Growth Hormone Clinical Trial for Kids with ASD

What's the study about?

This clinical trial is examining the use of recombinant human growth hormone (rhGH) in children (aged 2 – 12) with autism spectrum disorder (ASD). The study will follow a randomized, placebo-controlled, double-bind, crossover format, in which patients will receive 12 weeks of either rhGH or placebo, followed by 12 weeks of the opposite. Every patient will receive rhGH at some point in the trial.

Who can participate?

  • Kids aged 2-12 (inclusive) with a diagnosis of autism spectrum disorder (ASD).
  • No genetic cause identified on appropriate genetic testing; or willing to complete genetic testing.
  • Other eligibility criteria will be discussed and screened for.

What will participants be doing?

The study will involve routine monitoring for safety and progress with 4 in-person visits and 4 Zoom “visits”. In person visits will include a blood draw, X-ray, EKG, EEG, and other assessments. Extensive parent questionnaires and clinician-completed tools will be used to measure the effect of rhGH on social withdrawal, language, and repetitive behaviors.

Why is this important?

Pilot studies have shown that rhGH improves symptoms of social withdrawal in children with ASD. rhGH induces the release of IGF-1, a growth factor that is essential for brain development. It is known to promote brain cell survival and the maturation and plasticity of the connections between brain cells.