial
    An interventional, multicenter, single arm, phase I/IIa clinical trial to investigate the efficacy and safety of allo-APZ2-EB on epidermolysis bullosa (EB)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to investigate the efficacy and safety of allo-APZ2-EB on wound healing of epidermolysis bullosa (EB)
    A.4.1Sponsor's protocol code numberallo-APZ2-EB-II-01
    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 SponsorRHEACELL GmbH & Co. KG
    B.1.3.4CountryGermany
    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 supportRHEACELL GmbH & Co. KG
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRHEACELL GmbH & Co. KG
    B.5.2Functional name of contact pointInformation Office
    B.5.3 Address:
    B.5.3.1Street AddressIm Neuenheimer Feld 517
    B.5.3.2Town/ cityHeidelberg
    B.5.3.3Post code69120
    B.5.3.4CountryGermany
    B.5.4Telephone number496221718330
    B.5.5Fax number4962217183329
    B.5.6E-mailoffice@rheacell.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 nameallo-APZ2-EB
    D.3.2Product code allo-APZ2-EB
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeT202-3
    D.3.9.3Other descriptive nameAllogeneic skin-derived ABCB5-positive mesenchymal stem cells
    D.3.10 Strength
    D.3.10.1Concentration unit million organisms/ml million organisms/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Recessive dystrophic epidermolysis bullosa (RDEB)
    E.1.1.1Medical condition in easily understood language
    Epidermolysis bullosa patients (butterfly children) have extremely fragile skin and innermost linings of the gastric and intestinal tract, leading to blisters, erosions and open wounds.
    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 aim of this clinical trial is to investigate the efficacy (by monitoring overall improvement of EB symptoms measured by epidermolysis bullosa disease activity and scarring index [EBDASI] score and instrument for scoring clinical outcome of research for epidermolysis bullosa [iscorEB], wound healing assessed by photo documentation, and patients quality of life in EB [QOLEB] assessment) and safety (by monitoring adverse events [AEs]) of the IMP allo APZ2 EB administered intravenously to patients with EB in three applications (Day 0, Day 17 ±3, Day 35 ±3).
    E.2.2Secondary objectives of the trial
    Not applicable.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female patients aged between ≥12 months and ≤55 years,
    Staggered design for patient enrollment:
    1.) at least 3 adult patients ≥18 to ≤55 years (safety assessment 2 weeks after last treatment of third patient),
    2.) at least 3 patients ≥12 to <18 years (safety assessment 2 weeks after first treatment of third patient),
    3.) at least 3 patients ≥5 to <12 years (safety assessment 2 weeks after first treatment of third patient) and
    4.) at least 3 patients ≥12 months to <5 years (safety assessment 2 weeks after first treatment of third patient);
    2. Diagnosed with RDEB (combined diagnoses by genotype assessment [mutation analysis] and correlating phenotype assessment [wound assessment]), patients must have a negative immunofluorescence test result on salt-split skin against proteins of the basement membrane at Visit 1 (existing test results will be accepted);
    3. Patient is eligible to participate in this clinical trial based on general health condition at the investigator’s discretion;
    4. Patient/legal representative understands the nature of the procedure and are providing written informed consent prior to any clinical trial procedure;
    5. Women of childbearing potential must have a negative urine pregnancy test at Visit 1;
    6. Women of childbearing potential and their partner must be willing to use highly effective contraceptive methods during the course of the clinical trial.
    E.4Principal exclusion criteria
    1. Tumor diseases or history of tumor disease;
    2. Positive test result for human immunodeficiency virus 1 and/or 2 at Screening;
    3. Any known allergies to components of the IMP;
    4. Evidence of any other medical conditions (such as psychiatric illness or active infection) based on physical examination, or laboratory findings that may interfere with the planned treatment, affect the patient’s compliance, or place the patient at high risk of complications related to the treatment; at investigators discretion;
    5. Inadequate pulmonary function with evidence of chronic obstructive or severe restrictive pulmonary disease;
    6. Inadequate cardiac function with evidence of uncontrolled high blood pressure, congestive heart failure, angina pectoris, acute myocardial infarction within 6 months prior to treatment;
    7. History of prior thrombosis or patients at risk for thrombosis
    8. Clinically significant or unstable concurrent disease or other clinical contraindications to IMP application (based upon investigator’s judgment);
    9. Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol;
    10. Pregnant or lactating women;
    11. Current or previous (within 30 days of enrollment) treatment with another IMP, or participation and/or under follow-up in another clinical trial;
    12. Previous participation in this clinical trial (except for screening failures due to an exclusion criterion);
    13. Known abuse of alcohol, drugs, or medicinal products;
    14. Employees of the sponsor, or employees or relatives of the investigator.
    E.5 End points
    E.5.1Primary end point(s)
    1. Efficacy endpoint:
    Overall improvement of EB symptoms after 12 weeks (measured by percentage change of a patient’s EBDASI score), or last available post-baseline measurement if the Week 12 measurement is missing (last observation carried forward [LOCF]).
    2. Safety endpoint:
    Adverse events.
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Week 12, after first IMP application or timepoint of last observation carried forward.
    2. During all patient visits except screening.
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints:
    1. Overall improvement of EB symptoms after 12 weeks (measured by percentage change of a patient’s EBDASI score, without LOCF);
    2. Overall improvement of EB symptoms after 12 weeks (measured by percentage change of patient’s iscorEB), or last available post-baseline measurement if the Week 12 measurement is missing (LOCF);
    3. Overall improvement of EB symptoms after 12 weeks (measured by percentage change of patient’s iscorEB, without LOCF);
    4. Overall improvement of EB symptoms at Day 17 (measured by percentage change of a patient’s EBDASI score and patient’s iscorEB);
    5. Overall improvement of EB symptoms at Day 35 (measured by percentage change of a patient’s EBDASI score and patient’s iscorEB);
    6. Inflammation (measured by panel of inflammation markers);
    7. Pain assessment as per numerical rating scale (NRS);
    8. Itch assessment as per NRS;
    9. Assessment of QOLEB.

    Secondary safety endpoint:
    10. Physical examination and vital signs until Week 12;
    11. Overall survival at month 12.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Week 12, after first IMP application;
    2. Week 12, after first IMP application or timepoint of last observation carried forward;
    3. Week 12, after first IMP application;
    4. Day 17, after first IMP application;
    5. Day 35, after first IMP application;
    6. Day 0, 17 and 35 and week 12 after first IMP application;
    7. Day 17 and 35 and week 12, after first IMP application;
    8. Day 17 and 35 and week 12, after first IMP application;
    9. Day 17 and 35 and week 12, after first IMP application;
    10. Day 17, 35 and week 12 after first IMP application;
    11. Month 12, after first IMP application;
    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 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 Yes
    E.7.1.3.1Other trial type description
    Safety and efficacy in patients
    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 No
    E.8.1.1Randomised No
    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.8.1.7.1Other trial design description
    Patients are enrolled in a staggered fashion in 4 age cohorts
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA7
    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
    Austria
    France
    Germany
    Italy
    United Kingdom
    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 years3
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    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: 9
    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.1.1.5.1Number of subjects for this age range: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number 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 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 14
    F.4.2.2In the whole clinical trial 16
    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 withdrawal patients will receive standard medical care according to individual needs.
    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 Decision2019-03-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-08-02
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-11-26
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