Rett SyndromeNCT03259971
The Role of Probiotics PS128 in Movement Disorders
A study testing Probiotic-Lactobacillus plantarum PS128 in people with Rett Syndrome and Tourette Syndrome, run by National Taiwan University Hospital at 1 site in Taiwan.
Trial summary
Pediatric movement disorders comprise of a large number of different neurological diseases including Rett syndrome (RTT) and Tourette syndrome. Studies in the literature as well as our preliminary reports showed that Rett syndrome and Tourette syndrome/Tic disorders are associated with a dysbiosis of the gut… Read more about this study
- Who is it for?
- Ages 1 year to 50 years
- Conditions the study is for
- Rett Syndrome, Tourette Syndrome, Tic Disorders
- What is being tested?
- Probiotic-Lactobacillus plantarum PS128
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 500 participants
- Where
- 1 siteTaiwan
- Study dates
- 25 Aug 2017to 20 Aug 2021
- Phase
- Interventional
- 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 1 year to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Rett syndrome:
- Males and females who have classic or atypical RTT diagnosed under consensus criteria
- Age: 1-50 years
- Tic disorders:
- males and females with clinical diagnosis
- Age: 8-18years
Show all 11Show fewer
- Treatment naive 4 weeks prior to study
- Those with standard medications, dosage stable 4 weeks prior to study
- AADC syndrome:
- Males and females with AADC diagnosis
- Age: 1-50 years
Who cannot join
- Those who took probiotic or probiotic related product 4 weeks prior to the study or during the study
- Those who took antibiotic 4 weeks prior to the study
- Those showing poor compliance with any aspect of the study
- Those had adverse reactions to PS128
Trial locations and contacts
1 site in 1 country.
Taiwan1 site, 1 recruiting
- Recruiting
National Taiwan University Children Hospital
Taipei, 100
Wang-Tso Lee, PhD, 02-23123456 ext. 71618, leeped@hotmail.com
About this study
Pediatric movement disorders comprise of a large number of different neurological diseases including Rett syndrome (RTT) and Tourette syndrome. Studies in the literature as well as our preliminary reports showed that Rett syndrome and Tourette syndrome/Tic disorders are associated with a dysbiosis of the gut microbiota compared to normal control. Probiotic Lactobacillus plantarum PS128 is a diet supplement that available for human consumption. Probiotic Lactobacillus plantarum PS128 had showed psychotropic effects such as ameliorate anxiety- and depression-like behaviors as well as altered the level of neurotransmitters such as dopamine in the brain in animal models, which might be through microbiota-gut-brain axis. Therefore, the purpose of this study is to access the possible neurobehavior effects of Probiotic Lactobacillus plantarum PS128 in Rett syndrome and Tic disorders/Tourette syndrome.
Read the detailed description
The study is a placebo-controlled double-blind randomized study, to determine the benefits of probiotic Lactobacillus plantarum PS128 vs placebo on symptoms for Tic disorders; and the symptoms, behavior, or cognition for Rett syndrome.
Patients with the Rett syndrome and Tic disorders/Tourette syndrome will be recruited into our study.The study period will last 4 months for Rett syndrome patients, age one year-50years; while 2 months for Tic disorders/Tourette syndrome patient, age 5 years-18 years.
The study will be performed in National Taiwan University Children Hospital. The patients will have neuropsychological and gut microbiota evaluation before and at the end of the study. There will also be interim follow up evaluations each month after the patient start taking the probiotic or placebo.
- Conditions
- Rett Syndrome, Tourette Syndrome, Tic Disorders
- Being tested
- Probiotic-Lactobacillus plantarum PS128, Placebo
- Keywords
- Rett syndrome, Tourette syndrome, Probiotic
- Medical subject headings
- Rett Syndrome, Tourette Syndrome, Tic Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Probiotic-Lactobacillus plantarum PS128Dietary supplement
Also called Lactobacillus plantarum PS128
Probiotic supplement of Lactobacillus plantarum PS128 , 1 tablet , twice a day , 12 hrs apart, lasting 2 months for Tic disorders and 4 months for Rett syndrome
PlaceboDietary supplement
placebo , 1 tablet , twice a day , 12 hrs apart, lasting 2 months for Tic disorders and 4 months for Rett syndrome
| Study group | Role | What they receive |
|---|---|---|
| Placebo (for tic disorder) | Placebo comparator | The placebo will be dispensed to equal the volume of 1 tablet of Probiotic PS128, which is taken orally in 2 divided doses, 12 hours apart during the study period of 2 months for Tic disorders. Receives: Dietary Supplement: Placebo |
| PS128 (tic disorder) | Active comparator | The Probiotic group in tic disorder will take Probiotic-Lactobacillus plantarum PS128, which contain 300mg (3x10^10 CFU) Lactobacillus plantarum PS128 and 100mg of Microcrystalline Cellulose in each tablet. It will be taken orally in 2 divided doses, 12 hours apart during the study period of 2 months for Tic disorders. Receives: Dietary Supplement: Probiotic-Lactobacillus plantarum PS128 |
| Placebo (for Rett syndrome) | Placebo comparator | The placebo will be dispensed to equal the volume of 1 tablet of Probiotic PS128, which is taken orally in 2 divided doses, 12 hours apart during the study period of 4 months for Rett syndrome Receives: Dietary Supplement: Placebo |
| PS128 (Rett syndrome) | Active comparator | The Probiotic group will take Probiotic-Lactobacillus plantarum PS128, which contain 300mg (3x10^10 CFU) Lactobacillus plantarum PS128 and 100mg of Microcrystalline Cellulose in each tablet. It will be taken orally in 2 divided doses, 12 hours apart during the study period of 4 months for Rett Syndrome. Receives: Dietary Supplement: Probiotic-Lactobacillus plantarum PS128 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Neuropsychological test (Mullen Scales of Early Learning)
for Rett syndrome
Measured: Change at four months from baseline
Yale Global Tic Severity Scale (YGTSS)
for Tic disorders
Measured: Change at 2 months from baseline
Secondary outcomes (14)
RTT severity score
For Rett syndrome
Measured: Change at four months from baseline
Dystonia status (Fahn Marsden rating scale/Unified Dystonia Rating Scale )
For Rett syndrome
Measured: Visit 1, Visit 2, Visit 3, Visit 4 (Up to 4 months)
Anxiety, Depression, and Mood Scale (ADAMS)
For Rett syndrome
Measured: Change at four months from baseline
Ghuman-Folstein Screen for Social Interaction (SSI)
For Rett syndrome
Measured: Change at four months from baseline
Vineland Adaptive Behavioral scale
For Rett syndrome
Measured: Change at four months from baseline
Early social communication scales
For Rett syndrome
Measured: Change at four months from baseline
Pediatric Evaluation of Disability Inventory
For Rett syndrome
Measured: Change at four months from baseline
Stool and gut microbiota evaluation
For Rett and Tic disorders
Measured: Change at four months from baseline
Child Behavior Checklist (CBCL)
For Tic disorders
Measured: Change at 2 months from baseline
Continuous performance test
For Tic disorders
Measured: Change at 2 months from baseline
The Migraine Disability Assessment Test
For Tic disorders
Measured: Change at 2 months from baseline
Children's Depression Inventory
For Tic disorders
Measured: Change at 2 months from baseline
Obsessive-compulsive inventory
For Tic disorders
Measured: Change at 2 months from baseline
Swanson, Nolan and Pehlam version IV
For Tic disorders
Measured: Change at 2 months from baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Aug 2017
- Study start25 Aug 2017
- Primary completion20 Aug 2021 (estimated)
- Study completion20 Aug 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Rett SyndromeStudies of the same condition, open and under way.
- More trials run by National Taiwan University HospitalThe 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
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 500 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Taiwan University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Wang-Tso Lee, MD,PhD, Principal investigator, National Taiwan University Children Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03259971
- Sponsor's protocol ID
- 201703061MIPA
- Status last verified
- Apr 2020
- First submitted
- 16 Aug 2017
- Last updated
- 22 Oct 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Wong LC, Hsu CJ, Wu YT, Chu HF, Lin JH, Wang HP, Hu SC, Tsai YC, Tsai WC, Lee WT. Investigating the impact of probiotic on neurological outcomes in Rett syndrome: A randomized, double-blind, and placebo-controlled pilot study. Autism. 2024 Sep;28(9):2267-2281. doi: 10.1177/13623613231225899. Epub 2024 Feb 15.
PubMed 38361371
Study at a glance
From the registry record, last updated by the sponsor on 22 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Role of Probiotics PS128 in Movement Disorders
- ClinicalTrials.gov ID
- NCT03259971
- Acronym / sponsor ID
- 201703061MIPA
- Conditions
- Rett Syndrome, Tourette Syndrome, Tic Disorders
- Keywords
- Rett syndrome, Tourette syndrome, Probiotic
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- National Taiwan University HospitalUniversity, hospital or other
- Study start
- 25 Aug 2017 (actual)
- Primary completion
- 20 Aug 2021 (estimated)
- Study completion
- 20 Aug 2021 (estimated)
- First posted
- 24 Aug 2017 (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.
Pediatric movement disorders comprise of a large number of different neurological diseases including Rett syndrome (RTT) and Tourette syndrome. Studies in the literature as well as our preliminary reports showed that Rett syndrome and Tourette syndrome/Tic disorders are associated with a dysbiosis of the gut microbiota compared to normal control. Probiotic Lactobacillus plantarum PS128 is a diet supplement that available for human consumption. Probiotic Lactobacillus plantarum PS128 had showed psychotropic effects such as ameliorate anxiety- and depression-like behaviors as well as altered the level of neurotransmitters such as dopamine in the brain in animal models, which might be through microbiota-gut-brain axis. Therefore, the purpose of this study is to access the possible neurobehavior effects of Probiotic Lactobacillus plantarum PS128 in Rett syndrome and Tic disorders/Tourette syndrome.
Detailed description
The study is a placebo-controlled double-blind randomized study, to determine the benefits of probiotic Lactobacillus plantarum PS128 vs placebo on symptoms for Tic disorders; and the symptoms, behavior, or cognition for Rett syndrome.
Patients with the Rett syndrome and Tic disorders/Tourette syndrome will be recruited into our study.The study period will last 4 months for Rett syndrome patients, age one year-50years; while 2 months for Tic disorders/Tourette syndrome patient, age 5 years-18 years.
The study will be performed in National Taiwan University Children Hospital. The patients will have neuropsychological and gut microbiota evaluation before and at the end of the study. There will also be interim follow up evaluations each month after the patient start taking the probiotic or placebo.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Placebo (for tic disorder) | Placebo comparator | Dietary Supplement: PlaceboThe placebo will be dispensed to equal the volume of 1 tablet of Probiotic PS128, which is taken orally in 2 divided doses, 12 hours apart during the study period of 2 months for Tic disorders. |
| PS128 (tic disorder) | Active comparator | Dietary Supplement: Probiotic-Lactobacillus plantarum PS128The Probiotic group in tic disorder will take Probiotic-Lactobacillus plantarum PS128, which contain 300mg (3x10^10 CFU) Lactobacillus plantarum PS128 and 100mg of Microcrystalline Cellulose in each tablet. It will be taken orally in 2 divided doses, 12 hours apart during the study period of 2 months for Tic disorders. |
| Placebo (for Rett syndrome) | Placebo comparator | Dietary Supplement: PlaceboThe placebo will be dispensed to equal the volume of 1 tablet of Probiotic PS128, which is taken orally in 2 divided doses, 12 hours apart during the study period of 4 months for Rett syndrome |
| PS128 (Rett syndrome) | Active comparator | Dietary Supplement: Probiotic-Lactobacillus plantarum PS128The Probiotic group will take Probiotic-Lactobacillus plantarum PS128, which contain 300mg (3x10^10 CFU) Lactobacillus plantarum PS128 and 100mg of Microcrystalline Cellulose in each tablet. It will be taken orally in 2 divided doses, 12 hours apart during the study period of 4 months for Rett Syndrome. |
Who may qualify
- Ages
- 1 year to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- Rett syndrome:
- Males and females who have classic or atypical RTT diagnosed under consensus criteria
- Age: 1-50 years
- Tic disorders:
- males and females with clinical diagnosis
Key exclusion criteria (4 listed)
- Those who took probiotic or probiotic related product 4 weeks prior to the study or during the study
- Those who took antibiotic 4 weeks prior to the study
- Those showing poor compliance with any aspect of the study
- Those had adverse reactions to PS128
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 |
|---|---|---|
| Probiotic-Lactobacillus plantarum PS128Also: Lactobacillus plantarum PS128Probiotic supplement of Lactobacillus plantarum PS128 , 1 tablet , twice a day , 12 hrs apart, lasting 2 months for Tic disorders and 4 months for Rett syndrome | Dietary supplement | PS128 (Rett syndrome), PS128 (tic disorder) |
| Placeboplacebo , 1 tablet , twice a day , 12 hrs apart, lasting 2 months for Tic disorders and 4 months for Rett syndrome | Dietary supplement | Placebo (for Rett syndrome), Placebo (for tic disorder) |
Primary outcome
Neuropsychological test (Mullen Scales of Early Learning)
Time frame: Change at four months from baseline
for Rett syndrome
Yale Global Tic Severity Scale (YGTSS)
Time frame: Change at 2 months from baseline
for Tic disorders
14 secondary outcomes
RTT severity score
Time frame: Change at four months from baseline
Dystonia status (Fahn Marsden rating scale/Unified Dystonia Rating Scale )
Time frame: Visit 1, Visit 2, Visit 3, Visit 4 (Up to 4 months)
Anxiety, Depression, and Mood Scale (ADAMS)
Time frame: Change at four months from baseline
Ghuman-Folstein Screen for Social Interaction (SSI)
Time frame: Change at four months from baseline
Vineland Adaptive Behavioral scale
Time frame: Change at four months from baseline
Early social communication scales
Time frame: Change at four months from baseline
Pediatric Evaluation of Disability Inventory
Time frame: Change at four months from baseline
Stool and gut microbiota evaluation
Time frame: Change at four months from baseline
Child Behavior Checklist (CBCL)
Time frame: Change at 2 months from baseline
Continuous performance test
Time frame: Change at 2 months from baseline
The Migraine Disability Assessment Test
Time frame: Change at 2 months from baseline
Children's Depression Inventory
Time frame: Change at 2 months from baseline
Obsessive-compulsive inventory
Time frame: Change at 2 months from baseline
Swanson, Nolan and Pehlam version IV
Time frame: Change at 2 months from baseline
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Oct 2020.
- Derived from this study: PubMed 38361371Wong LC, Hsu CJ, Wu YT, Chu HF, Lin JH, Wang HP, Hu SC, Tsai YC, Tsai WC, Lee WT. Investigating the impact of probiotic on neurological outcomes in Rett syndrome: A randomized, double-blind, and placebo-controlled pilot study. Autism. 2024 Sep;28(9):2267-2281. doi: 10.1177/13623613231225899. Epub 2024 Feb 15.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Wang-Tso Lee, MD,PhD principal investigator
National Taiwan University Children Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Probiotic-Lactobacillus plantarum PS128 in people with Rett Syndrome and Tourette Syndrome, run by National Taiwan University Hospital at 1 site in Taiwan.
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 11 inclusion and 4 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: status not confirmed, as of the sponsor's last update on 22 Oct 2020. 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 Taiwan. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Wang-Tso Lee, MD,PhD (+886-223123456 ext. 71618, leeped@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Neuropsychological test (Mullen Scales of Early Learning) (time frame: Change at four months from baseline). 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?
On the ClinicalTrials.gov record for NCT03259971, which also lists 1 linked publication.
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 22 Oct 2020.