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FMT in Children With Autism and Gastrointestinal Symptoms

Primary Purpose

Children With Autism

Status

Unknown status

Phase

Not Applicable

Locations

Israel

Study Type

Interventional

Intervention

FMT

FMT - placebo

About this trial

This is an interventional treatment trial for Children With Autism focused on measuring Fecal microbiota transplant, autism spectrum disorder, children

Eligibility Criteria

7 Years - 20 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

ASD Boys or girls 7 - 20 years of age
suffering gastro-intestinal symptoms and/or food selectivity that interfere with daily routine
Patients not started on immune-suppressive or anti-inflammatory medications.
Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Women of childbearing potential will have a urine pregnancy test, which must be negative, on Study Day 1, prior to receiving FMT. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study and for 3 months after FMT.
Hasn't taken antibiotics 2 days prior to 1st visit.
Ability of parents or other legal guardian to understand and the willingness to sign a written informed consent document, including the willingness to accept risk of unrelated donor stool.
Ability to swallow oral medications.

Exclusion Criteria:

Severe GI problems that require immediate treatment (life-threatening).
- recent/scheduled surgeries
- diagnosed as severely malnourished or underweight
- diagnosed with a single-gene disorder
- major brain malformations
- inflammatory bowel diseases
- known positive serology for celiac disease or eosinophilic esophagitis
- Pregnancy / breast-feeding
- Using immune-suppressive or anti-inflammatory medications
- Active or uncontrolled bacterial, viral, or fungal infection(s) requiring systemic therapy
- Delayed gastric emptying syndrome
- Known chronic aspiration
- History of significant allergy to foods
- Unable to swallow pills

Sites / Locations

Assaf-HarofehMC

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Fecal microbiota transplant (FMT)

placebo

Arm Description

10 capsules per dose for 6 consecutive weeks, twice a week, for a total of 120 capsules. Each inoculum will be prepared from the feces of 1 donor and a dose of 10 capsules contains sieved, concentrated material derived from a mean of 10 grams of fecal matter. Donors are healthy, nonpregnant adults aged 18 to 50 years, taking no medications, and with a normal body mass index (18.5-25 [calculated as weight in kilograms divided by height in meters squared]).

Placebo capsules will consist of a combination of saline/glycerol (same vehicle as FMT capsules). The inner capsules is dark (green colored), so the general appearance of the capsules is the same to the people who do not prepare them.

Outcomes

Primary Outcome Measures

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

The safety of using FMT capsules in autistic children with gastrointestinal symptoms is measured by the prevalence and severity of side effects that occurred after taking the capsules, and were documented in a daily follow-up questionnaire

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability])

Significant change in GI symptoms [efficiency]

effectiveness of FMT capsules in alleviation of GI symptoms in Autistic children is defined by a significant change in the Rome III questionnaire, used for diagnostic of pediatric functional GI disorders

Significant change in GI symptoms [efficiency]

Secondary Outcome Measures

Change in food selection [efficiency]

The effectiveness of FMT capsules on food selectivity in autistic children is measured using a eating habits questionnaire (Aut-Eat)

Change in food selection [efficiency]

The effectiveness of FMT capsules on food selectivity in autistic children is measured using a eating habits questionnaire (Aut-Eat)

Change in food selection [efficiency]

The effectiveness of FMT capsules on food selectivity in autistic children is measured using a eating habits questionnaire (Aut-Eat)

Improving ASD symptoms [efficiency]

The efficacy of FMT capsules in improving ASD symptoms is measured using the SRS questionnaire (Social Responsiveness Scale) The Total Score calculated separately for males and females. It can be expressed as a raw score or a T-score.

Improving ASD symptoms [efficiency]

Diversity and variability of the gut microbiome

Changing the diversity and variability of the gut microbiome measured by sequencing the 16's rRNA.

Diversity and variability of the gut microbiome

Changing the diversity and variability of the gut microbiome measured by sequencing the 16's rRNA.

Diversity and variability of the gut microbiome

Changing the diversity and variability of the gut microbiome measured by sequencing the 16's rRNA.

Diversity and variability of the gut microbiome

Changing the diversity and variability of the gut microbiome measured by sequencing the 16's rRNA.

Diversity and variability of the gut microbiome

Changing the diversity and variability of the gut microbiome measured by sequencing the 16's rRNA.

Full Information

NCT ID

NCT04246398

First Posted

January 6, 2020

Last Updated

February 2, 2020

Sponsor

Assaf Harofeh MC

1. Study Identification

Unique Protocol Identification Number

NCT04246398

Brief Title

FMT in Children With Autism and Gastrointestinal Symptoms

Official Title

FMT in Children With Autism and Gastrointestinal Symptoms

Study Type

Interventional

2. Study Status

Record Verification Date

February 2020

Overall Recruitment Status

Unknown status

Study Start Date

February 2020 (Anticipated)

Primary Completion Date

December 2022 (Anticipated)

Study Completion Date

December 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

Assaf Harofeh MC

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

5. Study Description

Brief Summary

ASD children with gastrointestinal problems that interfere with daily routine will be recruited at the center for Autism led by Prof Zachor, and at the Pediatric Neurology & Development Center at Shamir Medical Center. Publications in the social media- facebook and support groups for Autism will also be used. The decision whether or not to initiate any medical treatment will not be influenced by the study investigators and will be made solely by the treating doctor. If possible, participants will provide informed consent after receiving a thorough explanation by the study team, as will both parents or other legal guardians. Two groups randomization will be performed, group A and group B. The group randomization will be double blinded. Participants randomized to group A will receive 10 capsules FMT twice a week for 3 weeks, and at week 12 followed by 3 weeks placebo twice a week. Participants randomized to group B will receive 10 capsules placebo twice a week for 3 weeks and at week 12 followed by 3 weeks FMT twice a week. At baseline, participants will undergo a full physical examination, vital signs, medical questionnaire, ROME III questionnaire for kids >age4 />age 10, ATN-GISSI-17 questionnaire, Aut-Eat questionnaire regarding eating habbits, the Social Responsiveness Scale (SRS) and the Adaptive Behavior Assessment System (ABAS). Parents/ care providers will fill a daily symptoms follow-up questionnaire at baseline Fecal samples will be provided for analysis of microbiome and proteome at beseline, weeks 3,12, 15 and 6+9 months. At each FMT/Placebo administration a short questionnaire asking about possible adverse events, GI symptoms, overall well-being, and medication changes will be administered

Detailed Description

About 50% of children with Autism spectrum disorder (ASD) tend to suffer from one or more gastrointestinal (GI) symptoms.The GI symptoms vary from constipation (~20%), diarrhea (~14%), Abdominal pain/discomfort (14%), bloating (12%), incontinence (12%), reflux (7.4%), nausia (6%) and difficult with bowel movements (6%). The severity of ASD is associated with the observed GI symptoms, although no cause-effect relationship between GI symptoms and ASD was proven so far. ASD with GI symptoms patients might display significant behavioral manifestations, such as anxiety, self-injury and aggression and these may be attributed to the GI symptoms themselves. Therefore alleviation of the GI symptoms could be important also for improvement of ASD. In addition to the possibility that the nervous system influences the gut and the GI symptoms, there is also the possibility that the gut and the microbiome within the gut may influence the central nervous system. Considering the findings that children with ASD have a history of using significantly more antibiotics and that gut permeability increases in ASD it is reasonable to assume that ASD patients have distinct microbiomes and gut metabolites. Nevertheless, perhaps changing the gut microbiome could lead to improvement in some of the symptoms. So far, studies on influencing ASD (behavior and GI symptoms) by addressing the gut microbiome are optimistic but mostly open-labeled and still inconclusive.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Children With Autism

Keywords

Fecal microbiota transplant, autism spectrum disorder, children

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Crossover Assignment

Masking

Participant

Allocation

Randomized

Enrollment

50 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Fecal microbiota transplant (FMT)

Arm Type

Experimental

Arm Description

Arm Title

placebo

Arm Type

Placebo Comparator

Arm Description

Intervention Type

Other

Intervention Name(s)

FMT

Other Intervention Name(s)

Fecal microbiota transplant

Intervention Description

Participants will be asked to fast for 4 hours prior to and 1 hour following capsule intake. Each capsule will be taken with a sip of water, Capsules must not be crushed, chewed, or dissolved Participants will be evaluated for 15 minutes immediately following capsule administration.

Intervention Type

Other

Intervention Name(s)

FMT - placebo

Intervention Description

Primary Outcome Measure Information:

Title

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

Description

Time Frame

3 weeks from the start of the study

Title

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability])

Description

Time Frame

15 weeks from the start of the study

Title

Significant change in GI symptoms [efficiency]

Description

Time Frame

3 weeks from the start of the study

Title

Significant change in GI symptoms [efficiency]

Description

Time Frame

15 weeks from the start of the study

Title

Significant change in GI symptoms [efficiency]

Description

Time Frame

6 months from the start of the study

Title

Significant change in GI symptoms [efficiency]

Description

Time Frame

9 months from the start of the study

Secondary Outcome Measure Information:

Title

Change in food selection [efficiency]

Description

The effectiveness of FMT capsules on food selectivity in autistic children is measured using a eating habits questionnaire (Aut-Eat)

Time Frame

12 weeks from the start of the study

Title

Change in food selection [efficiency]

Description

The effectiveness of FMT capsules on food selectivity in autistic children is measured using a eating habits questionnaire (Aut-Eat)

Time Frame

6 months from the start of the study

Title

Change in food selection [efficiency]

Description

The effectiveness of FMT capsules on food selectivity in autistic children is measured using a eating habits questionnaire (Aut-Eat)

Time Frame

9 months from the start of the study

Title

Improving ASD symptoms [efficiency]

Description

Time Frame

3 months from the start of the study

Title

Improving ASD symptoms [efficiency]

Description

Time Frame

6 months from the start of the study

Title

Diversity and variability of the gut microbiome

Description

Changing the diversity and variability of the gut microbiome measured by sequencing the 16's rRNA.

Time Frame

3 weeks from the start of the study

Title

Diversity and variability of the gut microbiome

Description

Changing the diversity and variability of the gut microbiome measured by sequencing the 16's rRNA.

Time Frame

12 weeks from the start of the study

Title

Diversity and variability of the gut microbiome

Description

Changing the diversity and variability of the gut microbiome measured by sequencing the 16's rRNA.

Time Frame

15 weeks from the start of the study

Title

Diversity and variability of the gut microbiome

Description

Changing the diversity and variability of the gut microbiome measured by sequencing the 16's rRNA.

Time Frame

3 months from the start of the study

Title

Diversity and variability of the gut microbiome

Description

Changing the diversity and variability of the gut microbiome measured by sequencing the 16's rRNA.

Time Frame

6 months from the start of the study

10. Eligibility

Sex

All

Minimum Age & Unit of Time

7 Years

Maximum Age & Unit of Time

20 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: ASD Boys or girls 7 - 20 years of age suffering gastro-intestinal symptoms and/or food selectivity that interfere with daily routine Patients not started on immune-suppressive or anti-inflammatory medications. Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Women of childbearing potential will have a urine pregnancy test, which must be negative, on Study Day 1, prior to receiving FMT. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study and for 3 months after FMT. Hasn't taken antibiotics 2 days prior to 1st visit. Ability of parents or other legal guardian to understand and the willingness to sign a written informed consent document, including the willingness to accept risk of unrelated donor stool. Ability to swallow oral medications. Exclusion Criteria: Severe GI problems that require immediate treatment (life-threatening). recent/scheduled surgeries diagnosed as severely malnourished or underweight diagnosed with a single-gene disorder major brain malformations inflammatory bowel diseases known positive serology for celiac disease or eosinophilic esophagitis Pregnancy / breast-feeding Using immune-suppressive or anti-inflammatory medications Active or uncontrolled bacterial, viral, or fungal infection(s) requiring systemic therapy Delayed gastric emptying syndrome Known chronic aspiration History of significant allergy to foods Unable to swallow pills

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Ilan Youngster, M.D

Phone

0506301191

youngsteri@shamir.gov.il

Facility Information:

Facility Name

Assaf-HarofehMC

City

Zerifin

ZIP/Postal Code

70300

Country

Israel

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

FMT in Children With Autism and Gastrointestinal Symptoms

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