FTACMT study protocol: a multicentre, double-blind, randomised, placebo-controlled trial of faecal microbiota transplantation for autism spectrum disorder

Ye Chen; Zhang Xueying; Cui Jiaqu; Chen Qiyi; Qin Huanlong; Li Ning; Du Yasong; Zhao Xiaoxin; Yang Rong; Lu Jubao; Lv Xiaoqiong; Ma Chunlian; Wang Yu; Chen Shidong; Kuang Guifang; Zhao Dongmei; Fang Shuanfeng; Zhang Xujing; Yang Binrang; Wang Yanxia; Li Ling; Yuan Song; Zhou Xiang; Zhang Beihua; Jiang Lin; Ji Hong; FTACMT Working Group

doi:10.1136/bmjopen-2021-051613

FTACMT study protocol: a multicentre, double-blind, randomised, placebo-controlled trial of faecal microbiota transplantation for autism spectrum disorder

BMJ Open. 2022 Jan 31;12(1):e051613. doi: 10.1136/bmjopen-2021-051613.

Authors

Ye Chen^#¹, Zhang Xueying^#¹, Cui Jiaqu¹, Chen Qiyi², Qin Huanlong¹, Li Ning¹, Du Yasong³, Zhao Xiaoxin³, Yang Rong¹, Lu Jubao¹, Lv Xiaoqiong¹, Ma Chunlian¹, Wang Yu⁴, Chen Shidong⁵, Kuang Guifang⁶, Zhao Dongmei⁷, Fang Shuanfeng⁸, Zhang Xujing⁹, Yang Binrang¹⁰, Wang Yanxia¹¹, Li Ling¹², Yuan Song¹³, Zhou Xiang¹⁴, Zhang Beihua¹⁵, Jiang Lin¹⁶, Ji Hong¹⁷; FTACMT Working Group

Collaborators

FTACMT Working Group:
Qin Huanlong, Li Ning, Chen Qiyi, Ye Chen, Zhang Xueying, Cui Jiaqu, Yang Rong, Lu Jubao, Yan Yinmei, Ma Chunlian, Lv Xiaoqiong, Yan Peihua, Huang Linsheng, Zhang Shaoyi, Du Yasong, Zhao Xiaoxin, Wang Yu, Ma Chenhuan, Chen Shidong, Miao Yun, Kuang Guifang, Ling Wenqi, Zhao Dongmei, Li Ning, Fang Shuanfeng, Li Ke, Zhang Xujing, Geng Bojing, Zhang Xin, Yang Binrang, Wang Yanxia, Feng Xueying, Li Lin, Li Huilin, Liu Liyan, Wei Wei, Wu Weijia, Yuan Song, Zhou Xiang, Li Dexin, Zhang Beihua, Jiang Qianru, Jiang Lin

Affiliations

¹ Department of Colorectal Diseases, Shanghai Tenth People's Hospital, Shanghai, China.
² Department of Colorectal Diseases, Shanghai Tenth People's Hospital, Shanghai, China qiyichen2011@163.com.
³ Child and Adolescent Psychiatry, Shanghai Mental Health Center, Shanghai, China.
⁴ Child Healthcare Department, Shanghai Children's Hospital, Shanghai, China.
⁵ Rehabilitation Medicine Department, Shanghai First People's Hospital, Shanghai, China.
⁶ Department of Pediatric Mental Health, Qingdao Women and Children's Hospital, Qingdao, China.
⁷ Institute of Child Health, Qilu Children's Hospital of Shandong University, Jinan, China.
⁸ Child Healthcare Department, Zhengzhou University Third Hospital and Henan Province Women and Children's Hospital, Zhengzhou, China.
⁹ Clinical Psychology, Hebei Mental Health Center, Baoding, China.
¹⁰ Child Healthcare Department, Shenzhen Children's Hospital, Shenzhen, China.
¹¹ Child Healthcare Department, The Affiliated Hospital of Qingdao University, Qingdao, China.
¹² Child Rehabilitation Department, Hainan Women and Children's Medical Center, Haikou, China.
¹³ Psychiatry Department, Zhoushan Second People's Hospital, Zhoushan, China.
¹⁴ Department of Children Psychology, Zhuhai Maternal and Child Health Care Hospital, Zhuhai, China.
¹⁵ Yangzhi Affiliated Rehabilitation Hospital of Tongji University, Shanghai, China.
¹⁶ Psychiatry Department, Dalian Seveth People's Hospital, Dalian, China.
¹⁷ Wuhu No.1 People's Hospital, Anhui, China.

^# Contributed equally.

Abstract

Introduction: Autism spectrum disorder (ASD) is a complicated diffuse developmental disorder that commonly involves gastrointestinal distress and dysbacteriosis. Emerging lines of evidence have shown faecal microbiota transplantation (FMT) to be a potential therapeutic strategy for improving the clinical outcomes of patients with ASD by re-establishing their intestinal microflora. We are undertaking the first-ever multicentre, double-blind, randomised controlled trial of FMT for the treatment of children with both ASD and gastrointestinal symptoms and will assess the feasibility and efficacy outcomes of this strategy.

Methods: In total, 318 children with both ASD and gastrointestinal symptoms will be enrolled (from 15 hospitals in China) to receive either FMT intervention (n=212) or a placebo (control, n=106). Children aged 3-6 years will take two capsules two times a day, and those older than 6 years will take three capsules two times a day. Each patient will receive four treatment courses, with each 12-day course being repeated every month. Outcomes will be evaluated at baseline, throughout the period of intervention, and at subsequent follow-ups for 2 months. The primary trial objective is to investigate the remodelling effect of FMT on the intestinal microflora in patients with ASD. The secondary objective focuses on the clinical efficacy and safety of FMT, including its improvement of the clinical response and metabonomics.

Ethics and dissemination: Ethical approval was obtained from the hospital Ethics Committee of each Faecal Transfer for ASD China Multicenter Trial Working Group. The ongoing FMT clinical trial is intended to support the approval of the new technology and its administration. The results of this trial will provide high-quality evidence to inform the future clinical application of this new therapy.

Trial registration number: ChiCTR2100043906; Pre-results.

Keywords: child & adolescent psychiatry; gastroenterology; microbiology.

Publication types

Clinical Trial Protocol
Research Support, Non-U.S. Gov't

MeSH terms

Autism Spectrum Disorder* / etiology
Autism Spectrum Disorder* / therapy
Child
Child, Preschool
Double-Blind Method
Fecal Microbiota Transplantation / methods
Feces
Gastrointestinal Microbiome*
Humans
Multicenter Studies as Topic
Randomized Controlled Trials as Topic
Treatment Outcome

Associated data

ChiCTR/ChiCTR2100043906