Clinical trial NCT03259971: The Role of Probiotics PS128 in Movement Disorders
Official title: The Role of Probiotics PS128 in Movement Disorders
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Rett Syndrome
Tourette Syndrome
Tic Disorders
Keywords: Rett syndrome
Tourette syndrome
Probiotic
Lead sponsor: National Taiwan University Hospital
Sponsor class: OTHER
First posted: 2017-08-24
Enrollment: 500
Sex: ALL
Minimum age (years): 1
Maximum age (years): 50
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Neuropsychological test (Mullen Scales of Early Learning)
timeFrame: Change at four months from baseline
measure: Yale Global Tic Severity Scale (YGTSS)
timeFrame: Change at 2 months from baseline
Secondary outcomes:
measure: RTT severity score
timeFrame: Change at four months from baseline
measure: Dystonia status (Fahn Marsden rating scale/Unified Dystonia Rating Scale )
timeFrame: Visit 1, Visit 2, Visit 3, Visit 4 (Up to 4 months)
measure: Anxiety, Depression, and Mood Scale (ADAMS)
timeFrame: Change at four months from baseline
measure: Ghuman-Folstein Screen for Social Interaction (SSI)
timeFrame: Change at four months from baseline
measure: Vineland Adaptive Behavioral scale
timeFrame: Change at four months from baseline
measure: Early social communication scales
timeFrame: Change at four months from baseline
measure: Pediatric Evaluation of Disability Inventory
timeFrame: Change at four months from baseline
measure: Stool and gut microbiota evaluation
timeFrame: Change at four months from baseline
measure: Child Behavior Checklist (CBCL)
timeFrame: Change at 2 months from baseline
measure: Continuous performance test
timeFrame: Change at 2 months from baseline
measure: The Migraine Disability Assessment Test
timeFrame: Change at 2 months from baseline
measure: Children's Depression Inventory
timeFrame: Change at 2 months from baseline
measure: Obsessive-compulsive inventory
timeFrame: Change at 2 months from baseline
measure: Swanson, Nolan and Pehlam version IV
timeFrame: Change at 2 months from baseline
Other outcomes:
Locations:
city: Taipei
country: Taiwan
facility: National Taiwan University Children Hospital
Study officials:
affiliation: National Taiwan University Children Hospital
name: Wang-Tso Lee, MD,PhD
role: PRINCIPAL_INVESTIGATOR
References: