Clinical Trial to Evaluate the Efficacy of Umbilical Cord Blood to Improve Outcomes for Children with Autism Spectrum Disorder

This program of research is sponsored by the Marcus Foundation.

Researchers at the Duke University Medical Center are conducting a program of research to evaluate the efficacy of autologous and allogeneic cord blood for improving outcomes of individuals with autism spectrum disorder.  The first study is underway and is examining the safety and efficacy of using autologous cord blood to treat young children with autism spectrum disorder and to assess the feasibility of various outcome measures to determine which measures can be used as primary and secondary endpoints for a future randomized phase 2 clinical trial. Cord blood cells can be collected from the placenta and stored for future usage in cord blood banks for cellular therapies or blood stem cell transplantation. Previous research has shown that cord blood cells can help reduce inflammation and signal cells to help repair damaged brain areas. This study is investigating whether similar success will be shown in children with ASD. In the first of several studies at Duke, researchers are testing the effects of autologous cord blood on behavioral outcomes in 25 boys and girls with autism spectrum disorder, ages 2 to 6 years old. The study lasts one year for each participant. Participants travel to Duke University Medical Center to participate in a variety of behavioral assessments and receive their own umbilical cord blood cells on their first visit. They return 6 months and 1 year after the first visit to participate in the same behavioral assessments in order to determine any effects. Some parent report measures are also completed by parents 3 and 9 months after the infusion in order to consistently keep track of any changes throughout the year. At the end of this study, families receive a score report summary including results of multiple behavioral assessments and describing any changes that were seen over the year.

Duke is the only site for the first study. Participants do not need to live locally but need to come to the Duke Center for Autism and Brain Development and the McGovern-Davison Children’s Health Center (CHC) for study visits. Enrollment for the first study is closed, but future studies are planned. 

Please contact 844-800-CORD or cordbloodtherapyinfo@dm.duke.edu  for more information. 

 

Criteria
Children ages 2-6 with a clinical diagnosis of ASD. Banked autologous cord blood also required.
Enrollment status
Completed