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Wharton´s Jelly Derived Mesenchymal Stromal Cell Treatment of Adult Patients Diagnosed With Type I Diabetes

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ClinicalTrials.gov Identifier: NCT03406585
Recruitment Status : Recruiting
First Posted : January 23, 2018
Last Update Posted : June 5, 2019
Sponsor:
Information provided by (Responsible Party):
NextCell Pharma Ab

Tracking Information
First Submitted Date  ICMJE November 27, 2017
First Posted Date  ICMJE January 23, 2018
Last Update Posted Date June 5, 2019
Actual Study Start Date  ICMJE November 28, 2017
Estimated Primary Completion Date June 1, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 19, 2018)
  • Safety; measured through set safety parameters [ Time Frame: throughout the study untill day 372 ]
    measured through the registration of adverse events and other safety parameters such as hypoglycemia, allergic reactions, ophthalmologic examination, ECG, vital signs and laboratory assessments.
  • Efficacy; comparison of the intervention versus placebo at day 372 versus start of treatment [ Time Frame: Day 372 ]
    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.
Original Primary Outcome Measures  ICMJE
 (submitted: January 15, 2018)
  • Safety; measured through set safety parameters [ Time Frame: throughout the study untill day 372 ]
    measured through the registration of adverse events and other measurements
  • Efficacy; comparison of the intervention versus placebo at day 372 versus start of treatment [ Time Frame: Day 372 ]
    Measured throughout the study at various intervals
Change History Complete list of historical versions of study NCT03406585 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: January 19, 2018)
  • Number of patients insulin independent (ADA criteria) at days 187 and 372 [ Time Frame: Days 187 and 372 following WJMSC/Placebo infusion ]
    Insulin doses and measured plasma glucose values are written down in the subject's diary every day by the subject. Amount of insulin, recorded plasma glucose values, time and date will be included
  • Number of patients with daily insulin needs <0.25U/kg at days 187 and 372 [ Time Frame: Days 187 and 372 following WJMSC/Placebo infusion ]
    Insulin doses and measured plasma glucose values are written down in the subject's diary every day by the subject. Amount of insulin, recorded plasma glucose values, time and date will be included
  • Insulin requirement/kg BW at days 187 and 372 [ Time Frame: Days 187 and 372 following WJMSC/Placebo infusion ]
    Insulin doses and measured plasma glucose values are written down in the subject's diary every day by the subject. Amount of insulin, recorded plasma glucose values, time and date will be included
  • HbA1c at days 187 and 372. [ Time Frame: Days 187 and 372 following WJMSC/Placebo infusion ]
    Measurements of HbA1c will be performed to assess metabolic control during study.
  • Glucose variability at day 372 [ Time Frame: Day 372 following WJMSC/Placebo infusion ]
    Measurements of HbA1c will be performed to assess metabolic control during study.
  • Delta change of levels of fasting C-peptide at day 372 [ Time Frame: Day 372 following WJMSC/Placebo infusion ]
    Measured at day 372 compared to before start of treatment
  • Numbers of patients with peak C-peptide >0.20 nmol/l, in response to the MMTT, at day 372 [ Time Frame: Day 372 following WJMSC/Placebo infusion ]
    Measured at day 372 compared to before start of treatment
Original Secondary Outcome Measures  ICMJE
 (submitted: January 15, 2018)
  • Number of patients insulin independent (ADA criteria) at days 187 and 372 [ Time Frame: Days 187 and 372 following WJMSC/Placebo infusion ]
  • Number of patients with daily insulin needs <0.25U/kg at days 187 and 372 [ Time Frame: Days 187 and 372 following WJMSC/Placebo infusion ]
  • Insulin requirement/kg BW at days 187 and 372 [ Time Frame: Days 187 and 372 following WJMSC/Placebo infusion ]
  • HbA1c at days 187 and 372. [ Time Frame: Days 187 and 372 following WJMSC/Placebo infusion ]
  • Glucose variability at day 372 [ Time Frame: Day 372 following WJMSC/Placebo infusion ]
  • Delta change of levels of fasting C-peptide at day 372 when compared to test before start of treatment [ Time Frame: Day 372 following WJMSC/Placebo infusion ]
  • Numbers of patients with peak C-peptide >0.20 nmol/l, in response to the MMTT, at day 372 [ Time Frame: Day 372 following WJMSC/Placebo infusion ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Wharton´s Jelly Derived Mesenchymal Stromal Cell Treatment of Adult Patients Diagnosed With Type I Diabetes
Official Title  ICMJE A Double-blinded, Randomized, Placebo-controlled Trial With Wharton's Jelly Derived Allogeneic Mesenchymal Stromal Cells (WJMSCs) for Preserving Endogenous Insulin Production in Adult Patients Diagnosed for Type 1 Diabetes
Brief Summary To investigate the safety and tolerance after allogeneic infusion of WJMSCs intravenously in adult patients diagnosed with type 1 diabetes.
Detailed Description

This is a combined phase I and phase II study, where the first part is an open, dose escalating study consisting of 6 male patients, 18-40 years of age. The second part is a randomized, double-blinded, placebo-controlled, phase I/II study in parallel design comparing allogeneic WJMSC treatment to placebo in adult patients diagnosed with type 1 diabetes. Besides safety, preservation of endogenous insulin production (measured as C-peptide concentrations) together with metabolic control, diabetes treatment satisfaction and immunological profile will be assessed.

A total number of 24 patients will be enrolled in the study and followed for one year after WJMSC/placebo treatment. Patients 18-40 years of age, both male and female, diagnosed for type 1 diabetes will be eligible. Providing informed consent and fulfillment of inclusion criteria and no exclusion criteria, they will within two years of diagnosis be randomized.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Type1 Diabetes Mellitus
Intervention  ICMJE
  • Drug: ProTrans: Allogeneic transplantation with WJMSCs
    The IP is a cell suspension with MSC's procured from donated Wharton's Jelly from umbilical cord tissue and expanded in adherent culture over maximum of 3 passages. The treatment is allogenic.
  • Drug: Placebos
    Placebo treatment
Study Arms  ICMJE
  • Experimental: Allogeneic transplantation with WJMSCs (batch 1)
    Intervention: Drug: ProTrans: Allogeneic transplantation with WJMSCs
  • Experimental: Allogeneic transplantation with WJMSCs (batch 2)
    Intervention: Drug: ProTrans: Allogeneic transplantation with WJMSCs
  • Placebo Comparator: Sham transplantation (placebo)
    Intervention: Drug: Placebos
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 15, 2018)
24
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 1, 2020
Estimated Primary Completion Date June 1, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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/):

    1. Combined (estrogen and progestogen containing hormonal contraception associated with inhibition of ovulation.

      • oral
      • intravaginal
      • transdermal
    2. progestogen-only hormonal contracption associated with inhibition of ovulation

      • oral
      • injectable
      • implantable
    3. intrauterine device (IUD)
    4. intrauterine hormone-releasing system (IUS)
    5. bilateral tubal occlusion
    6. total abstinence or vasectomized partner.

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
  5. Patients with active infections unless treatment is not judged necessary by the investigators
  6. Patients exposed to tuberculosis or has travelled in areas with high risk of tuberculosis or mycosis within the last 3 months
  7. 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.
  8. Patients with any immune suppressive treatment
  9. Patients with known demyelinating disease
  10. 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. Known hypersensitivity against any excipients, i.e. dimethyl sulfoxide (DMSO).
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 40 Years   (Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE
Contact: Mathias Svahn +4687355595 mathias.svahn@nextcellpharma.com
Contact: Leo Groenewegen +4687355595 leo.groenewegen@nextcellpharma.com
Listed Location Countries  ICMJE Sweden
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03406585
Other Study ID Numbers  ICMJE ProTrans-T1D
2017-002766-50 ( EudraCT Number )
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party NextCell Pharma Ab
Study Sponsor  ICMJE NextCell Pharma Ab
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Per-Ola Carlsson, MD, PhD NextCell Pharma
PRS Account NextCell Pharma Ab
Verification Date June 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP