Growth Hormone for Autism Spectrum Disorder
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial will test if growth hormone can help children with Phelan-McDermid syndrome and idiopathic autism. Researchers hope it will improve their social skills, language, and reduce repetitive behaviors. The study will involve 45 children who will receive daily injections for a few months. Growth hormone has been previously tested in children with Phelan-McDermid syndrome, showing good tolerance without serious issues.
Will I have to stop taking my current medications?
The trial requires that participants be on stable medication regimens for at least three months before joining, as long as the medications are safe to use with growth hormone. So, you may not need to stop your current medications if they are considered safe.
Is growth hormone treatment generally safe for humans?
Growth hormone treatments like Omnitrope and Norditropin have been studied for safety in both children and adults with growth hormone deficiency and other conditions. These studies generally show a favorable safety profile, with most side effects being mild and not directly related to the treatment. Long-term studies in children and adults have not shown significant safety concerns.12345
How does the drug Growth Hormone differ from other treatments for Autism Spectrum Disorder?
What data supports the effectiveness of the drug Growth Hormone for Autism Spectrum Disorder?
While there is no direct evidence for Growth Hormone's effectiveness in Autism Spectrum Disorder, studies show that Growth Hormone therapy can significantly increase growth rates in children with growth hormone deficiency and other conditions like Turner's syndrome, suggesting potential benefits in growth-related outcomes.59101112
Who Is on the Research Team?
Alexander Kolevzon
Principal Investigator
Icahn School of Medicine at Mount Sinai
Are You a Good Fit for This Trial?
This trial is for children aged 2-12 with Phelan-McDermid syndrome or idiopathic autism. They must have open bone growth plates, be on stable treatments for three months, and not used growth hormone before. Kids with visual issues, severe health risks, allergies to growth hormone components, closed bone plates, or serious organ problems can't join.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Phase 1
Participants receive growth hormone or placebo as a once daily subcutaneous injection for 12 weeks
Wash-out
A four week wash-out period between treatment phases
Treatment Phase 2
Participants crossover to receive the alternate treatment (growth hormone or placebo) for another 12 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Growth Hormone
- Saline
Growth Hormone is already approved in United States, European Union, Canada for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
Icahn School of Medicine at Mount Sinai
Lead Sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborator