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Past Studies | Overview

SynapDx Autism Spectrum Disorder Gene Expression Analysis Study

The primary objectives of this study are:

  1. to develop an algorithm to classify blood RNA gene expression patterns to maximize agreement between classification and a clinical assessment of presence or absence of ASD
  2. to prospectively assess the clinical sensitivity and specificity of the blood RNA gene expression classification algorithm in a separate population of children referred to a developmental evaluation clinic for possible developmental disorder.

Parent-Child Cognitive Behavioral Therapy for Anxiety in Young Children with Autism Spectrum Disorders

  • PI: Katherine Driscoll, PhD, and Dina Hirsfeld-Becker, PhD
  • Participants: 3- to 12-year-olds with a clinical diagnosis of ASD and their parents

Patient somatic cell donation for reprogramming experiments in neurological disease

  • PI: Mira Irons, MD, and Lisa Prock, MD, MPH
  • Participants: Children and adults of any age, preference given to children under 10, and participants with ASD, 15q11 dup, 16p11 del, Shank3, TSC2, UBE3A

This study uses stem cells created from patient skin or blood cells to characterize the patient's neurogenetic disease and try to develop new treatments.

Open Label Extension Arbaclofen in ASD (STX209) Study

  • PI: Bill Barbaresi, MD
  • Participants: Children or adults ages 3 to 18+ with a confirmed ASD diagnosis

Will provide data on the long-term safety and tolerability of STX209 among subjects with ASD who receive treatment under conditions reflective of their typical medical care rather than more restricted conditions in pivotal efficacy trials.

Neural markers of the transition from risk for attention-deficit/hyperactivity disorder (ADHD) to stable ADHD diagnosis

A randomized, double-blind, placebo-controlled, parallel group study to evaluate AFQ056 in adult patients with Fragile X Syndrome

A randomized, double-blind, placebo-controlled, parallel group study to evaluate the effects of AFQ056 combined with a reading intervention the reading abilities of male patients with Fragile X Syndrome