A 2-arm randomized Phase II Open Label Study to evaluate the safety and feasibility of intralymphatic administration of Diamyd® (Diamyd) also known as retogatein in individuals at risk of Type 1 diabetes carrying the HLA DR3-DQ2 haplotype.
The trial is a 2-arm, randomized, open label clinical trial in individuals aged 8 - \<18 years with HLA DR3-DQ2 and multiple islet autoantibodies (Stage 1 or Stage 2) at increased risk for T1D. At baseline eligible individuals at risk of T1D will be randomized 1:1 into 2 or 3 injections of Diamyd® also known as retogatein administered into an inguinal lymph node. Subjects will be followed for a total of 12 months post-enrollment.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
PREVENTION
Masking
NONE
Enrollment
16
4 μg (0.1 mL) of Diamyd administered 1 month apart.
Lund University/CRC, Skåne University Hospital,
Malmo, Sweden
RECRUITINGOccurrence of AEs (including Injection site reactions) and SAEs
Time frame: From screening to 12 months.
Number of Clinically Significant Abnormal Results from Physical examinations, including neurological and Vital Signs assessments
Time frame: From screening to 12 months.
Number of Clinically Significant Abnormal Results From Laboratory measurements, including hematology, clinical chemistry, metabolic status parameters (fasting C-peptide, HbA1c, fasting glucose) and urine analysis
Time frame: From screening to 12 months.
Stage progression
Number of individuals progressing from stage 1 to stage 2 and from stage 2 to stage 3.
Time frame: From screening to 12 months.
T1D diagnosis
Time to T1D diagnosis.
Time frame: From screening to 12 months.
Time to stage progression
Time from stage 1 to stage 2 and time from stage 2 to stage 3.
Time frame: From screening to 12 months.
OGTT stimulated C-peptide
Change in C-peptide /insulin /glucose (Area Under the Curve \[AUC\]mean 0-120 min) during a 2-hour Oral Glucose Tolerance Test (OGTT) from screening to 12 months.
Time frame: From screening to 12 months.
Change in Hemoglobin A1c (HbA1c)
Change in Hemoglobin A1c (HbA1c) from baseline to 12 months.
Time frame: From baseline to 12 months.
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Change in time in glycemic target range
Change in time in glycemic target range 3.9 to 10 mmol/L (70 to 180 mg/dL) and 3.9 to 7.8 mmol/L (70 to 140 mg/dL) evaluated from continuous glucose monitoring (CGM) data from screening to 12 months.
Time frame: From screening to 12 months.
Change in time in range > 13.9 mmol/L
Change in time in range \> 13.9 mmol/L (\> 250 mg/dL) evaluated from CGM data from screening to 12 months.
Time frame: From screening to 12 months.
Change in time in range > 10 mmol/L
Change in time in range \> 10 mmol/L (\> 180 mg/dL) evaluated from CGM data from screening to 12 months.
Time frame: From screening to 12 months.
Change in time in range > 7.8 mmol/L
Change in time in range \> 7.8 mmol/L (\> 140 mg/dL) evaluated from CGM data from screening to 12 months.
Time frame: From screening to 12 months.
Change in glycemic variation
Change in glycemic variation (Coefficient of variation \[CV\], SD) evaluated from CGM data from screening to 12 months.
Time frame: From screening to 12 months.
Variables that indicate effects on the immune system
Variables that indicate effects on the immune system such as the concentration of serum autoantibodies.
Time frame: From baseline to 12 months.