rhGH and rhIGF-1 Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency

What Was Tested

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

IGF-1 (insulin-like growth factor-1) is a hormone that is normally produced in the body in response to another hormone called growth hormone.

Growth Hormone is produced by a small gland at the base of the brain (the pituitary).

Together IGF-1 and GH are large contributors to growth during infancy, childhood, and adolescence.

It had four arms (groups): 1. rhGH Alone (active comparator), one (experimental), one (experimental) and one (experimental).

The registry classes the interventions as drug.

Who Took Part

The registry lists 106 actual participants took part.

It was open to people of any sex aged from 5 years with no upper age limit.

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

Examples of who could join: Parents or legally authorized representatives must give signed informed consent before any trial-related activities. IGF-1 SDS of ≤ -1 for age and gender. Short stature, as defined by a height SDS of ≤ -2 for age and gender. Chronological age ≥ 5 years. Bone age ≤ 11 years in boys and ≤ 9 years in girls.

The full record lists 8 inclusion and 5 exclusion criteria; among the exclusions: severe primary igfd (defined as height and igf-1 sds ≤ 3, and stimulated gh response greater than or equal to 10 ng/ml); known or suspected allergy to rhgh, rhigf-1 or a constituent of their formulations; current use of medications for attention deficit disorder.

How the Study Was Run

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

Because people in this IGF-1 LR3 study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

Groups ran in parallel: each of the participants stayed in one arm throughout.

Nothing was hidden in this open-label IGF-1 LR3 study: the participants and staff both knew the treatment.

It started in 2007-12 with completion listed for 2010-04.

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 (time frame: First year of treatment).

It also tracked 12 secondary outcomes, for example Height Velocity, Cumulative Change in Height Standard Deviation Score (SDS), Predicted Adult Height (PAH), Total Change From Baseline (Day 1) in BMI SDS, Skeletal Maturation and Changes From Baseline (Day 1) in Serum Concentrations of Growth Hormone (GH).

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of IGF-1 LR3 and Mecasermin (rhIGF-1) in 106 people.

It ended early (terminated); 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.