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)

Study Overview

• Status: Withdrawn
• Conditions: Type1 Diabetes Mellitus
• Intervention / Treatment: Drug: Placebo; Drug: BOL-DP-o-05

Detailed Description

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

• Study Type: Interventional
• Phase: Phase 2; Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 30 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: TRIPLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
PLACEBO_COMPARATOR: Placebo	Drug: Placebo; Placebo
EXPERIMENTAL: BOL-DP-o-05	Drug: BOL-DP-o-05; BOL-DP-o-05

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the effect of BOL-DP-o-05 on preservation of beta-cell function	Plasma levels of C-peptide concentration	up to week 48

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of diabetic ketoacidosis episodes	Urine and plasma levels for ketones	Through study completion, an average of 48 weeks
Number of severe hypoglycaemic episodes	Plasma glucose level	Through study completion, an average of 48 weeks
Peak MMTT stimulated C-peptide concentration	Plasma levels of C-peptide concentration	Base line and week 48
To assess the change in fasting C-peptide	Plasma levels of C-peptide concentration	Baseline to week 24 and week 48
To assess the change in HbA1c	HbA1c in plasma	Baseline to weeks 24 and 48
To evaluate total daily insulin dose	Insulin levels in units per kg	Week 24 and week 48
To Assess the percentage of patients that maintain stimulated peak C-peptide ≥ 0.2nmol/L	Plasma levels of C-peptide	At week 48
To Assess the percentage of patients that achieve glycemic target of HbA1c ≤ 7.5%	HbA1c in plasma	At week 24 and week 48
To assess the percent of subjects who require a daily insulin dose < 0.5 IU/kg body weight	Plasma glucose levels	After 12 months of first treatment

Collaborators and Investigators

• Sponsor: Breath of Life International Pharma Ltd

Study record dates

Study Major Dates

• Study Start (ANTICIPATED): November 15, 2020
• Primary Completion (ANTICIPATED): March 22, 2021
• Study Completion (ACTUAL): March 22, 2021

Study Registration Dates

• First Submitted: September 15, 2019
• First Submitted That Met QC Criteria: September 22, 2019
• First Posted (ACTUAL): September 25, 2019

Study Record Updates

• Last Update Posted (ACTUAL): March 24, 2021
• Last Update Submitted That Met QC Criteria: March 22, 2021
• Last Verified: March 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Glucose Metabolism Disorders; Metabolic Diseases; Immune System Diseases; Autoimmune Diseases; Endocrine System Diseases; Diabetes Mellitus; Diabetes Mellitus, Type 1
• Other Study ID Numbers: BOL-P-023

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No