The Effects of GLP-1 in Maturity-Onset Diabetes of The Young (MODY)

What Was Tested

In this phase 2, phase 3 trial, researchers set out to test Liraglutide.

The purpose of this study is to evaluate the treatment potential of GLP-1-analogues in patients with Maturity Onset Diabetes of the Young (MODY) compared to common treatment.

It had two arms (groups): liraglutide (experimental) and glimepiride (active comparator).

The registry classes the interventions as drug.

More on the Plan

Clinically, MODY-patients resemble patients with type 2 diabetes (T2DM) more than patients with type 1 diabetes mellitus (T1DM).

MODY is genetically heterogeneous, with known mutations in eight different genes and mutations in either of these genes leads to specific forms of MODY.

The typical course of disease is characterised by a rapid progression from impaired glucose tolerance to diabetes.

After the diagnosis of diabetes, the glucose tolerance is further impaired due to a continuous loss of beta cell function.

Who Took Part

15 actual participants took part.

It was open to people of any sex aged from 18 years with no upper age limit.

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

Examples of who could join: Caucasian above 18 years of age. Well characterised MODY3. Body mass index (BMI) \> 19 kg/m2. Normal haemoglobin (males \> 8.2 mM, females \> 7.2 mM). Normal blood pressure (\< 160/100 mmHg).

The full record lists 8 inclusion and 13 exclusion criteria; among the exclusions: heart failure: new york heart association class iii-iv; uraemia, end-stage renal disease, or any other cause of impaired renal function with s-creatinine \> 130 µm and/or albuminuria; liver disease (alanine amino transferase (alat) and/or aspartate amino transferase (asat) \> 2 × upper normal serum levels).

How the Study Was Run

It was run as a randomized, crossover and quadruple-blind study.

Random assignment meant chance, not the researchers, decided who got Liraglutide and who got the comparison, which limits bias.

Each person switched between Liraglutide and the comparison at some point, usually with a gap in between.

As a double-blind Liraglutide study, the assignments were hidden from both participants and investigators until the end.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2012-08 with completion listed for 2013-08.

It ran at 1 site in Denmark.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Fasting Plasma Glucose (time frame: 14 weeks).

It also tracked 5 secondary outcomes, for example Serum Fructosamine, Hypoglycemic events, Plasma concentrations of insulin and C-peptide, Plasma glucagon and Plasma concentrations of incretin hormones.

What This Means in Plain Language

Put simply, this is a phase 2, phase 3, randomized trial of Liraglutide in 15 people.

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

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Liraglutide compound page, all human trials.