ASD Accompanied by Moderate to Severe Gastrointestinal SymptomsEarly Phase 1NCT04142255
Explore Efficacy of FMT Treating Childhood ASD Patients
A Early Phase 1 study testing FMT in people with ASD Accompanied by Moderate to Severe Gastrointestinal Symptoms, run by Xinhua Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 17 years
- Conditions the study is for
- ASD Accompanied by Moderate to Severe Gastrointestinal Symptoms
- What is being tested?
- FMT
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteChina
- Study dates
- 18 Nov 2019to 1 Jun 2020
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read the 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.
- Being tested
- FMT
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FMTBiological
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.
| Study group | Role | What they receive |
|---|---|---|
| FMT | Experimental | 20 subjects will be enrolled in this arm to receive FMT treatment. Receives: Biological: FMT |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Baseline to 12 weeks
Secondary outcomes (7)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: [16, 20, 24 weeks]
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2019
- Study start18 Nov 2019
- Primary completion1 Jun 2020
- Study completion1 Jun 2020
Why it stopped: The review board rejected our request of changing the protocol and advised us to terminate the trial.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ASD Accompanied by Moderate to Severe Gastrointestinal SymptomsStudies of the same condition, open and under way.
- More trials run by Xinhua Hospital, Shanghai Jiao Tong University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Fei Li, Professor, Principal investigator, Xinhua hospital Affilated to Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04142255
- Sponsor's protocol ID
- XH-19-008
- Status last verified
- Aug 2021
- First submitted
- 8 Sep 2019
- Last updated
- 3 Nov 2021
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Study to Explore the Efficacy of Fecal Mircobiota Transplantation (FMT) in the Treatment of Childhood ASD Patients With Gastrointestinal Symptoms
- ClinicalTrials.gov ID
- NCT04142255
- Acronym / sponsor ID
- XH-19-008
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The review board rejected our request of changing the protocol and advised us to terminate the trial.
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Study start
- 18 Nov 2019 (actual)
- Primary completion
- 1 Jun 2020 (actual)
- Study completion
- 1 Jun 2020 (actual)
- First posted
- 29 Oct 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FMT | Experimental | Biological: FMT20 subjects will be enrolled in this arm to receive FMT treatment. |
Who may qualify
- Ages
- 3 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- 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;
Key exclusion criteria (12 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FMTFMT 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. | Biological | FMT |
Primary outcome
Gastrointestinal Symptom Rating Scale (GSRS)
Time frame: Baseline to 12 weeks
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.
Childhood Autism Rating Scale (CARS)
Time frame: Baseline to 12 weeks
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.
7 secondary outcomes
Daily Stool Records (DSR)
Time frame: From the treatment period to Week 12
Safety and Tolerability
Time frame: Baseline to 12 weeks
Developmental Quotient/Intelligence Quotient Scale (DQ/IQ)
Time frame: Baseline to 12 weeks
Autism Diagnostic Observation Schedule (ADOS)
Time frame: Baseline to 12 weeks
Social Responsiveness Scale (SRS)
Time frame: Baseline to 12 weeks
Clinical Global Impressions-Improvement (CGI-I) Scale
Time frame: Baseline to 12 weeks
Long Term Safety Assessed Through Adverse Events
Time frame: [16, 20, 24 weeks]
Results and safety
The study was stopped early (reason given: The review board rejected our request of changing the protocol and advised us to terminate the trial.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2021.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Fei Li, Professor principal investigator
Xinhua hospital Affilated to Shanghai Jiao Tong University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing FMT in people with ASD Accompanied by Moderate to Severe Gastrointestinal Symptoms, run by Xinhua Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 3 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: The review board rejected our request of changing the protocol and advised us to terminate the trial.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Gastrointestinal Symptom Rating Scale (GSRS) (time frame: Baseline to 12 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Nov 2021.