A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder
| Status: | Recruiting |
|---|---|
| Conditions: | Neurology, Psychiatric, Psychiatric, Autism |
| Therapuetic Areas: | Neurology, Psychiatry / Psychology |
| Healthy: | No |
| Age Range: | 5 - 12 |
| Updated: | 3/15/2019 |
| Start Date: | May 2014 |
| End Date: | January 2020 |
| Contact: | Jordana Norry |
| Email: | jordana.norry@mssm.edu |
| Phone: | 212-241-3072 |
The proposed project will pilot the use of IGF-1 as a novel treatment for core symptoms of
autism. We will use a double-blind, placebo-controlled crossover trial design in five
children with autism to evaluate the impact of IGF-1 treatment on autism-specific impairments
in socialization, language, and repetitive behaviors. We expect to provide evidence for the
safety and feasibility of IGF-1 in ameliorating social withdrawal in children with Autistic
Disorder. Further, we expect to demonstrate that IGF-1 is associated with improvement on
secondary outcomes of social impairment, language delay, and repetitive behavior, as well as
on functional outcomes of global severity.
autism. We will use a double-blind, placebo-controlled crossover trial design in five
children with autism to evaluate the impact of IGF-1 treatment on autism-specific impairments
in socialization, language, and repetitive behaviors. We expect to provide evidence for the
safety and feasibility of IGF-1 in ameliorating social withdrawal in children with Autistic
Disorder. Further, we expect to demonstrate that IGF-1 is associated with improvement on
secondary outcomes of social impairment, language delay, and repetitive behavior, as well as
on functional outcomes of global severity.
IGF-1 is an FDA approved, commercially available compound that crosses the blood-brain
barrier and has beneficial effects on synaptic development by promoting neuronal cell
survival, synaptic maturation, and synaptic plasticity. IGF-1 is effective in reversing mouse
and neuronal models of Rett syndrome and Phelan McDermid syndrome, both single gene causes of
ASD and may therefore be effective in treating autism spectrum disorders (ASD) more broadly.
barrier and has beneficial effects on synaptic development by promoting neuronal cell
survival, synaptic maturation, and synaptic plasticity. IGF-1 is effective in reversing mouse
and neuronal models of Rett syndrome and Phelan McDermid syndrome, both single gene causes of
ASD and may therefore be effective in treating autism spectrum disorders (ASD) more broadly.
Inclusion Criteria:
- Meet DSM-5 criteria for Autism Spectrum Disorder confirmed by the Autism Diagnostic
Interview-Revised (ADI-R) and the Autism Diagnostic Observation Schedule- Generic
(ADOS-G)
- 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
- Must be on stable medication regimens for at least three months prior to enrollment,
assuming the concomitant medication is safe for use with IGF-1
Exclusion Criteria:
- Closed epiphyses
- Active or suspected neoplasia
- Intracranial hypertension
- Hepatic insufficiency
- Renal insufficiency
- Cardiomegaly/valvulopathy
- History of allergy to IGF-1
- Patients with comorbid conditions deemed too medically compromised to tolerate the
risk of experimental treatment with IGF-1
We found this trial at
1
site
1428 Madison Ave
New York, New York 10029
New York, New York 10029
(212) 241-6500
Principal Investigator: Alexander Kolevzon, MD
Icahn School of Medicine at Mount Sinai Icahn School of Medicine at Mount Sinai is...
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