Rett Syndrome, Tic Disorders, Tourette Syndrome
Conditions
Keywords
Rett syndrome, Tourette syndrome, Probiotic
Brief 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 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.
Interventions
DIETARY_SUPPLEMENTProbiotic-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
DIETARY_SUPPLEMENTPlacebo
placebo , 1 tablet , twice a day , 12 hrs apart, lasting 2 months for Tic disorders and 4 months for Rett syndrome
Sponsors
National Taiwan University Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
Randomized double blind placebo-controlled
Eligibility
Sex/Gender
ALL
Age
1 Years to 50 Years
Healthy volunteers
No
Inclusion criteria
* 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 * 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
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Neuropsychological test (Mullen Scales of Early Learning) | Change at four months from baseline | for Rett syndrome |
| Yale Global Tic Severity Scale (YGTSS) | Change at 2 months from baseline | for Tic disorders |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Swanson, Nolan and Pehlam version IV | Change at 2 months from baseline | For Tic disorders |
| RTT severity score | Change at four months from baseline | For Rett syndrome |
| Dystonia status (Fahn Marsden rating scale/Unified Dystonia Rating Scale ) | Visit 1, Visit 2, Visit 3, Visit 4 (Up to 4 months) | For Rett syndrome |
| Anxiety, Depression, and Mood Scale (ADAMS) | Change at four months from baseline | For Rett syndrome |
| Ghuman-Folstein Screen for Social Interaction (SSI) | Change at four months from baseline | For Rett syndrome |
| Vineland Adaptive Behavioral scale | Change at four months from baseline | For Rett syndrome |
| Children's Depression Inventory | Change at 2 months from baseline | For Tic disorders |
| Pediatric Evaluation of Disability Inventory | Change at four months from baseline | For Rett syndrome |
| Stool and gut microbiota evaluation | Change at four months from baseline | For Rett and Tic disorders |
| Child Behavior Checklist (CBCL) | Change at 2 months from baseline | For Tic disorders |
| Continuous performance test | Change at 2 months from baseline | For Tic disorders |
| The Migraine Disability Assessment Test | Change at 2 months from baseline | For Tic disorders |
| Early social communication scales | Change at four months from baseline | For Rett syndrome |
| Obsessive-compulsive inventory | Change at 2 months from baseline | For Tic disorders |
Countries
Taiwan
Contacts
Primary ContactWang-Tso Lee, MD,PhD
Backup ContactLee Chin Wong, MD
Outcome results
None listed