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    Summary
    EudraCT Number:2020-004520-42
    Sponsor's Protocol Code Number:WJMSC-01
    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:2020-12-22
    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 number2020-004520-42
    A.3Full title of the trial
    “A Double-blinded, Randomized, Parallel, Placebo-controlled trial of Wharton´s Jelly-derived Allogeneic Mesenchymal Stromal Cells to treat Type I Diabetes in Children and Adolescents”
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Mesenchymal Stromal Cells to prevent further loss of own insulin production in case of illness in Type I Diabetes in children and young adults
    Mesenkymala stromaceller för att förhindra fortsatt förlust av egen insulinproduktion vid insjuknande i typ 1 diabetes hos barn och unga vuxna
    A.4.1Sponsor's protocol code numberWJMSC-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 SponsorUppsala University Hospital
    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 supportResearch funding
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUppsala University Hospital
    B.5.2Functional name of contact pointDpt of Endocrin and Diabet, entr 40
    B.5.3 Address:
    B.5.3.1Street AddressSjukhusvägen
    B.5.3.2Town/ cityUppsala
    B.5.3.3Post code751 85
    B.5.3.4CountrySweden
    B.5.4Telephone number+46(0)18471 44 25
    B.5.5Fax number+46(0)18471 40 59
    B.5.6E-mailper-ola.carlsson@mcb.uu.se
    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.3.2Product code WJMSC
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNWharton´s jelly-derived mesenchymal stromal cells
    D.3.9.2Current sponsor codeWJMSC
    D.3.9.3Other descriptive nameAllogeneic Wharton's jelly-derived mesenchymal stem cells
    D.3.9.4EV Substance CodeSUB197611
    D.3.10 Strength
    D.3.10.1Concentration unit Munit million units
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number200
    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 placeboInfusion
    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 I diabetes
    Typ 1 diabetes
    E.1.1.1Medical condition in easily understood language
    Diabetes type I, i.e. inability to regulate the blood sugar concentration due to low or no production of insulin.
    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 21.1
    E.1.2Level PT
    E.1.2Classification code 10067584
    E.1.2Term Type 1 diabetes mellitus
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    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, tolerance and efficacy after allogeneic infusion of WJMSCs intravenously in children and adolescents recently (<6 months) 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 the first year following treatment.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Written informed consent for participation of the study (for subjects below 18 years of age also
    from both caregivers), given before undergoing any study-specific procedures
    2.Clinical history compatible with type 1 diabetes diagnosed less than 6 months before enrolment
    3.In the first part of the study, six subjects, three between 7-11 and three between 12-18 years of
    age (both groups inclusive at both ends), will be included. The sixty subjects in the second part of
    the study are stratified by age (12-21 and 7-11 years, respectively) and randomized to one of two
    treatment arms (active or placebo), with a 6-month safety delay for the younger stratum.
    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.Subjects of child-bearing potential must agree to using adequate contraception until one year
    after the administration of WJMSC/Placebo. Adequate contraception is as follows:
    a)oral (except low-dose gestagen (lynestrenol and noretisteron), injectable or implanted
    hormonal contraceptives.
    b)intrauterine device
    c)intrauterine system (for example progestin-releasing coil)
    d)vasectomized male (with appropriate postvasectomy documentation of the absence of sperm in the ejaculate)
    E.4Principal exclusion criteria
    1.Subjects with bodyweight >100 kg
    2.Subjects with unstable cardiovascular status incl. NYHA class III/IV or symptoms of angina pectoris.
    3.Subjects with uncontrolled hypertension (≥160/105 mmHg).
    4.Subjects with active on-going infections.
    5.Subjects with latent or previous as well as on-going therapy against tuberculosis, or exposed to tuberculosis or has traveled in areas with a high risk of tuberculosis or mycosis within the last 3 months.
    6.Subjects with serological evidence of infection with HIV, Treponema pallidum, hepatitis B antigen(subjects with serology consistent with previous vaccination and a history of vaccination are acceptable), or hepatitis C.
    7.Subjects with any systemic immune suppressive treatment
    8.Subjects with a known demyelinating disease or with symptoms or physical examination findings consistent with possible demyelinating disease.
    9.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.
    10.Subjects with known, or previous, malignancy.
    11.Taking oral anti-diabetic therapies or any other concomitant medication which may interfere with glucose regulation other than insulin.
    12.Subjects with GFR <60 ml/min/1.73 m2 body surface.
    13.Subject with any condition or any circumstance that, in the opinion of the investigator, would make it unsafe to undergo treatment with MSC.
    14.Known hypersensitivity against any excipients, i.e., dimethyl sulfoxide (DMSO).
    E.5 End points
    E.5.1Primary end point(s)
    Primary Safety Endpoint:
    Safety parameters will be evaluated in the intervention trial at each study visit and recorded as adverse events (AEs). Any grade 3 event (or higher) will be evaluated by DSMB.

    Primary Efficacy Endpoint:
    Change in C-peptide Area Under the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at 12 months following WJMSC/Placebo infusion when compared to test performed before the start of treatment (baseline).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety: Continuously from infusion of study medication until study end. Followed-up at each study visit.

    Efficacy: At baseline and 12 month visits.
    E.5.2Secondary end point(s)
    •The proportion of study participants independent of insulin (ADA criteria) at 6 and 12 months.
    •The proportion of participants with daily insulin needs <0.25U/kg at 6 and 12 months.
    •Insulin requirement/kg body weight at 6 and 12 months.
    •Glycosylated Hb (HbA1c) and insulin-dose adjusted HbA1c (IDAA1c) at 6 and 12 months.
    •Time-in-target (4-8 mmol/l) and Time-in-range (3.9-10 mmol/l) as measured by flash glucose monitoring for 14 days at 6 and 12 months.
    •Change in C-peptide Area Under the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at 6 months following WJMSC/Placebo infusion when compared to test performed before the start of treatment (baseline).
    •Change in peak C-peptide concentration during the first 6 months or the first year after treatment.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At 6 and 12 month visits.
    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 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
    New age groups (7-21 years)
    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 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 trial2
    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 concerned3
    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 years6
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    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: 60
    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: 33
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 27
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 6
    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.3.3.6.1Details of subjects incapable of giving consent
    All children will be informed, but for all children below 18 years informed consent will be required from caregivers.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state66
    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)
    It will not differ from normal 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 Decision2021-03-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-04-07
    P. End of Trial
    P.End of Trial StatusOngoing
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