Type 1 diabetes MedDRA version: 20.0 Level: LLT Classification code 10012594 Term: Diabetes System Organ Class: 100000004861
Conditions
Interventions
Product Name: ProTrans
Pharmaceutical Form: Suspension for injection
Pharmaceutical form of the placebo: Suspension for injection
Route of administration of the placebo: Intravenous use
Sponsors
NextCell Pharma
Eligibility
Sex/Gender
All
Inclusion criteria
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/): a. Combined (estrogen and progestogen containing hormonal contraception associated with inhibition of ovulation. -oral -intravaginal -transdermal b. progestogen-only hormonal contracption associated with inhibition of ovulation - oral - injectable - implantable c. intrauterine device (IUD) d. intrauterine hormone-releasing system (IUS) e. bilateral tubal occlusion f. total abstinence or vasectomized partner. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 24 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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. 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. Patients with GFR <80 ml/min/1.73 m2 body surface. 15. Patients with proliferative retinopathy 16. Patient with any condition or any circumstance that in the opinion of the investigator would make it unsafe to undergo treatment with MSC. 17. Known hypersensitivity against any excipients, i.e. dimethyl sulfoxide (DMSO).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To investigate the safety and tolerance after allogeneic infusion of WJMSCs intravenously in adult patients diagnosed with type 1 diabetes.;Secondary Objective: Study changes in beta-cell function, metabolic control and Diabetes Treatment Satisfaction during one year.;Primary end point(s): Primary Safety Endpoint: The primary safety endpoint in this study is; safety parameters include adverse events and hypoglycemia, allergic reactions, ophthalmologic examination, ECG, vital signs, laboratory assessments. Primary Efficacy Endpoint: 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.;Timepoint(s) of evaluation of this end point: Primary Safety Endpoint: During the whole trial. The primary safety endpoint in this study is; safety parameters include adverse events and hypoglycemia, allergic reactions, ophthalmologic examination, ECG, vital signs, laboratory assessments. Primary Efficacy Endpoint: 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. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary Endpoint: • Number of patients insulin independent (ADA criteria) at days 187 and 372. • Number of patients with daily insulin needs 0.20 nmol/l, in response to the MMTT, at day 372. • To study changes during one year following treatment in: -Insulin doses -HbA1c -Glucose variability -Diabetes Treatment Satisfaction Exploratory Endpoints: • Delta change of levels of diabetes related autoantibodies (GADab, IA2ab) after 37 and 372 days when compared to test before start of treatment (day 21). • Delta change in reactivity and cytokine production of peripheral blood mononuclear cells (PBMC) after 37, 97, 187 and 372 days when compared to test before start of treatment). ;Timepoint(s) of evaluation of this end point: Secondary endpoints: 1-4: Days 187 and 372 following WJMSC/Placebo infusion 5-6: Day 372 following WJMSC/Placebo infusion | — |
Countries
Sweden
Contacts
Public ContactMathias Svahn
NextCell Pharma
Outcome results
None listed