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

Sponsor

NextCell Pharma Ab (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT03973827

Collaborator

(none)

Enrollment

Location

Arms

65.5

Anticipated Duration (Months)

0.2

Patients Per Site Per Month

Study Details

Study Description

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

Condition or Disease	Intervention/Treatment	Phase
Type1diabetes	Drug: ProTrans	Phase 1/Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

15 participants

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

May 17, 2019

Actual Primary Completion Date :

Dec 10, 2020

Anticipated Study Completion Date :

Oct 30, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Low dose 3 patients receiving low dose	Drug: ProTrans ProTrans is an pooled allogenic mesenchymal stromal cell treatment
Experimental: Medium dose 3 patients receiving medium dose	Drug: ProTrans ProTrans is an pooled allogenic mesenchymal stromal cell treatment
Experimental: High dose 3 patients receiving high dose	Drug: ProTrans ProTrans is an pooled allogenic mesenchymal stromal cell treatment
No Intervention: Control 6 patients

Outcome Measures

Primary Outcome Measures

The primary safety endpoint in this study is; safety parameters include adverse events, hypoglycemia and allergic reactions [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.
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. [372 days]

Secondary Outcome Measures

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]
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]
Delta change of levels of fasting C-peptide at day 372 when compared to test before start of treatment [372 days]
Numbers of patients with peak C-peptide >0.20 nmol/l, in response to the MMTT, at day 372. [372 days]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 41 Years

Sexes Eligible for Study:

Male

Accepts Healthy Volunteers:

Inclusion Criteria:

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.

Exclusion Criteria:

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 infections unless treatment is not judged necessary by the investigators
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.
Known hypersensitivity against any excipients, i.e. dimethyl sulfoxide (DMSO).