Prepubertal Children With Growth Failure Associated With Primary Insulin-Like Growth Factor-1 (IGF-1) Deficiency

What Was Tested

This registered phase 3 trial tested IGF-1 LR3 and Mecasermin (rhIGF-1).

Subjects will be randomized to either an observation arm or to active treatment.

It had four arms (groups): Untreated (no intervention), one (experimental), one (experimental) and one (experimental).

Observational Group.

The registry classes the interventions as drug.

More on the Plan

Primary IGFD is a term that has been used to describe patients with intrinsic cellular defects in GH action.

Primary IGFD is believed to result from a lower than normal ability to produce IGF-1 when exposed to normal levels of GH, i.e., a type of GH insensitivity or GH resistance.

Who Took Part

137 actual participants took part.

It was open to people of any sex aged from 3 years up to 12 years.

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

Examples of who could join: Chronological age ≥ 3 and chronological or bone age less than or equal to 11 years inclusive in girls;. Chronological age ≥ 3 and chronological or bone age less than or equal to 12 years inclusive for boys. Height SD score of \< -2. IGF-1 SD score of \< -2.

The full record lists 5 inclusion and 3 exclusion criteria; among the exclusions: prior treatment with rhgh, rhigf-1, or other growth-influencing medications; chronic illness such as diabetes, cystic fibrosis, etc.

How the Study Was Run

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

The participants were allocated by chance, so the IGF-1 LR3 group and the comparison group should start out alike.

The IGF-1 LR3 group and the comparison group ran side by side rather than switching.

Being open-label, the IGF-1 LR3 study did not mask who received what.

It started in 2004-03 with completion listed for 2008-07.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Height Velocity During the First Year - Intent to Treat (ITT)Population (time frame: Measured at baseline and at one year).

It also tracked 8 secondary outcomes, for example Change From Baseline in Height Standard Deviation (SD) Score at One Year - ITT Population, Changes in Bone Age From Baseline to One Year, Percent Changes From Baseline in Serum Concentrations of IGF-1 at One Year, Percent Changes From Baseline in Serum Concentrations of IGF-2 at One Year, Percent Changes From Baseline in Serum Concentrations of Insulin-like Growth Factor Binding Protein-2 (IGFBP-2) at One Year and Percent Changes From Baseline in Serum Concentrations of Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) at One Year.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of IGF-1 LR3 and Mecasermin (rhIGF-1) in 137 people.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the IGF-1 LR3 compound page and the Mecasermin (rhIGF-1) compound page, all human trials.