Familial Fecal Microbiota Transplant for the Treatment of Subjects With Autism Spectrum Disorders

Study Overview
=================
Brief Summary
-----------------
This is an open-label clinical trial to evaluate the benefits of familial fecal microbiota transplant following a 6-week treatment with Vancomycin in minor and adult subjects with ASD for treatment of social deficits and language delays.

Detailed Description
-----------------
This is an open-label clinical trial to evaluate the benefits of familial fecal microbiota transplant following a 6-week treatment with Vancomycin in minor and adult subjects with ASD for treatment of social deficits and language delays. Participants are given FMT by colonoscopy

Official Title
-----------------
An Open-Label Phase I Trial of Vancomycin Followed by Familial Fecal Microbiota Transplant in Minor and Adult Subjects With Autism Spectrum Disorder (ASD) for Treatment of Social Deficits and Language Delays.

Conditions
-----------------
Autism, Autism Spectrum Disorder

Intervention / Treatment
-----------------
* Biological: Fecal Microbiota Transplant


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Subject at least two years of age. Subject has confirmed diagnosis of ASD based on the DSM-V. Subject with ASD whose parents/caregiver/guardian/LAR can complete informed consent process. A reliable parent or caregiver who can report the side effects and communicate effectively with the research team. Stable medications during the two months prior to enrollment. Currently receiving interventions in the community or school for ASD. If male, and partner is of childbearing potential, must be willing to utilize at least one highly effective method of birth control for the duration of the study. Exclusion Criteria: Female subjects who are pregnant, nursing, or intend to become pregnant during the study period. Subjects whose caregivers are unable/unwilling to cooperate with the protocol requirements. Subject will be excluded who are suffering with Rett syndrome and Childhood Disintegrative Disorder. Other serious co-morbid medical disorders affecting brain function and behaviour, including uncontrolled seizures. Subjects unable to refrain from taking non-study antibiotics for the period of the study. Subjects diagnosed with cancer, except small localized basal cell carcinoma. Subjects known to abuse alcohol or drugs. Subjects who have undergone gastric bypass or total colectomy, or who are scheduled for these procedures. Infection with HIV. Infection with Hepatitis B or C. Hepatic values greater than 20 Allergy to benzodiazepine. Inability to stop loperamide, diphenoxylatye/atropine, or cholestyramine before the study Unable to stop opiate treatment unless on a stable dose including PRN dosing, and no dose increase planned for the duration of the study Known positive stool cultures for enteropathogens including, but not limited to Shigella, Salmonella, and Campylobacter within 30 days before enrollment. Known stool studies positive for ova and/or parasites in 30 days prior to enrollment. Planned travel outside United States during study period.

Ages Eligible for Study
-----------------
Minimum Age: 2 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Autism Subjects<br>These subjects will be administered fecal microbiota transplant by colonoscopy | Biological: Fecal Microbiota Transplant<br>* FMT utilizing stool from first degree relatives<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse Events | Number of participants with grade III or higher adverse events | 52 Weeks | 
| Serious Adverse Events | Number of participants with serous adverse events | 52 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| ATEC | Number of participants with changes in the Autism Treatment Evaluation Checklist scores | 52 Weeks | 
| CARS-2 | Number of participants with changes in Childhood Autism Rating Scale 2 scores | 52 Weeks | 
| QoLA | Number of participants with changes in Quality of Life Autism scores | 52 Weeks | 
| SRS-II | Number of participants with changes in Social Responsiveness Scale scores | 52 Weeks | 
| Shannon Diversity Index | Changes in Shannon Diversity index compared between pre and post FMT scores | 52 Weeks |