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

NCT03973827 | Completed Phase 1 DMC

• 2 other identifiers: ProTrans-Repeat2018-004158-11
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Type1diabetes

Keywords

type 1 diabetes; diabetes; MSC's; Mesenchymal; Stem Cells; Stromal; Wharton´s Jelly

Outcome Measures

Primary Outcomes (2)

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

  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 when compared to control patients.

  To study changes in insulin requirements during one year following treatment .

  372 days

Secondary Outcomes (9)

• Number of patients insulin independent (ADA criteria) at day 372.

  372 days

• Number of patients with daily insulin needs <0.25U/kg at day 372.

  372 days

• Insulin requirement/kg BW at day 372. HbA1c at day 372.

  372 days

• HbA1c at day 372

  372 days

• Glucose variability (mean amplitude of glycaemic excursions and glycaemic lability index) duration derived from the continuous glucose monitoring system® at day 372

  372 days

Study Arms (4)

Low dose

EXPERIMENTAL

3 patients receiving low dose

Drug: ProTrans

Medium dose

EXPERIMENTAL

3 patients receiving medium dose

Drug: ProTrans

High dose

EXPERIMENTAL

3 patients receiving high dose

Drug: ProTrans

Control

OTHER

6 patients

Other: Control

Interventions

ProTrans DRUG

Single infusion of 25, 100 or 200 million cells per patient.

Also known as: Allogeneic transplantation with WJMSCs

High dose; Low dose; Medium dose

Control OTHER

No intervention. Control subjects.

Control

Eligibility Criteria

• Age: 18 Years - 41 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• A new written informed consent for participation of the study is required to be given before undergoing any study-specific procedures.
• Only patients that have previously been dosed by the IMP according to protocol Protrans-1 are eligible for a second dose of Protrans.
• 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.
• Clinical history compatible with type 1 diabetes diagnosed less than 3 years before enrolment. This also includes control patients not receiving IMP.
• Only male patients between 18-41 years of age will be included.
• Mentally stable and, in the opinion of the investigator, able to comply with the procedures of the study protocol.

You may not qualify if:

• Inability to provide informed consent
• Patients with body mass index (BMI) \> 30, or weight \>100 kg
• Patients with weight \<50 kg
• Patients with unstable cardiovascular status incl. NYHA class III/IV or symptoms of angina pectoris.
• Patients with uncontrolled hypertension (≥160/105 mmHg).
• Patients with active on-going infections.
• 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.
• 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.
• Patients with any immune suppressive treatment
• Patients with known demyelinating disease or with symptoms or physical examination findings consistent with possible demyelinating disease.
• Patients with known, or previous, malignancy.
• Taking oral anti-diabetic therapies or any other concomitant medication which may interfere with glucose regulation other than insulin
• Patients with GFR \<80 ml/min/1.73 m2 body surface
• Patients with proliferative retinopathy
• Patient with any condition or any circumstance that in the opinion of the investigator would make it unsafe to undergo treatment with MSC.

Sponsors & Collaborators

• NextCell Pharma Ab: lead

Study Sites (1)

Karolinska Trial Alliance, Fas 1 enheten, Karolinska Universitetssjukhuset Huddinge

Huddinge, Sweden

Location

MeSH Terms

Conditions

Diabetes Mellitus, Type 1; Diabetes Mellitus

Interventions

Transplantation, Homologous

Condition Hierarchy (Ancestors)

Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Transplantation; Surgical Procedures, Operative

Study Officials

• Per-Ola Carlsson, PhD

  Uppsala University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 16, 2019
• First Posted: June 4, 2019
• Study Start: May 17, 2019
• Primary Completion: December 10, 2020
• Study Completion: November 20, 2024
• Last Updated: March 7, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

SE (1)