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

NCT04630847 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: VCT-1
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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 adult subjects with ASD for treatment of social deficits and language delays.

Conditions

Autism; Autism Spectrum Disorder

Keywords

microbiome; dysbiosis; FMT; fecal transpant; fecal microbiota transplant

Outcome Measures

Primary Outcomes (8)

• ATEC

  Number of participants with changes in the Autism Treatment Evaluation Checklist scores

  52 Weeks

• QoLA

  Number of participants with changes in Quality of Life Autism scores

  52 Weeks

• CARS-2

  Number of participants with changes in Childhood Autism Rating Scale 2 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 Outcomes (2)

• Shannon Diversity Index

  52 Weeks

• Microbiome changes

  52 weeks

Other Outcomes (2)

• Adverse Events

  52 Weeks

• Serious Adverse Events

  52 Weeks

Study Arms (1)

Autism Subjects

EXPERIMENTAL

These subjects will be administered fecal microbiota transplant by colonoscopy

Biological: Fecal Microbiota Transplant

Interventions

Fecal Microbiota Transplant BIOLOGICAL

FMT utilizing stool from first degree relatives

Autism Subjects

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subject at least six 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.
• Comorbid gastrointestinal symptoms
• Stable medications during the two months prior to enrollment.
• Currently receiving interventions in the community or school for ASD.
• 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)
• 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)

You may not qualify if:

• 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 behavior, 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.
• 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. (These infection agents should be treated in advance of participation in this FMT study because they affect the good flora).
• Known stool studies positive for ova and/or parasites in 30 days prior to enrollment. (Same as above).

Sponsors & Collaborators

• ProgenaBiome: lead
• Ventura Clinical Trials: collaborator
• Microbiome Research Foundation: collaborator

Study Sites (1)

ProgenaBiome

Ventura, California, 93003, United States

Location

MeSH Terms

Conditions

Autistic Disorder; Autism Spectrum Disorder; Dysbiosis

Interventions

Fecal Microbiota Transplantation

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics

Study Officials

• Sabine Hazan, MD

  ProgenaBiome

  STUDY DIRECTOR

Central Study Contacts

Sabine Hazan, MD

CONTACT

18053390549; drhazan@progenabiome.com

Jordan Daniels, MS

CONTACT

18053390549; jordan@progenabiome.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 11, 2020
• First Posted: November 16, 2020
• Study Start: December 1, 2025
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): July 1, 2027
• Last Updated: September 23, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)