Liraglutide (Victoza) lowered A1C in children and teens with type 2 diabetes, trial finds

Liraglutide, sold under the brand name Victoza, lowered average blood sugar more than placebo over 52 weeks in children and teenagers with type 2 diabetes, according to trial results published April 28, 2019, in the New England Journal of Medicine ^[1].

The study, called Ellipse, enrolled people 10 to under 17 years old. Of 135 who were randomly assigned, 134 received at least one dose: 66 got liraglutide and 68 got placebo. The mean age was 14.6 years ^[1].

Participants were assigned in a 1:1 ratio to subcutaneous liraglutide up to 1.8 mg per day or to placebo for a 26-week period in which neither group knew which treatment they were getting, followed by a 26-week period in which the assignment was open ^[1]. Everyone in the trial also took metformin, and some patients also took basal insulin. To join, patients had to have a body-mass index above the 85th percentile and an A1C between 7.0% and 11.0% if they were treated with diet and exercise alone, or between 6.5% and 11.0% if they were taking metformin, with or without insulin ^[1].

What the trial found

At 26 weeks, A1C — a measure of average blood sugar over roughly three months — fell by 0.64 percentage points in the liraglutide group and rose by 0.42 percentage points in the placebo group ^[1]. That is an estimated treatment difference of -1.06 percentage points, which the authors reported as statistically significant (P<0.001). By 52 weeks the difference had widened to -1.30 percentage points ^[1]. Fasting plasma glucose fell at both time points in the liraglutide group and rose in the placebo group ^[1].

The number of patients who reported side effects was similar between the two groups: 56 (84.8%) with liraglutide and 55 (80.9%) with placebo. However, the overall rate of adverse events and the rate of gastrointestinal adverse events were higher with liraglutide ^[1]. Which gastrointestinal symptoms occurred, and how severe they were, is not described in the published summary of the trial.

The trial was funded by Novo Nordisk and registered as NCT01541215 ^[1]. A follow-up comment on the trial appeared in the same journal on Oct 31, 2019 ^[1].

Why it matters for patients

Metformin is the regulatory-approved treatment of choice for most young people with type 2 diabetes early in the disease, but the researchers note that early loss of blood sugar control has been observed when metformin is used alone ^[1]. That leaves few well-tested options for children and teenagers whose A1C rises while they are taking metformin.

Some details shape how the results apply. Everyone in the trial took metformin, so the numbers describe liraglutide as an add-on, not on its own. The trial ran for 52 weeks, so effects on growth, on diabetes complications, and on blood sugar beyond that period are not reported in this paper. The trial tested doses up to 1.8 mg per day, so the findings cover that range only ^[1].

The report also does not say what regulators decided about using liraglutide in children, or whether any label change followed. Those decisions have not been disclosed in the trial publication.

What happens next

The 2019 publication does not describe further pediatric trials of liraglutide or a timeline for regulatory review, so any next steps have not been reported.