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

An Open-Label Phase I Clinical Trial of Vancomycin Followed by Familial Fecal Microbiota Transplant in Adult Subjects With Autism Spectrum Disorder (ASD)

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

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Sabine Hazan, MD; Phone Number: 18053390549; Email: drhazan@progenabiome.com
• Study Contact Backup: Name: Jordan Daniels, MS; Phone Number: 18053390549; Email: jordan@progenabiome.com

Study Locations

• United States
  • California
    • Ventura, California, United States, 93003
      • ProgenaBiome
      • Contact: Sabine Hazan, MD; Phone Number: 805-339-0549; Email: drsabinehazan@progenabiome.com
      • Contact: Jordan Daniels, MS; Phone Number: 805-339-0549; Email: jordan@progenabiome.com
      • Principal Investigator: Sabine Hazan, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Subject at least six 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. Comorbid gastrointestinal symptoms
6. Stable medications during the two months prior to enrollment.
7. Currently receiving interventions in the community or school for ASD.
8. If female and of childbearing potential, must be willing to utilize at least one highly effective method of birth control for the duration of the study. This includes oral birth control pills, contraceptive implants, and intra-uterine devices. Diaphragms and condoms are not considered highly effective. Subjects not of reproductive potential will be exempt (e.g., post-menopausal, surgically sterilized)
9. 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. This includes oral birth control pills, contraceptive implants, and intra-uterine devices Diaphragms and condoms are not considered highly effective. Subjects not of reproductive potential will be exempt (surgically sterilized)

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 behavior, 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. Allergy to benzodiazepine.
12. Inability to stop loperamide, diphenoxylatye/atropine, or cholestyramine before the study
13. Unable to stop opiate treatment unless on a stable dose including PRN dosing, and no dose increase planned for the duration of the study
14. Known positive stool cultures for enteropathogens including, but not limited to Shigella, Salmonella, and Campylobacter within 30 days before enrollment. (These infection agents should be treated in advance of participation in this FMT study because they affect the good flora).
15. Known stool studies positive for ova and/or parasites in 30 days prior to enrollment. (Same as above).
16. Planned travel outside United States during study period.
17. Hypersensitivity to vancomycin
18. Renal insufficiency
19. Colitis

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
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-2	Number of patients with changes in SRS-2 scores	52 weeks
Behavioral changes	Changes in reported incidence of disturbances in non-core aspects of behavior or function	52 weeks
GSRS	Number of patients with reported changes in GSRS measure of GI disturbances	52 weeks
abbrvCDAI	Number of patients with changes in abbrevCDAI scores	52 weeks
HSPP	Number of subjects with changes in quality of life metric HSPP	52 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Shannon Diversity Index	Changes in Shannon Diversity index compared between pre and post FMT scores	52 Weeks
Microbiome changes	Number of subjects with changes to microbiome relative abundance and composition	52 weeks

Other 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

Collaborators and Investigators

• Sponsor: ProgenaBiome
• Collaborators: Ventura Clinical Trials; Microbiome Research Foundation
• Investigators: Study Director: Sabine Hazan, MD, ProgenaBiome

Study record dates

Study Major Dates

• Study Start (Estimated): December 1, 2025
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): July 1, 2027

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 (Estimated): September 23, 2025
• Last Update Submitted That Met QC Criteria: September 18, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: FMT; microbiome; fecal microbiota transplant; dysbiosis; fecal transpant
• Additional Relevant MeSH Terms: Mental Disorders; Pathologic Processes; Neurodevelopmental Disorders; Child Development Disorders, Pervasive; Pathological Conditions, Signs and Symptoms; Autism Spectrum Disorder; Autistic Disorder; Dysbiosis; Therapeutics; Biological Therapy; Fecal Microbiota Transplantation
• Other Study ID Numbers: VCT-1

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: HIPAA

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No