Novel Therapy to Preserve Beta Cell Function in New Onset Type 1 Diabetes

What Was Tested

This registered phase 2 trial tested Insulin (regular human).

It had 1 arm (groups): T1D group (other).

This study was terminated prior to full subject accrual because of changes to study personnel. The original study design was changed from a double-blind, placebo-controlled study to an open-label pilot study in order to collect safety data on enrolled subjects prior to study termination.

The registry classes the interventions as biological and drug.

More on the Plan

The presence of residual beta cell mass may signify a complex interplay between the auto-destructive immune response and the capacity for limited beta cell regeneration.

Selectively targeting the pathogenic T-cells involved in T1D development and progression could achieve the same objective with less toxicity.

Preclinical studies have also identified several growth factors, including epidermal growth factor (EGF), glucagon-like peptide 1 (GLP-1), and gastrin, that appear to promote beta cell proliferation.

The researchers seek to test the potential for preserving beta cell function early in the disease course of T1D by combining antigen-specific immunomodulation with regenerative stimuli.

Who Took Part

7 actual participants took part.

It was open to people of any sex aged from 16 years up to 30 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Ages between 16 and 30 years, inclusive.

The full record lists 5 inclusion and 10 exclusion criteria; among the exclusions: regular use of a proton pump inhibitor within 3 months of enrollment; use of glp-1r agonist or dpp-4 inhibitor within 6 months prior to enrollment; use of immunosuppressive therapy in the preceding 12 months.

How the Study Was Run

The set-up was single-group and open-label.

Nothing was hidden in this open-label Insulin (regular human) study: the participants and staff both knew the treatment.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2009-02 with completion listed for 2011-03.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Change in C-peptide (time frame: 6 months following the protocol subject's randomization/treatment initiation).

It also tracked 5 secondary outcomes, for example Glycemia Control (Change in HbA1c Level), Change in Anti-GAD Autoantibody Titers, Change in Anti-IA2 Titer and Change in ZnT8 Autoantibody Titer.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Insulin (regular human) in 7 people.

Its size is small, so on its own it can only give an early look.

It ended early (terminated); results are posted on the registry, and this page does not restate them.

More context: the Insulin (regular human) compound page, all human trials.