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

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.

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Autism Spectrum Disorder; Autism
• Intervention / Treatment: Biological: Fecal Microbiota Transplant

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

• Study Type: Interventional
• Enrollment (Anticipated): 30
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Ventura, California, United States, 93003
      • ProgenaBiome

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 7 months and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Subject at least two years of age.
2. Subject has confirmed diagnosis of ASD based on the DSM-V.
3. Subject with ASD whose parents/caregiver/guardian/LAR can complete informed consent process.
4. A reliable parent or caregiver who can report the side effects and communicate effectively with the research team.
5. Stable medications during the two months prior to enrollment.
6. Currently receiving interventions in the community or school for ASD.
7. 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:

1. Female subjects who are pregnant, nursing, or intend to become pregnant during the study period.
2. Subjects whose caregivers are unable/unwilling to cooperate with the protocol requirements.
3. Subject will be excluded who are suffering with Rett syndrome and Childhood Disintegrative Disorder.
4. Other serious co-morbid medical disorders affecting brain function and behaviour, including uncontrolled seizures.
5. Subjects unable to refrain from taking non-study antibiotics for the period of the study.
6. Subjects diagnosed with cancer, except small localized basal cell carcinoma.
7. Subjects known to abuse alcohol or drugs.
8. Subjects who have undergone gastric bypass or total colectomy, or who are scheduled for these procedures.
9. Infection with HIV.
10. Infection with Hepatitis B or C.
11. Hepatic values greater than 20
12. Allergy to benzodiazepine.
13. Inability to stop loperamide, diphenoxylatye/atropine, or cholestyramine before the study
14. Unable to stop opiate treatment unless on a stable dose including PRN dosing, and no dose increase planned for the duration of the study
15. Known positive stool cultures for enteropathogens including, but not limited to Shigella, Salmonella, and Campylobacter within 30 days before enrollment.
16. Known stool studies positive for ova and/or parasites in 30 days prior to enrollment.
17. Planned travel outside United States during study period.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

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

Collaborators and Investigators

• Sponsor: ProgenaBiome
• Collaborators: Ventura Clinical Trials

Study record dates

Study Major Dates

• Study Start (Anticipated): May 1, 2022
• Primary Completion (Anticipated): June 1, 2025
• Study Completion (Anticipated): December 1, 2025

Study Registration Dates

• First Submitted: November 11, 2020
• First Submitted That Met QC Criteria: November 11, 2020
• First Posted (Actual): November 16, 2020

Study Record Updates

• Last Update Posted (Actual): February 25, 2022
• Last Update Submitted That Met QC Criteria: February 24, 2022
• Last Verified: February 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive
• Other Study ID Numbers: VCT-1

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No