Explore Efficacy of FMT Treating Childhood ASD Patients

A Clinical Study to Explore the Efficacy of Fecal Mircobiota Transplantation (FMT) in the Treatment of Childhood ASD Patients With Gastrointestinal Symptoms

To explore the efficacy of fecal microbiota transplantation (FMT) in improving the gastrointestinal symptoms and autism symptoms in patients diagnosed as autism spectrum disorder (ASD) comorbid with gastrointestinal symptoms.

Study Overview

• Status: Terminated
• Conditions: ASD Accompanied by Moderate to Severe Gastrointestinal Symptoms
• Intervention / Treatment: Biological: FMT

Detailed Description

This trial is designed to reconstruct the intestinal micro-ecology of children with ASD by the interventional treatment of FMT. At the same time, combined with metagenomics and 16S rRNA sequencing techniques, the trial aims to study the efficacy of intestinal micro-ecology for the treatment of autism symptoms and the improvement of gastrointestinal problems and autism symptoms in autistic patients, and to explore potential new techniques for treating children with ASD.

• Study Type: Interventional
• Enrollment (Actual): 3
• Phase: Early Phase 1

Contacts and Locations

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China, 200092
      • ShanghaiXinhua

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 17 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Patients diagnosed with ASD. Diagnostic criteria: diagnosed by pediatric psychiatrists in accordance with the criteria in the Fifth Edition of Diagnostic and Statistical Manual of Mental Disorders (DSM-V), Autism Diagnostic Observation Schedule (ADOS) and Autism Diagnostic Interview-Revised (ADI-R);
2. Patients with moderate to severe gastrointestinal symptoms which are not life-threatening and do not require immediate surgery;
3. Patients aged 3-17 years, male or female;
4. Patients who are suitable for receiving oral FMT capsules, and able to swallow the capsules without chewing;
5. Patients who are able to complete the study as required by the trial protocol;
6. Patients without known immunodeficiency diseases;
7. Subjects and/or their guardians are able to fully understand the informed consent of the trial, and voluntarily sign the informed consent form.

Exclusion Criteria：

1. Patients diagnosed with severe malnutrition;
2. Patients depend on tube feeding (liquid food);
3. Patients with a serious gastrointestinal disease requiring immediate treatment (or life-threatening);
4. Patients with primary immunodeficiency disease during screening;
5. Patients with a single-gene disorder;
6. Patients with a history of severe allergies;
7. Patients with severe fever and/or serious infection within 7 days prior to enrollment;
8. Patients with renal insufficiency and liver dysfunction (or creatinine > 2 mg/dl, direct bilirubin > 2 mg/dl);
9. Subjects with congenital or acquired immunodeficiency or immunosuppression (e.g., neoplastic disease or organ transplant) who have received or are undergoing chemotherapy, or have been diagnosed with HIV;
10. Patients with inflammatory bowel disease, celiac disease, large intestine irritation or eosinophilia, esophagitis, eosinophilic gastroenteritis or similar diseases;
11. Subjects with severe sensory or motor disorders (e.g., blindness, deafness, seizures, cerebral palsy);
12. Patients who are participating in other clinical studies, or have received interventions in other interventional clinical studies within 4 weeks prior to enrollment.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: FMT; 20 subjects will be enrolled in this arm to receive FMT treatment.	Biological: FMT; FMT administration starts on the first week of enrollment. Age-based dose of FMT is taken once a day for the first three days of the first week. Then once a week for 3 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Gastrointestinal Symptom Rating Scale (GSRS)	The Gastrointestinal Symptom Rating Scale (GSRS) is an interview-based rating scale consisting of 15 items for assessment of bowel function, which refers to the period of the previous week and requires a short time to fill and has easy-to-understand questions on gastrointestinal symptoms. Changes in the Gastrointestinal Symptom Rating Scale (GSRS) before and after treatment.	Baseline to 12 weeks
Childhood Autism Rating Scale (CARS)	The CARS is a 15-item measure that clinicians complete based on observations from the child and subsequent interviews with the caregivers.The total score on the CARS reflects the symptom level of ASD with lower scores indicating minimal ASD symptoms and higher scores representing severe ASD symptoms. Changes in the Childhood Autism Rating Scale (CARS) before and after treatment.	Baseline to 12 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Daily Stool Records (DSR)	The Daily Stool Records (DSR) is a self-designed questionaire to record each participant's stool per day during the study. Changes in the Daily Stool Records (DSR) before and after treatment.	From the treatment period to Week 12
Safety and Tolerability	Safety and Tolerability refer to treatment-related adverse events as assessed by CTCAE v5.0 in the 12-week observation after the treatment.	Baseline to 12 weeks
Developmental Quotient/Intelligence Quotient Scale (DQ/IQ)	The (DQ/IQ) is evaluated by specialists in an intelligence test designed for children. Three versions of the intelligence test are the Gesell Developmental Schedules, the Wechsler Preschool and Primary Scale of Intelligence (WPPSI) and the Wechsler Intelligence Scale for Children-Revised (WISC-R), which are selected according to each participant's age. Changes in the Developmental Quotient/Intelligence Quotient Scale (DQ/IQ) before and after treatment.	Baseline to 12 weeks
Autism Diagnostic Observation Schedule (ADOS)	The ADOS is a structured play session conducted by clinicians, which includes a series of social communication and play "presses" to diagnose ASD. For younger children, social interest, joint attention, communication, symbolic play, and repetitive behaviors are assessed. Changes in the Autism Diagnostic Observation Schedule (ADOS) before and after treatment.	Baseline to 12 weeks
Social Responsiveness Scale (SRS)	The SRS measures the severity of social interaction impairments in ASD populations, sensitive between 3 to 18 years of age.It provides an impression of observed social impairments, assessing domains of communication, social interactions, and repetitive and stereotyped behaviors and interests. Changes in the Social Responsiveness Scale (SRS) before and after treatment.	Baseline to 12 weeks
Clinical Global Impressions-Improvement (CGI-I) Scale	The CGI-I assesses how much the patient's condition has improved or worsened relative to a baseline state, ranging from 1 (very much improved) to 7 (very much worse), and is recommended for all clinical trials involving participants with ASD.Improvement was defined as a score of 3 (minimally improved) or lower. Changes in the Clinical Global Impressions-Improvement (CGI-I) Scale before and after treatment.	Baseline to 12 weeks
Long Term Safety Assessed Through Adverse Events	Long Term Safety refers to treatment-related as assessed by CTCAE v5.0 after the 12-week study. Evaluation of safety telephone follow-up after 12 weeks.	[16, 20, 24 weeks]

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Head Magnetic resonance imaging (MRI)	Head MRI is used to detect the structure of the brain. Changes in the head MRI results of the subject before and after treatment.	Baseline to 12 weeks
Children's Dietary Diversity Questionnaire	Children's Dietary Diversity Questionnaire is a scale consisting of 8 categories of food each scoring from 0 to 2 based on the frequency. Changes in the subject's dietary diversity questionnaire results before and after treatment.	Baseline to 12 weeks

Collaborators and Investigators

• Sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
• Investigators: Principal Investigator: Fei Li, Professor, Xinhua hospital Affilated to Shanghai Jiao Tong University School of Medicine

Study record dates

Study Major Dates

• Study Start (ACTUAL): November 18, 2019
• Primary Completion (ACTUAL): June 1, 2020
• Study Completion (ACTUAL): June 1, 2020

Study Registration Dates

• First Submitted: September 8, 2019
• First Submitted That Met QC Criteria: October 25, 2019
• First Posted (ACTUAL): October 29, 2019

Study Record Updates

• Last Update Posted (ACTUAL): November 3, 2021
• Last Update Submitted That Met QC Criteria: October 27, 2021
• Last Verified: August 1, 2021

More Information

Terms related to this study

• Other Study ID Numbers: XH-19-008

Drug and device information, study documents

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