A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder

What Was Tested

In this phase 2 trial, researchers set out to test IGF-1 LR3.

The researchers expect to provide evidence for the safety and feasibility of IGF-1 in ameliorating social withdrawal in children with Autistic Disorder.

It had two arms (groups): IGF-1 (experimental) and Placebo (placebo comparator).

Placebo.

The registry classes the interventions as drug.

Who Took Part

Enrollment: 3 actual participants took part.

It was open to people of any sex aged from 5 years up to 12 years.

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

Examples of who could join: Children between the ages of 5-12 years of age. Language delay (lack of fluent phrase speech) reflected by use of ADOS Module 1 or 2.

The full record lists 4 inclusion and 8 exclusion criteria; among the exclusions: closed epiphyses; active or suspected neoplasia; intracranial hypertension.

How the Study Was Run

The set-up was randomized, crossover and quadruple-blind.

Random assignment meant chance, not the researchers, decided who got IGF-1 LR3 and who got the comparison, which limits bias.

Each person switched between IGF-1 LR3 and the comparison at some point, usually with a gap in between.

Neither the participants nor the staff measuring them knew who was on IGF-1 LR3, so expectations could not easily colour the readings.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2014-05 with completion listed for 2017-09-13.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Aberrant Behavior Checklist - Social Withdrawal Subscale (time frame: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8).

It also tracked 9 secondary outcomes, for example Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI), Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI), Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI), Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI), Repetitive Behavior and Caregiver Strain.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of IGF-1 LR3 in 3 people.

Its size is small, so on its own it can only give an early look.

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, all human trials.