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

March 30, 2023 updated by: NextCell Pharma Ab

An Open Label, Parallel Single Center Trial of Wharton's Jelly Derived Allogeneic Mesenchymal Stromal Cells Repeatedly Treated to Preserve Endogenous Insulin Production in Adult Patients Diagnosed With Type 1 Diabetes

An open label, parallel single centre trial of Wharton's Jelly derived allogenic mesenchymal stromal cells repeated treatment to preserve endogenous insulin production in adult patients diagnosed with type 1 diabetes

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

This is a phase (I)/II study, and the purpose of this study is to determine whether, in adult patients diagnosed for type 1 diabetes, a repeated allogeneic infusion of WJMSCs is safe and to study changes in beta-cell function, metabolic control and Diabetes Treatment Satisfaction. The study population will consist of 18 adult male patients, 18-41 years of age (inclusive at both ends) diagnosed (<3,5 years) with type 1 diabetes mellitus.

Study Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Sweden
      • Huddinge, Sweden
        • Karolinska Trial Alliance, Fas 1 enheten, Karolinska Universitetssjukhuset Huddinge

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 41 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  1. A new written informed consent for participation of the study is required to be given before undergoing any study-specific procedures.
  2. Only patients that have previously been dosed by the IMP according to protocol Protrans-1 are eligible for a second dose of Protrans.
  3. No identified IMP related on-going adverse event, neither history of any adverse event that is evaluated potentially to be related to the previous IMP dosing in Protrans I.
  4. Clinical history compatible with type 1 diabetes diagnosed less than 3 years before enrolment. This also includes control patients not receiving IMP.
  5. Only male patients between 18-41 years of age will be included.
  6. Mentally stable and, in the opinion of the investigator, able to comply with the procedures of the study protocol.

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. 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. Patients with GFR <80 ml/min/1.73 m2 body surface
  14. Patients with proliferative retinopathy
  15. Patient with any condition or any circumstance that in the opinion of the investigator would make it unsafe to undergo treatment with MSC.
  16. Known hypersensitivity against any excipients, i.e. dimethyl sulfoxide (DMSO).- This criterion is only applicable to patients which receive study drug.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Low dose
3 patients receiving low dose
Drug: ProTrans
Single infusion of 25, 100 or 200 million cells per patient.
Other Names:
  • Allogeneic transplantation with WJMSCs
Experimental: Medium dose
3 patients receiving medium dose
Drug: ProTrans
Single infusion of 25, 100 or 200 million cells per patient.
Other Names:
  • Allogeneic transplantation with WJMSCs
Experimental: High dose
3 patients receiving high dose
Drug: ProTrans
Single infusion of 25, 100 or 200 million cells per patient.
Other Names:
  • Allogeneic transplantation with WJMSCs
Sham Comparator: Control
6 patients
Drug: Placebo
Placebo Comparator: Sham transplantation (placebo) Single infusion with albumin and dmso in sodium chloride (identical concentrations as active treatment)
Other Names:
  • Placebo treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The primary safety endpoint in this study is; safety parameters include adverse events, hypoglycemia and allergic reactions
Time Frame: 372 days
To investigate the safety and tolerance after a repeated allogeneic infusion of Whartons Jelly Mesenchymal Stromal Cells (WJMSCs) intravenously in adult patients diagnosed with type 1 diabetes after one year following the repeated treatment.
372 days
Delta-change of C-peptide AreaUnder the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at day 372 following WJMSC infusion when compared to test performed before start of treatment.
Time Frame: 372 days
To study changes in insulin requirements during one year following treatment .
372 days

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

NextCell Pharma Ab

Investigators

  • Principal Investigator: Per-Ola Carlsson, PhD, Uppsala University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 17, 2019

Primary Completion (Actual)

December 10, 2020

Study Completion (Anticipated)

October 30, 2024

Study Registration Dates

First Submitted

May 16, 2019

First Submitted That Met QC Criteria

June 3, 2019

First Posted (Actual)

June 4, 2019

Study Record Updates

Last Update Posted (Actual)

April 3, 2023

Last Update Submitted That Met QC Criteria

March 30, 2023

Last Verified

March 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ProTrans-Repeat
  • 2018-004158-11 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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