What side effects are associated with this type of intervention?

Common treatments for Autism Spectrum Disorder (ASD) include behavioral therapies, medications, and dietary interventions. Behavioral therapies generally have minimal side effects but can be time-consuming and require significant effort from caregivers. Medications such as antipsychotics (e.g., risperidone, aripiprazole) can help manage irritability and aggression but may cause weight gain, drowsiness, and hormonal changes. Stimulants used for co-occurring ADHD symptoms can lead to decreased appetite, sleep problems, and increased heart rate. Growth hormone therapy, as studied in the trial, aims to improve neurological and developmental functions but may cause side effects like joint pain, insulin resistance, and swelling. It is crucial to weigh the potential benefits and risks of these treatments with a healthcare provider.

What prior FDA approvals exist?

The FDA has not approved growth hormone specifically for the treatment of Autism Spectrum Disorder (ASD). However, other treatments have been approved to manage symptoms associated with ASD. For instance, Risperidone and Aripiprazole are FDA-approved for treating irritability in children and adolescents with ASD. These medications help manage symptoms such as aggression, self-injury, and severe tantrums. While these treatments do not directly stimulate growth or cell reproduction like growth hormone, they address some of the behavioral and neurological symptoms associated with ASD.

What other types of research exist?

Promising, novel alternatives to Growth Hormone for Autism Spectrum Disorder patients include gene therapy, antisense oligonucleotide therapies, trofinetide, and glatiramer acetate. These treatments aim to address underlying genetic and molecular dysfunctions and have shown potential in related conditions such as Rett syndrome.

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Hormone Therapy

Growth Hormone for Autism Spectrum Disorder

Phase 2

Waitlist Available

Led By Alexander Kolevzon, MD

Research Sponsored by Icahn School of Medicine at Mount Sinai

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Children between 2 and 12 years of age

No prior use of Growth Hormone or IGF-1

Must not have

Active or suspected neoplasia

Intracranial hypertension

Timeline

Screening 3 weeks

Treatment Varies

Follow Up after 12 weeks of growth hormone therapy

Summary

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.

Who is the study for?

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.

What is being tested?

The study tests if growth hormone can help kids with Phelan-McDermid syndrome and idiopathic autism improve social skills, language, and reduce repetitive behaviors. It's a double-blind test where half get the real medicine and half get saline (a placebo), without knowing which one they receive.

What are the potential side effects?

Possible side effects of the growth hormone may include joint pain, swelling due to fluid in the body's tissues (edema), muscle pain, tingling skin sensations (paresthesia), and changes in blood sugar levels.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My child is between 2 and 12 years old.

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I have never used Growth Hormone or IGF-1.

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I have a confirmed SHANK3 gene mutation.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have or might have a new tumor.

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I have high pressure in my brain.

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My liver is not working properly.

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My kidneys do not work well.

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My growth plates have closed.

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I have an enlarged heart or heart valve problems.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ after 12 weeks of growth hormone therapy

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~after 12 weeks of growth hormone therapy

This trial's timeline: 3 weeks for screening, Varies for treatment, and after 12 weeks of growth hormone therapy for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Visual Evoked Potentials (VEP)

Secondary study objectives

Aberrant Behavior Checklist (ABC) Social Withdrawal Subscale

Repetitive Behavior Scale-Revised (RBS-R)

Sensory Assessment for Neurodevelopmental Disorders (SAND)

+1 more

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Growth Hormone then SalineExperimental Treatment1 Intervention

12 weeks in each treatment phase (rhGH then placebo) and a four week wash-out period between phases.

Group II: Placebo (saline) then Growth HormonePlacebo Group1 Intervention

12 weeks in each treatment phase (placebo then rhGH) and a four week wash-out period between phases.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Growth Hormone

2014

Completed Phase 4

~1490

0 / 9Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Autism Spectrum Disorder (ASD) often target neurological and developmental functions. Growth hormone therapy, for instance, stimulates growth and cell reproduction, potentially improving neurological and developmental outcomes. Omega-3 fatty acids, although not consistently effective, are thought to support brain health and development. Melatonin is used to improve sleep patterns, which can significantly affect overall behavior and cognitive function. Oxytocin, a hormone involved in social bonding, is being studied for its potential to enhance social interactions and reduce repetitive behaviors in ASD patients. These treatments matter because they aim to address core symptoms and improve the quality of life for individuals with ASD by targeting underlying biological mechanisms.

Quantitative facial expression analysis revealed the efficacy and time course of oxytocin in autism.Neural Mechanisms of Qigong Sensory Training Massage for Children With Autism Spectrum Disorder: A Feasibility Study.Neurologic treatment strategies in autism: an overview of medical intervention strategies.