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    Summary
    EudraCT Number:2017-002766-50
    Sponsor's Protocol Code Number:ProTrans-T1D
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2017-07-25
    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 ConcernedSweden - MPA
    A.2EudraCT number2017-002766-50
    A.3Full title of the trial
    A DOUBLE-BLINDED, RANDOMIZED, PARALLEL, PLACEBO-CONTROLLED TRIAL OF WHARTON’S JELLY DERIVED ALLOGENEIC MESENCHYMAL STROMAL CELLS TO PRESERVE ENDOGENOUS INSULIN PRODUCTION IN ADULT PATIENTS DIAGNOSED WITH TYPE 1 DIABETES.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Wharton´s jelly derived mesenchymal stromal cell treatment of adult patients diagnosed with type I diabetes.
    A.4.1Sponsor's protocol code numberProTrans-T1D
    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 SponsorNextCell Pharma
    B.1.3.4CountrySweden
    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 supportNextCell Pharma
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNextCell Pharma
    B.5.2Functional name of contact pointMathias Svahn
    B.5.3 Address:
    B.5.3.1Street AddressKarolinska Institutet Science Park, Hälsovägen 7
    B.5.3.2Town/ cityHuddinge
    B.5.3.3Post codeSE-141 57
    B.5.3.4CountrySweden
    B.5.4Telephone number004608735 20 10
    B.5.6E-mailmathias.svahn@nextcellpharma.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 nameProTrans
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSuspension for injection
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Type 1 diabetes
    Typ 1 diabetes
    E.1.1.1Medical condition in easily understood language
    Type 1 diabetes
    Typ 1 diabetes
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10012594
    E.1.2Term Diabetes
    E.1.2System Organ Class 100000004861
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To investigate the safety and tolerance after allogeneic infusion of WJMSCs intravenously in adult patients diagnosed with type 1 diabetes.
    E.2.2Secondary objectives of the trial
    Study changes in beta-cell function, metabolic control and Diabetes Treatment Satisfaction during one year.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written informed consent for participation of the study, given before undergoing any study-specific procedures
    2. Clinical history compatible with type 1 diabetes diagnosed less than 2 years before enrolment
    3. In the first part of the study patients 1-6 only male patients between 18-40 years of age will be included. In the second part of the study, patients 7-21, both male and female patients 18 to 40 years of age (inclusive at both ends) will be included.
    4. Mentally stable and, in the opinion of the investigator, able to comply with the procedures of the study protocol
    5. Fasting plasma C-peptide concentration >0.12 nmol/L.
    6. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, if they are using effective methods of contraception during the study. Acceptable birth control methods are those with a failure rate of less than 1% per year when used consistently and correctly. Such methods include (in “Recommendations related to contraception and pregnancy testing in clinical trials”, supplied from www.hma.eu/):
    a. Combined (estrogen and progestogen containing hormonal contraception associated with inhibition of ovulation.
    -oral
    -intravaginal
    -transdermal
    b. progestogen-only hormonal contracption associated with inhibition of ovulation
    - oral
    - injectable
    - implantable
    c. intrauterine device (IUD)
    d. intrauterine hormone-releasing system (IUS)
    e. bilateral tubal occlusion
    f. total abstinence or vasectomized partner.
    E.4Principal exclusion criteria
    1. Inability to provide informed consent
    2. Patients with body mass index (BMI) > 30, or weight >100 kg
    3. Patients with weight <50 kg
    4. Patients with unstable cardiovascular status incl. NYHA class III/IV or symptoms of angina pectoris.
    5. Patients with uncontrolled hypertension (≥160/105 mmHg).
    6.Patients with active on-going infections.
    7. Patients with latent or previous as well as on-going therapy against tuberculosis, or exposed to tuberculosis or has travelled in areas with high risk of tuberculosis or mycosis within the last 3 months.
    8. Patients with serological evidence of infection with HIV, Treponema pallidum, hepatitis B antigen (patients with serology consistent with previous vaccination and a history of vaccination are acceptable) or hepatitis C.
    9. Patients with any immune suppressive treatment
    10. Patients with known demyelinating disease or with symptoms or physical examination findings consistent with possible demyelinating disease-
    11. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test
    11. Patients with known, or previous, malignancy.
    12. Taking oral anti-diabetic therapies or any other concomitant medication which may interfere with glucose regulation other than insulin
    13. Patient with any condition or any circumstance that in the opinion of the investigator would make it unsafe to undergo treatment with MSC.
    14. Patients with GFR <80 ml/min/1.73 m2 body surface.
    15. Patients with proliferative retinopathy
    16. Patient with any condition or any circumstance that in the opinion of the investigator would make it unsafe to undergo treatment with MSC.
    17. Known hypersensitivity against any excipients, i.e. dimethyl sulfoxide (DMSO).
    E.5 End points
    E.5.1Primary end point(s)
    Primary Safety Endpoint:

    The primary safety endpoint in this study is; safety parameters include adverse events and hypoglycemia, allergic reactions, ophthalmologic examination, ECG, vital signs, laboratory assessments.

    Primary Efficacy Endpoint:

    Delta-change of C-peptide Area Under the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at day 372 following WJMSC/Placebo infusion when compared to test performed before start of treatment.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Primary Safety Endpoint:

    During the whole trial. The primary safety endpoint in this study is; safety parameters include adverse events and hypoglycemia, allergic reactions, ophthalmologic examination, ECG, vital signs, laboratory assessments.


    Primary Efficacy Endpoint:

    Delta-change of C-peptide Area Under the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at day 372 following WJMSC/Placebo infusion when compared to test performed before start of treatment.
    E.5.2Secondary end point(s)
    Secondary Endpoint:

    • Number of patients insulin independent (ADA criteria) at days 187 and 372.
    • Number of patients with daily insulin needs <0.25U/kg at days 187 and 372.
    • Insulin requirement/kg BW at days 187 and 372.
    • HbA1c at days 187 and 372.
    • Glucose variability (mean amplitude of glycaemic excursions and glycaemic lability index) and hypo/hyper glycaemia duration derived from the continuous glucose monitoring system® at day 372.
    • Delta change of levels of fasting C-peptide at day 372 when compared to test before start of treatment.
    • Numbers of patients with peak C-peptide >0.20 nmol/l, in response to the MMTT, at day 372.
    • To study changes during one year following treatment in:
    -Insulin doses
    -HbA1c
    -Glucose variability
    -Diabetes Treatment Satisfaction

    Exploratory Endpoints:

    • Delta change of levels of diabetes related autoantibodies (GADab, IA2ab) after 37 and 372 days when compared to test before start of treatment (day 21).
    • Delta change in reactivity and cytokine production of peripheral blood mononuclear cells (PBMC) after 37, 97, 187 and 372 days when compared to test before start of treatment).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary endpoints:
    1-4: Days 187 and 372 following WJMSC/Placebo infusion
    5-6: Day 372 following WJMSC/Placebo infusion

    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 No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Phase I/II
    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
    P1 pts 1-3: 25x10e6 cells,pts 4-6:100 x10e6 cells,pts 7-9:200 x10e6cells,P2 pts 10-24: 200x10e6cells
    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 No
    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
    LVLS
    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 months0
    E.8.9.1In the Member State concerned days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 24
    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 state24
    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)
    Study is completed at one year follow up after infusion of WJMSC or sham. The patients will thereafter continue standard insulin treatment, similar to other patients not in the study. Similarly, patients who are prematurely withdrawn from the study will receive standard treatment for patients with type 1 diabetes with exogenous insulin treatment.
    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 Decision2017-10-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-08-30
    P. End of Trial
    P.End of Trial StatusOngoing
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