Clinical trial of insulin-like growth factor-1 in Phelan-McDermid syndrome

What Was Tested

The paper reports a study of IGF-1 LR3, published as a randomized controlled trial.

Phelan-McDermid syndrome (PMS) is caused by haploinsufficiency of the SHANK3 gene and is characterized by global developmental delays and autism spectrum disorder (ASD).

Based on several converging lines of preclinical and clinical evidence supporting the use of insulin-like growth factor-1 (IGF-1) in PMS, this study aims to follow-up a previous pilot study with IGF-1 to further evaluate this novel therapeutic for core symptoms of ASD in children with PMS.

Who Took Part

By PubMed's age tags, the people studied were children.

Ten children aged 5-9 with PMS were enrolled.

Participants were randomized to receive IGF-1 or placebo (saline) using a 12-week, double-blind, crossover design.

How the Study Was Run

The researchers set it up as a randomized, double-blind, placebo-controlled, crossover and pilot 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.

As a double-blind IGF-1 LR3 study, the assignments were hidden from both participants and investigators until the end.

Some participants received a placebo instead of IGF-1 LR3, giving a reference point.

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

What Was Measured

Based on several converging lines of preclinical and clinical evidence supporting the use of insulin-like growth factor-1 (IGF-1) in PMS, this study aims to follow-up a previous pilot study with IGF-1 to further evaluate this novel therapeutic for core symptoms of ASD in children with PMS.

The small sample size and expectancy bias due to relying on parent reported outcome measures may contribute to limitations in interpreting results.

The subject headings attached to it are Chromosome Deletion, Chromosome Disorders, Chromosomes, Human, Pair 22, Insulin-Like Growth Factor I and Pilot Projects.

Author keywords: ASD, Autism spectrum disorder, IGF-1, Insulin-like growth factor-1, PMS and Phelan-McDermid syndrome.

What This Means in Plain Language

The paper is a randomized, double-blind and placebo-controlled study of IGF-1 LR3.

Its findings take up roughly 5 sentences of the abstract and are left out here on purpose; the source paper is the place to read them.

The publication is from 2022 (Molecular autism), a journal based in England.

More context: the IGF-1 LR3 compound page, all human trials.