BOL-DP-o-05 as an Add-On Treatment of Subjects With Newly Diagnosed Type 1 Diabetes Mellitus

A Phase IIa, Randomized, Double-Blind, Placebo-Controlled, Parallel-Groups, Safety & Efficacy Trial of BOL-DP-o-05 as an Add-On Treatment for Preservation of Beta-Cell Function in Subjects With Newly-Diagnosed T1DM

BOL-DP-o-05 as an Add-On Treatment for Preservation of Beta-Cell Function in Subjects With Newly-Diagnosed Type 1 Diabetes Mellitus (T1DM)

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Groups study in which subjects will be randomized to receive either BOL-DP-o-05 or placebo as an Add-On Treatment. The study evaluates the effect of BOL-DP-o-05 for Preservation of Beta-Cell Function in Subjects with Newly-Diagnosed Type 1 Diabetes Mellitus (T1DM). The study includes a screening period up to three weeks followed by a 48-week treatment period

Inclusion Criteria:

• Informed consent obtained before any trial-related activities.
• T1DM ≤ 20 weeks prior to screening.
• Male or female, aged 5-30 years old (both inclusive) at the time of signing the informed consent form.
• Non-fasting peak C-peptide ≥0.2 nmol/l during mixed-meal tolerance test (MMTT) at Visit 1.
• BMI ≥18.5 kg/m2.
• Presence of one or more islet-specific autoantibodies at the screening.
• Insulin dependence, unless in temporary spontaneous remission ("honeymoon period").

Exclusion Criteria:

• Daily insulin usage > 1 U/kg per day at screening
• History of recurrent (e.g. several times a year) of severe (e.g. pneumonia) or chronic infections or conditions predisposing to chronic infections.
• History of severe systemic fungal infection within the past 12 months prior to screening unless treated and resolved with appropriate documented therapy.
• Vaccination within 4 weeks before randomization.
• Receipt of any other concomitant medications or herbal products that can influence the immune system within 90 days prior to screening.
• History of pancreatitis (acute or chronic).
• Any past or current diagnosis of malignant neoplasms.
• Known impairment of the immune system, except for T1DM, coeliac disease, alopecia, autoimmune antibodies not considered clinical important (e.g. thyroid antibodies without any clinically important thyroid disease), and vitiligo.
• Patients with a psychiatric condition (e.g. severe anxiety, psychosis) that would interfere with the study as determined by the primary investigator.
• Patients with known allergy to one or more of the study drug components.
• Female patients who are pregnant, lactating, or who want to get pregnant during the study period. In the case of young patients, the PI should raise this point with the patient/family.
• Male subjects who want their partner to get pregnant.
• Female of child-bearing potential who do not agree to utilize medically acceptable and reliable means of birth control during the study and for 1 month following the last dose of the study unless the patient is young and the PI speaks with the patient/family and waived the criteria due to young age.
• Patients with a history of alcohol or any psychoactive substance abuse or dependence (including alcohol but excluding nicotine and caffeine).
• Patients with a first-degree family history of a psychiatric condition diagnosed at age<30 years.
• Patients with congestive heart failure or any other chronic disease.
• Patients with heart failure, psychotic state in the past, anxiety disorder, and heredity significant psychiatric inheritance in first-degree family relative, especially in patients younger than 30, and a history of addiction or drug abuse.