Natural History Study of Individuals With Autism and Germline Heterozygous PTEN Mutations

Status: Recruiting
Location: See all (5) locations...
Study Type: Observational
SUMMARY

The purpose of this study is to determine cross-sectional and longitudinal medical, behavioral, and cognitive differences between PTEN ASD and other groups, as well as to identify cognitive, neural systems, and molecular biomarkers specific to PTEN ASD. In addition, this study will be creating and maintaining a biorepository and linked phenotypic database for PTEN ASD.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 1
Healthy Volunteers: t
View:

• Individuals above the age of 18 months old at the time of consent who have documentation of a clinical diagnosis of autism spectrum disorder and/or a verified PTEN mutation from a medical or mental health professional for inclusion in the PTEN ASD, PTEN no-ASD or ASD macrocephaly groups.

• Macrocephaly (head circumference greater than or equal to 98th percentile) for inclusion in the ASD macrocephaly group.

• For youths, consent from parents or legal guardian. For adults, consent from self or legal guardian.

• Youths who are able (some young or severely impaired participants may not be able to provide assent) will be asked to provide assent as per IRB guidelines.

• Families with multiple children who meet the above inclusion criteria will be permitted to have as many children participate as they wish. A separate consent form will be filled out for each child enrolled in the study.

• Primary communicative language must be English

Locations
United States
California
University of California at Los Angeles
RECRUITING
Los Angeles
Stanford University Medical Center
RECRUITING
Stanford
Massachusetts
Boston Children's Hospital
RECRUITING
Boston
Ohio
Cincinnati Children's Hospital Medical Center
RECRUITING
Cincinnati
Cleveland Clinic
RECRUITING
Cleveland
Contact Information
Primary
Rajna Filip-Dhima, MS
Rajna.Filip-Dhima@childrens.harvard.edu
617-919-7068
Time Frame
Start Date: 2015-05
Estimated Completion Date: 2026-12
Participants
Target number of participants: 170
Treatments
PTEN ASD
PTEN participants with Autism Spectrum Disorder group
PTEN no ASD
PTEN participants without Autism Spectrum Disorder group
Controls
Healthy control group
Authors
Charis Eng
Sponsors
Collaborators: National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH), Office of Rare Diseases (ORD), Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), National Institute of Neurological Disorders and Stroke (NINDS)
Leads: Boston Children's Hospital

This content was sourced from clinicaltrials.gov