This is a combined phase 1 and 2 study in 66 subjects, male or female, between 7-21 years of age that have recently (\< 6 months) been diagnosed with type 1 diabetes. The first phase 1 part of the study includes six subjects openly receiving allogeneic Wharton's jelly derived mesenchymal stromal cells as the Advanced Therapy Medicinal Product (ATMP) Protrans, three each in the age ranges 7-11 and 12-18.The second part is a randomized, double-blinded placebo-controlled phase 2 study in parallel design comparing allogeneic Wharton's jelly derived mesenchymal stromal cells treatment (as Protrans) to placebo in children and adolescent subjects (7-21 years of age) diagnosed with type 1 diabetes, The primary objectives of this study will be to investigate the safety, tolerance and efficacy after an allogieneic infusion of Wharton's jelly derived mesenchymal stromal cells.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
66
Protrans consists of Wharton's jelly derived mesenchymal stromal cells
Uppsala University Hospital
Uppsala, Sweden
RECRUITINGSafety at one year evaluated as adverse events
Safety parameters will be evaluated at each study visit and recorded as adverse events.
Time frame: One year
Safety at five years evaluated as adverse events
Safety parameters will be evaluated at each study visit and recorded as adverse events.
Time frame: Five years
Efficacy measured as change in C-peptide Area under the curve to a mixed mealtolerance test.
Change in C-peptide Area under the curve (AUC) (0-120 min) for mixed meal tolerance test (MMTT) at 12 months following Protrans/Placebo infusion when compared to test performed before the start of treatment (baseline).
Time frame: One year
Insulin independency
The proportion of study participants independent of insulin at 6 months
Time frame: One year
Insulin independency
The proportion of study participants independent of insulin at 12 months
Time frame: One year
Low insulin needs
The proportion of study participants with daily insulin needs \<0.25 U/kg at 6 months
Time frame: 6 months
Low insulin needs
The proportion of study participants with daily insulin needs \<0.25 U/kg at 12 months
Time frame: 12 months
Insulin needs
Insulin requirement/kg body weigh at 6 months
Time frame: 6 months
Insulin needs
Insulin requirement/kg body weigh at 12 months
Time frame: 12 months
HbA1c
HbA1c at 6 months
Time frame: 6 months
HbA1c
HbA1c at 12 months
Time frame: 12 months
Time in target
Time in target (4-8 mmol/l) as measured by flash glucose monitoring for 14 days at 6 months
Time frame: 6 months
Time in target
Time in target (4-8 mmol/l) as measured by flash glucose monitoring for 14 days at 12 months
Time frame: 12 months
Time in range
Time in target (3.9-10 mmol/l) as measured by flash glucose monitoring for 14 days at 6 months
Time frame: 6 months
Time in range
Time in target (3.9-10 mmol/l) as measured by flash glucose monitoring for 14 days at 12 months
Time frame: 12 months
C-peptide
Change in C-peptide Area under the curve (AUC) (0-120 min) for mixed meal tolerance test (MMTT) at 6 months following Protrans/Placebo infusion when compared to test performed before the start of treatment (baseline).
Time frame: 6 months
Change in peak C-peptide
Change in peak C-peptide concentration during the first 6 months
Time frame: 6 months
Change in peak C-peptide
Change in peak C-peptide concentration during the first 12 months
Time frame: 12 months
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