Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Short Stature Associated With IGF-1 Deficiency

What Was Tested

This registered phase 2, phase 3 trial tested IGF-1 LR3 and Mecasermin (rhIGF-1).

It had 1 arm (groups): rhIGF-1 QD (experimental).

Subjects received subcutaneous injection (SC) injections of rhIGF-1 once a day.

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 growth hormone (GH) action.

In this protocol, primary IGFD is defined as short stature (\<-2 standard deviations \[SDs\] below the mean for age and gender), and abnormal serum IGF-1 (\<-2 SDs below the mean for age and gender).

The trial is an open-label, concentration-controlled trial conducted at up to 20 centers throughout the United States.

Who Took Part

45 actual participants took part.

It was open to people of any sex aged from 3 years with no upper age limit.

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

Examples of who could join: Chronological age ≥ 3. Chronological age or bone age ≤ 12 for boys and ≤ 11 for girls. Prepubertal at Visit 1. 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 gh, igf-1, or other growth-influencing medications; chronic illness such as diabetes, cystic fibrosis, etc.

How the Study Was Run

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

Nothing was hidden in this open-label IGF-1 LR3 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 2005-07 with completion listed for 2009-01.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Height Velocity From Pretreatment (Week 0) to Week 34 (time frame: Pretreatment to Week 34). Height Velocity From Week 34 to 86 (time frame: Week 34 to 86).

It also tracked 5 secondary outcomes, for example Change in Height SD Score From Pretreatment to Week 34, Change in Height SD Score From Pretreatment to Week 86, Change in Bone Age From Pretreatment to Week 86 Minus Change in Chronological Age, Percent Change in Serum Concentration of IGFBP-1, IGFBP-2 and IGFBP-3 From Pretreatment to Week 86 and Percent Change in Serum Concentration of ALS From Pretreatment to Week 86.

What This Means in Plain Language

Put simply, this is a phase 2, phase 3 trial of IGF-1 LR3 and Mecasermin (rhIGF-1) in 45 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.