Clinical Research

Through our extensive Clinical Research Program, the Seaver Autism Center for Research and Treatment has gained significant expertise in the medical and behavioral treatment of children, adolescents, and adults with autism spectrum disorder and related conditions.

Our group has developed an effective experimental therapeutics program in psychosocial and psychopharmacological treatment approaches, incorporating established and novel treatment approaches. Our clinicians provide state-of-the-art assessments, among the best available in the world today. Their work also supports autism research across programs at the Icahn School of Medicine at Mount Sinai and enhances communication between the various programs and individual investigators, generating new ideas and innovative opportunities for outstanding clinical and basic science research.

The Seaver Autism Center is conducting the following clinical trials:

Study of Oxytocin in Autism to Improve Reciprocal Social Behavior (SOARS-B): The goal of this project is to evaluate intranasal oxytocin as a treatment for improving social difficulties in individuals with autism spectrum disorder. This is a phase 2 clinical trial using a multi-site, placebo-controlled, parallel group design followed by an open label extension phase. Children ages 3-17 may be eligible.

A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder: 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. Children ages 5-12 may be eligible.

Piloting Treatment with Insulin-like Growth Factor in Phelan-McDermid Syndrome: The purpose of this study is to pilot the use of Insulin-Like Growth Factor-1 (IGF-1) treatment in SHANK3 deficiency in order to evaluate safety, tolerability, and efficacy for a core deficit of autism – social impairment. IGF-1 is an injection under the skin that contains human IGF-1. IGF-1 is approved by the FDA under the brand name Increlex for the treatment of children with short stature due to primary IGF-1 deficiency. It is being used off-label in the current study and is not FDA approved, nor has it yet been studied in humans for the treatment of SHANK3 deficiency. Children ages 5-12 may be eligible.

Vasopressin Antagonist to Improve Social Communication in Autism (VANILLA): The VANILLA Autism Study will evaluate the efficacy of 12-week treatment with RO5285119 compared with placebo in treating social communication deficits in adult males with ASD. Males ages 18-45 may be eligible.

The Seaver Autism Center is conducting the following behavioral, neuroimaging, and genetic studies:

Mapping the Genotype, Phenotype, and Natural History of Phelan-McDermid Syndrome: The goal of this study is to gain a better understanding of Phelan-McDermid syndrome (PMS). This is a multi-center research study conducted as part of the Rare Disease Clinical Research Network (RDCRN) and sponsored by the National Institutes of Health (NIH).  Individuals between the ages of 3-21 diagnosed with PMS are eligible to participate in this study.

Early-Stage Visual Processing in ASD: Neurophysiological Biomarkers Using Visual Evoked Potentials: The purpose of this study is to identify biological markers for autism spectrum disorder using a type of brain wave measurement known as a visual evoked potential (VEP). This involves placing three non-invasive electrodes on top of the head while watching patterns on a computer monitor for approximately 10 minutes. Individuals may be eligible to participate if they or a sibling are affected by ASD or if they are typically developing.

Brain Imaging of Children with Developmental Delays or Autism: This study is examining the ways in which the brains of children with developmental delay or autism respond to speech and other sounds. We are also studying brain structure and the connections between brain regions. Children ages 2-17 may be eligible.

Genetic Testing: Genetic testing is available to all patients who participate in Seaver Autism Center research. Our clinicians work directly with our clinical geneticists to carry out basic and advanced genetic analyses on all consenting families. Our genetic testing can determine an etiological diagnosis in more than 20 percent of families, and this information may have important ramifications for predicting recurrence risk in families and identifying new genes in autism.

Autism BrainNet: The Icahn School of Medicine at Mount Sinai is one of the nodes of Autism BrainNet, a network of research institutions dedicated to groundbreaking brain research in autism.

Developing Scalable Measures of Behavior Change for ASD Treatment: The goal of this study is to develop and evaluate a new instrument for measuring change in social-communication behaviors in verbally fluent children with autism spectrum disorder. Participating in the study involves evaluating cognitive abilities, autism symptoms, social behaviors, treatment outcomes and adaptive functioning. Children between the ages of 4 and 16 may be eligible to participate.