Explore Efficacy of FMT Treating Childhood ASD Patients

NCT04142255 | Terminated Early Phase 1

• 1 other identifier: XH-19-008
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

ASD Accompanied by Moderate to Severe Gastrointestinal Symptoms

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (7)

• Daily Stool Records (DSR)

  From the treatment period to Week 12

• Safety and Tolerability

  Baseline to 12 weeks

• Developmental Quotient/Intelligence Quotient Scale (DQ/IQ)

  Baseline to 12 weeks

• Autism Diagnostic Observation Schedule (ADOS)

  Baseline to 12 weeks

• Social Responsiveness Scale (SRS)

  Baseline to 12 weeks

Other Outcomes (2)

• Head Magnetic resonance imaging (MRI)

  Baseline to 12 weeks

• Children's Dietary Diversity Questionnaire

  Baseline to 12 weeks

Study Arms (1)

FMT

EXPERIMENTAL

20 subjects will be enrolled in this arm to receive FMT treatment.

Biological: FMT

Interventions

FMT BIOLOGICAL

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.

FMT

Eligibility Criteria

• Age: 3 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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);
• Patients with moderate to severe gastrointestinal symptoms which are not life-threatening and do not require immediate surgery;
• Patients aged 3-17 years, male or female;
• Patients who are suitable for receiving oral FMT capsules, and able to swallow the capsules without chewing;
• Patients who are able to complete the study as required by the trial protocol;
• Patients without known immunodeficiency diseases;
• Subjects and/or their guardians are able to fully understand the informed consent of the trial, and voluntarily sign the informed consent form.

You may not qualify if:

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

Sponsors & Collaborators

• Xinhua Hospital, Shanghai Jiao Tong University School of Medicine: lead

Study Sites (1)

ShanghaiXinhua

Shanghai, Shanghai Municipality, 200092, China

Location

Study Officials

• Fei Li, Professor

  Xinhua hospital Affilated to Shanghai Jiao Tong University School of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: September 8, 2019
• First Posted: October 29, 2019
• Study Start: November 18, 2019
• Primary Completion: June 1, 2020
• Study Completion: June 1, 2020
• Last Updated: November 3, 2021

Record last verified: 2021-08

Locations

CN (1)