Tourette SyndromePhase 1WMTNCT03764748
Washed Microbiota Transplantation for Tourette's Syndrome
A Phase 1 study testing Washed Microbiota Transplantation in people with Tourette Syndrome, run by The Second Hospital of Nanjing Medical University at 2 sites in China.
Trial summary
This study aimed to evaluate the efficacy of washed microbiota transplantation in the treatment of Tourette's syndrome (TS). Read more about this study
- Who is it for?
- Ages 6 to 17 yearsHealthy volunteers can take part
- Conditions the study is for
- Tourette Syndrome
- What is being tested?
- Washed Microbiota Transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 2 sitesChina
- Study dates
- 17 Apr 2024to 31 Oct 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 6 to 17 years
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- Patients were aged 6 to 17 years (inclusive);
- Met the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition criteria for TS;
- Had a Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) of at least 20 at screening and baseline.
- Underwent WMT.
Who cannot join
- Complicated with certain brain disease, including tumor,injury,cerebrovascular disease;
- Complicated with other severe disease, including cancers, organ failure, heart diseases;
- Stereotypy associated with autism spectrum disorder;
- With a confirmed diagnosis of psychiatric diseases, such as bipolar disorders; schizophrenia and major depressive disorders;
- Clinically significant obsessive-compulsive disorder at baseline considered to be the primary cause of impairment at baseline;
- Medications use affecting gut microbiota such as antibiotics and probiotics three months before WMT.
- Other neurologic disorders other than TS that could influence the evaluation of tics.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites, 2 recruiting
- Recruiting
Department of Microbiota Medicine & Medical Centre for Digestive Diseases, The Second Affiliated Hospital of Nanjing Medical University
Nanjing, Jiangsu, 210011
Faming Zhang, PhD,MD, 86-025-58509883, fzhang@njmu.edu.cn
Faming Zhang, MD,PhD
- Recruiting
SIR RUN RUN hospital of Nanjing Medical University
Nanjing, Jiangsu, 210011
Faming Zhang, MD, PhD, 086-25-58509883, fzhang@njmu.edu.cn
About this study
Official title: Efficacy and Safety of Washed Microbiota Transplantation for Tourette's Syndrome
This study aimed to evaluate the efficacy of washed microbiota transplantation in the treatment of Tourette's syndrome (TS).
Read the detailed description
Emerging scientific data support the significant role of microbiota in the modulation of the central nervous system. The reconstitution of gut microbiota might be a potential option to treat Tourette's syndrome (TS). FMT has been reported to be a highly effective therapy to restore the gut microbiota dysbiosis by transferring gut microbiota from healthy donors to patients. The newly improved methodology of FMT based on the automatic washing process and the related delivering consideration was named as washed microbiota transplantation (WMT) by the FMT-standardization study group. FMT has shown treatment potential in several gut-brain axis-related disorders, such as amyotrophic lateral sclerosis and Parkinson's disease, promoting us to investigate its treatment potential for Tourette's syndrome. In this study, investigators aimed to evaluate the efficacy of WMT for Tourette's syndrome and its comorbidities using specialized questionnaires for Tourette's syndrome and investigate the underlying mechanism.
- Conditions
- Tourette Syndrome
- Being tested
- Washed Microbiota Transplantation
- Keywords
- Tic Disorders, Gut microbiota, Brain-gut axis, Washed Microbiota Transplantation, Tourette syndrome
- Medical subject headings
- Tourette Syndrome, Tic Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Washed Microbiota TransplantationDrug
Also called Fecal microbiota transplantation
The prepared microbiota suspension was infused into the participates' lower gut.
| Study group | Role | What they receive |
|---|---|---|
| Washed microbiota transplantation | Experimental | WMT Receives: Drug: Washed Microbiota Transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
The change of Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS)
The YGTSS rating scale is a semistructured clinician- or investigator-administered rating instrument in which participants or caregivers evaluate the number, frequency, intensity, complexity, and interference of motor and phonic tics in the prior week on a 6-point scale from 0 (none) to 5 (severe) [1]. The total Motor Tic Severity Score (MTSS; 0-25 points) is added to the total Vocal Tic Severity Score (VTSS; 0-25 points) to determine the TTS (0-50 points).
Measured: baseline, 4 weeks, 8 weeks, 12 weeks, 6 months post transplantation
Secondary outcomes (6)
the Clinical Global Impressions-Improvement scale
Positive treatment response was defined by a score of 1 (very much improved) or 2 (much improved) on the Clinical Global Impressions-Improvement scale. The Clinical Global Impression - Improvement scale is a 7 point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. and rated as:1.Very much improved; 2.Much improved; 3. Minimally improved; 4. No change; 5. Minimally worse; 6. Much worse; 7.Very much worse.
Measured: 4 weeks, 8 weeks, 12 weeks, 6 months post transplantation
the change of Gilles de la Tourette Syndrome Quality-of-Life Scale score
The Gilles de la Tourette Syndrome Quality-of-Life Scale with 27 items addresses the issue of how the illness affects patients' well-being. It ranges from 0 to108, and higher values represent a worse outcome.
Measured: baseline, 4 weeks, 8 weeks, 12 weeks, 6 months post transplantation
the change of The Swanson, Nolan, and Pelham IV Scale (SNAP-IV)
It is an 26-item questionnaire that measured three of the ADHD symptoms, inattention, hyperactivity and oppositional defiant disorder. The 26-item checklist is scored on a 4-point Likert scale ranging between Not At All (0) and Very Much (3).
Measured: baseline, 4 weeks, 8 weeks, 12 weeks, 6 months post transplantation
change of the Sleep Disturbance Scale for Children (SDSC)
The SDSC was 26-item test measuring sleep problems and each item received a value from 0 to 4, with higher scores indicating more severe sleep disorders.
Measured: baseline, 4 weeks, 8 weeks, 12 weeks, 6 months post transplantation
change of the Gastrointestinal Symptom Rating Scale (GSRS) .
It is based on 15 questions which are scored into 5 domains: abdominal pain, reflux, indigestion, diarrhea, and constipation. Each item weights from 1 to 7, with higher scores reflecting more severe symptoms.
Measured: baseline, 4 weeks, 8 weeks, 12 weeks, 6 months post transplantation
the difference of the gut microbe composition between individuals with TS and healthy children by sequencing faecal metagenome.
The composition of the gut microbiota is evaluated by sequencing faecal metagenome. We evaluate the differences in the structure of the flora and its metabolism between individuals with TS and control children at the phylum, genus and species levels of the intestinal flora, and to develop a model for predicting the structure of the flora.
Measured: baseline, 12 weeks, 6 months post transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Dec 2018
- Study start17 Apr 2024
- Primary completion31 Oct 2027 (estimated)
- Study completion31 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Tourette SyndromeStudies of the same condition, open and under way.
- 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
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The Second Hospital of Nanjing Medical University
- Sponsor type
- University, hospital or other
- Collaborators
- Nanjing Medical University
- Responsible party
- Principal investigator, Faming Zhang, The Second Hospital of Nanjing Medical University
- Investigators
- Faming Zhang, MD; PHD, Study chair, The Second Hospital of Nanjing Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03764748
- Sponsor's protocol ID
- WMT-TS
- Acronym
- WMT
- Status last verified
- Aug 2024
- First submitted
- 30 Nov 2018
- Last updated
- 19 Aug 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Hoffman BU, Lumpkin EA. A gut feeling. Science. 2018 Sep 21;361(6408):1203-1204. doi: 10.1126/science.aau9973. No abstract available.
PubMed 30237346Foster JA. Targeting the Microbiome for Mental Health: Hype or Hope? Biol Psychiatry. 2017 Oct 1;82(7):456-457. doi: 10.1016/j.biopsych.2017.08.002. No abstract available.
PubMed 28870296Roessner V, Plessen KJ, Rothenberger A, Ludolph AG, Rizzo R, Skov L, Strand G, Stern JS, Termine C, Hoekstra PJ; ESSTS Guidelines Group. European clinical guidelines for Tourette syndrome and other tic disorders. Part II: pharmacological treatment. Eur Child Adolesc Psychiatry. 2011 Apr;20(4):173-96. doi: 10.1007/s00787-011-0163-7.
PubMed 21445724Peng Z, Xiang J, He Z, Zhang T, Xu L, Cui B, Li P, Huang G, Ji G, Nie Y, Wu K, Fan D, Zhang F. Colonic transendoscopic enteral tubing: A novel way of transplanting fecal microbiota. Endosc Int Open. 2016 Jun;4(6):E610-3. doi: 10.1055/s-0042-105205. Epub 2016 Apr 28.
PubMed 27556065Zhang F, Cui B, He X, Nie Y, Wu K, Fan D; FMT-standardization Study Group. Microbiota transplantation: concept, methodology and strategy for its modernization. Protein Cell. 2018 May;9(5):462-473. doi: 10.1007/s13238-018-0541-8. Epub 2018 Apr 24.
PubMed 29691757
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Washed Microbiota Transplantation for Tourette's Syndrome
- ClinicalTrials.gov ID
- NCT03764748
- Acronym / sponsor ID
- WMT / WMT-TS
- Conditions
- Tourette Syndrome
- Keywords
- Tic Disorders, Gut microbiota, Brain-gut axis, Washed Microbiota Transplantation, Tourette syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- The Second Hospital of Nanjing Medical UniversityUniversity, hospital or other
- Collaborators
- Nanjing Medical University
- Study start
- 17 Apr 2024 (actual)
- Primary completion
- 31 Oct 2027 (estimated)
- Study completion
- 31 Oct 2027 (estimated)
- First posted
- 5 Dec 2018 (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.
This study aimed to evaluate the efficacy of washed microbiota transplantation in the treatment of Tourette's syndrome (TS).
Detailed description
Emerging scientific data support the significant role of microbiota in the modulation of the central nervous system. The reconstitution of gut microbiota might be a potential option to treat Tourette's syndrome (TS). FMT has been reported to be a highly effective therapy to restore the gut microbiota dysbiosis by transferring gut microbiota from healthy donors to patients. The newly improved methodology of FMT based on the automatic washing process and the related delivering consideration was named as washed microbiota transplantation (WMT) by the FMT-standardization study group. FMT has shown treatment potential in several gut-brain axis-related disorders, such as amyotrophic lateral sclerosis and Parkinson's disease, promoting us to investigate its treatment potential for Tourette's syndrome. In this study, investigators aimed to evaluate the efficacy of WMT for Tourette's syndrome and its comorbidities using specialized questionnaires for Tourette's syndrome and investigate the underlying mechanism.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Washed microbiota transplantation | Experimental | Drug: Washed Microbiota TransplantationWMT |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Patients were aged 6 to 17 years (inclusive);
- Met the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition criteria for TS;
- Had a Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) of at least 20 at screening and baseline.
- Underwent WMT.
Key exclusion criteria (7 listed)
- Complicated with certain brain disease, including tumor,injury,cerebrovascular disease;
- Complicated with other severe disease, including cancers, organ failure, heart diseases;
- Stereotypy associated with autism spectrum disorder;
- With a confirmed diagnosis of psychiatric diseases, such as bipolar disorders; schizophrenia and major depressive disorders;
- Clinically significant obsessive-compulsive disorder at baseline considered to be the primary cause of impairment at baseline;
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 |
|---|---|---|
| Washed Microbiota TransplantationAlso: Fecal microbiota transplantationThe prepared microbiota suspension was infused into the participates' lower gut. | Drug | Washed microbiota transplantation |
Primary outcome
The change of Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS)
Time frame: baseline, 4 weeks, 8 weeks, 12 weeks, 6 months post transplantation
The YGTSS rating scale is a semistructured clinician- or investigator-administered rating instrument in which participants or caregivers evaluate the number, frequency, intensity, complexity, and interference of motor and phonic tics in the prior week on a 6-point scale from 0 (none) to 5 (severe) [1]. The total Motor Tic Severity Score (MTSS; 0-25 points) is added to the total Vocal Tic Severity Score (VTSS; 0-25 points) to determine the TTS (0-50 points).
6 secondary outcomes
the Clinical Global Impressions-Improvement scale
Time frame: 4 weeks, 8 weeks, 12 weeks, 6 months post transplantation
the change of Gilles de la Tourette Syndrome Quality-of-Life Scale score
Time frame: baseline, 4 weeks, 8 weeks, 12 weeks, 6 months post transplantation
the change of The Swanson, Nolan, and Pelham IV Scale (SNAP-IV)
Time frame: baseline, 4 weeks, 8 weeks, 12 weeks, 6 months post transplantation
change of the Sleep Disturbance Scale for Children (SDSC)
Time frame: baseline, 4 weeks, 8 weeks, 12 weeks, 6 months post transplantation
change of the Gastrointestinal Symptom Rating Scale (GSRS) .
Time frame: baseline, 4 weeks, 8 weeks, 12 weeks, 6 months post transplantation
the difference of the gut microbe composition between individuals with TS and healthy children by sequencing faecal metagenome.
Time frame: baseline, 12 weeks, 6 months post transplantation
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2024.
- Background: PubMed 30237346Hoffman BU, Lumpkin EA. A gut feeling. Science. 2018 Sep 21;361(6408):1203-1204. doi: 10.1126/science.aau9973. No abstract available.
- Background: PubMed 28870296Foster JA. Targeting the Microbiome for Mental Health: Hype or Hope? Biol Psychiatry. 2017 Oct 1;82(7):456-457. doi: 10.1016/j.biopsych.2017.08.002. No abstract available.
- Background: PubMed 21445724Roessner V, Plessen KJ, Rothenberger A, Ludolph AG, Rizzo R, Skov L, Strand G, Stern JS, Termine C, Hoekstra PJ; ESSTS Guidelines Group. European clinical guidelines for Tourette syndrome and other tic disorders. Part II: pharmacological treatment. Eur Child Adolesc Psychiatry. 2011 Apr;20(4):173-96. doi: 10.1007/s00787-011-0163-7.
- Background: PubMed 27556065Peng Z, Xiang J, He Z, Zhang T, Xu L, Cui B, Li P, Huang G, Ji G, Nie Y, Wu K, Fan D, Zhang F. Colonic transendoscopic enteral tubing: A novel way of transplanting fecal microbiota. Endosc Int Open. 2016 Jun;4(6):E610-3. doi: 10.1055/s-0042-105205. Epub 2016 Apr 28.
- Background: PubMed 29691757Zhang F, Cui B, He X, Nie Y, Wu K, Fan D; FMT-standardization Study Group. Microbiota transplantation: concept, methodology and strategy for its modernization. Protein Cell. 2018 May;9(5):462-473. doi: 10.1007/s13238-018-0541-8. Epub 2018 Apr 24.
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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.
Faming Zhang, MD; PHD study chair
The Second Hospital of Nanjing Medical University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Washed Microbiota Transplantation in people with Tourette Syndrome, run by The Second Hospital of Nanjing Medical University at 2 sites 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 4 inclusion and 7 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: looking for participants now, as of the sponsor's last update on 19 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Faming Zhang, MD; PHD (025-58509883, fzhang@njmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The change of Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS) (time frame: baseline, 4 weeks, 8 weeks, 12 weeks, 6 months post transplantation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03764748, which also lists 5 linked publications.
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 19 Aug 2024.