Microbiota Transplant Therapy for Children With Both Autism Spectrum Disorder and Gastrointestinal Disorders
NCT Number: NCT06503978
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE2
Conditions: Autism Spectrum Disorder|Gastro-Intestinal Disorder|Constipation|Diarrhea
Interventions: COMBINATION_PRODUCT: MTP-101P|COMBINATION_PRODUCT: Vancomycin|COMBINATION_PRODUCT: Miralax|COMBINATION_PRODUCT: Antacid|COMBINATION_PRODUCT: Placebo MTP-101P|COMBINATION_PRODUCT: Placebo Vancomycin
Sponsor: Gut-Brain-Axis Therapeutics Inc.
Sex: ALL
Age: CHILD, ADULT
Enrollment: 60
Study Design: Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: QUADRUPLE (PARTICIPANT, CARE_PROVIDER, INVESTIGATOR, OUTCOMES_ASSESSOR)|Primary Purpose: TREATMENT
Start Date: 2024-07-31
Completion Date: 2026-08-17
Locations: Autism/Asperger's Research Group at Arizona State University, Tempe, Arizona, 85287, United States

Summary:
The investigators propose to investigate Microbiota Transfer Therapy (MTT) for treating patients with Autism-spectrum disorder with Gastrointestinal disorders (constipation, diarrhea, and/or abdominal pain). MTT involves a combination of 14 days of oral vancomycin (an antibiotic to kill pathogenic bacteria), followed by 1 day of bowel cleanse using Miralax, followed by 5 days of high dose MTP-101P with an antacid, followed by 12 weeks of a lower maintenance dose of MTP-101P with an antacid.

Primary Outcome Measures: Childhood Autism Rating Scale (CARS-2), A 15-item scale that can be used to both diagnose autism and ASD and to assess the overall severity of symptoms, Baseline (0-30 days prior to treatment) vs End of Treatment (Day 104)|Daily Stool Record (DSR), Participants will report the number of abnormal events during a 14 day period. An event includes no bowel movement during 1 day, unusually hard stool (Bristol Stool Form type 1-2), unusually soft stool (Bristol Stool Form type 6-7), four or more bowel movements in 1 day, abdominal pain, or use of GI medication. The units are number of events regardless of type of event., Screening (0-120 days prior to Baseline) vs Baseline (0-30 days prior to treatment) vs End of Treatment (Day 104)
Secondary Outcome Measures: Gastrointestinal Symptom Rating Scale (GSRS), An assessment of GI symptoms based on 15 questions, which are then scored in five domains (abdominal pain, reflux, indigestion, diarrhea, and constipation). Symptoms are rated on a 7-point Likert scale including no discomfort at all (1), slight discomfort (2), mild discomfort (3), moderate discomfort (4), moderately severe discomfort (5), severe discomfort (6), and very severe discomfort (7)., Baseline (0-30 days prior to treatment) VS Day 14, 28,56,84 and 104|Clinical Global Impression - Gastrointestinal (CGI-GI), This assessment is conducted by the study physician and assess the severity of GI symptoms on a 7-point scale. The severity scale rates the severity of the patient's illness including normal, not at all ill (1), borderline mentally ill (2), mildly ill (3), moderately ill (4), markedly ill (5), severely ill (6), among the most extremely ill patients (7)., Baseline (0-30 days prior to treatment) VS End of Treatment (Day 104)|Clinical Global Impression - Autism (CGI-A), This assessment is conducted by the study physician and assess the severity of Autism Symptoms on a 7-point scale. The severity scale rates the severity of the patient's illness including normal, not at all ill (1), borderline mentally ill (2), mildly ill (3), moderately ill (4), markedly ill (5), severely ill (6), among the most extremely ill patients (7)., Baseline (0-30 days prior to treatment) VS End of Treatment (Day 104)|National Survey on treatment Effectiveness for Autism (NSTEA), Assess the effectiveness of MTT on overall benefit (no benefit (0)/slight benefit(1)/moderate benefit (2)/good benefit (3)/great benefit (4))., End of Treatment (Day 104)|Social Responsiveness Scale (SRS 2), 65-item scale that assesses social impairments, a core issue in autism, including social awareness, social information processing, capacity for reciprocal social communication, social anxiety/avoidance, and autistic preoccupations and traits., Baseline (0-30 days prior to treatment) VS End of Treatment (Day 104)|Aberrant Behaviour Checklist (ABC-2), assesses problem behaviors in five areas common in individuals with ASD, including irritability, social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech., Baseline (0-30 days prior to treatment) VS End of Treatment (Day 104)|CAAAS (subscale on GI symptoms), This tool assesses both core ASD symptoms and a comprehensive list of common co-occurring symptoms in ASD, including gastrointestinal, behavioral, mental health, and physical health symptoms.

9-point severity scale from "none" to "major". There is also a "change", Baseline (0-30 days prior to treatment) VS Day 14, 28,56,84 and 104|Comprehensive Assessment of Autism and Associated Symptoms (CAAAS), This tool assesses both core ASD symptoms and a comprehensive list of common co-occurring symptoms in ASD, including gastrointestinal, behavioral, mental health, and physical health symptoms.

9-point severity scale from "none" to "major". There is also a "change", Baseline (0-30 days prior to treatment) VS Day 14, 28,56,84 and 104|Vineland Adaptive Behavior Scale -Third Edition (Vineland 3), Comprehensive form consisting of 381 items covering three domains (Communication, Daily Living Skills, and Socialization) and the Maladaptive Behavior subscale (44 items)., Baseline (0-30 days prior to treatment) VS End of Treatment (Day 104)