Cystic Fibrosis - Insulin Deficiency, Early Action

What Was Tested

In this phase 3 trial, researchers set out to test Insulin Detemir.

It had two arms (groups): Control group (no intervention) and Once-daily insulin detemir (experimental).

Observation only. Does not receive once-daily insulin detemir.

Once-daily insulin detemir.

The registry classes the interventions as drug.

More on the Plan

As well as progressive lung disease, patients with Cystic Fibrosis (CF) suffer pancreatic destruction, leading to slow but progressive insulin deficiency.

Deficiency of insulin, a powerful anabolic hormone, causes accelerated decline of weight and lung function (important predictors of early mortality in CF).

In the CF-IDEA Trial, subjects with CF aged \>=5 years with early glucose abnormalities (CFID1 or 2) will be randomised to once-daily insulin detemir (Levemir) for 12 months, or to observation only.

Sample size calculations for the CF-IDEA Trial are based on their pilot studies.

Who Took Part

The registry lists 100 planned participants were the target.

It was open to people of any sex aged from 5 years up to 19 years.

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

Examples of who could join: Patients with CF aged \>=5 yrs attending one of the study sites.

The full record lists 2 inclusion and 3 exclusion criteria; among the exclusions: treatment with systemic glucocorticoids of more than 1 month duration, within the last 12 months.

How the Study Was Run

It was run as a randomized, parallel-group and open-label study.

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

The Insulin Detemir group and the comparison group ran side by side rather than switching.

Nothing was hidden in this open-label Insulin Detemir study: the participants and staff both knew the treatment.

It started in 2010-12 with completion listed for 2023-02.

It ran at 6 sites in Australia (5) and United States (1).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Change in Weight SDS (Standard Deviation Score) (time frame: 12 months). Change in lung function (FEV1, FVC) (time frame: 12 months).

It also tracked 8 secondary outcomes, for example Change in glycaemic status assessed by HbA1c and CGM, Body composition by DEXA. Patients at CHW will also have pQCT, Change in Grip-strength, Bacterial colonisation of sputum and Change in effort-dependent lung function: MIP, MEP, SnIP.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Insulin Detemir in 100 people.

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

More context: the Insulin Detemir compound page, all human trials.