A whole new research area studying the function of intestinal microorganisms, also known as gut microbiota, has emerged during the last decade. As a result, dietary supplementation with specific bacteria (or probiotics) holds great promise as a therapeutic strategy for a wide range of diseases, from obesity to anxiety and depression, all of which are major characteristics of Prader-Willi syndrome (PWS). The main objective of the current proposal is to determine the effects of Bifidobacterium animalis subsp. lactis (strain BPL1) supplementation in children and young adults with PWS. Specifically, participants will receive placebo or BPL1 for 6 months, and then this phase will be followed by a 6-month extension period in which all participants will receive BPL1. This study will allow us to 1) determine the effects on fat mass and glucose metabolism; and 2) explore the effects on mental health symptoms by studying potential structural changes in the brain by magnetic resonance imaging (MRI) as well as using a number of psychiatric questionnaires.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
41
Intervention with a daily dose of placebo for 6 months
Intervention with a daily dose of probiotic for 6 months
Intervention with a daily dose of probiotic for 6 months
Hospital Sant Joan de Deu
Barcelona, Spain
Change in percent body fat content
Body fat content will be measured by dual energy x-ray absorptiometry (DXA)
Time frame: 6 months
Change in BMI and BMI z-score
Weight and height will be combined to calculate changes in BMI and BMI z scores from all subjects will be assessed
Time frame: Baseline, 6, and 12 months
Change in height (cm)
Changes in height from subjects that have not yet reached final height will be measured.
Time frame: Baseline, 6, and 12 months
Change in insulin sensitivity
Fasting blood glucose and insulin concentrations will be combined to calculate HOMA-IR (Homeostatic Model Assessment for Insulin Resistance).
Time frame: Baseline, 6, and 12 months
Change in lipid profile (triglyceride, cholesterol)
Blood test after overnight fasting
Time frame: Baseline, 6, and 12 months
Change in hyperphagia
Measured by the validated Hyperphagia Questionnaire for Clinical Trials (HQ-CT), specific for subjects with Prader-Willi syndrome (scale 0-36, higher values indicate higher degree of hiperphagia). Parental or caregiver reported.
Time frame: Baseline, 6, and 12 months
Change in Aberrant Behavior Checklist (ABC) score
The Aberrant Behavior Checklist (ABC) is a 58-item questionnaire. It consists of five subscales: hyperactivity (0-48), lethargy (0-48), stereotypical behavior (0-21), irritability (0-45), and inappropriate speech (0-12). Higher scores indicate worse outcome. Parental or caregiver reported.
Time frame: Baseline, 6, and 12 months
Change in Repetitive Behavior Scale (RBS) score
The Repetitive Behavior Scale (RBS) measures repetitive behaviors that are related to autism. It consists of 43-items grouped in two different scores: higher-order (ritualistic, sameness, compulsive and restricted subscales; score 0-87) and lower-order (stereotypy and self-injury; score 0-42) repetitive behaviors. Higher scores indicate worse outcome. Parental or caregiver reported.
Time frame: Baseline, 6, and 12 months
Change in Social Responsiveness Scale (SRS) score
Measured by the validated Social Responsiveness Scale (SRS-2) which assesses autism-related symptoms focusing on social function. Higher values indicate worse outcome. A total score of 76 or higher is considered severe; a score between 66 and 75 is considered moderate; a score between 60 and 65 is considered mild; a score of 59 or lower is considered within normal limits. Parental or caregiver reported.
Time frame: Baseline, 6, and 12 months
Change in Clinical Global Impression (GCI score) score
Measured by the validated Clinical Global Impression Scale (CGI, scale 0-14, higher values indicate worse outcome). This toll will be completed by the clinician.
Time frame: Baseline, 6, and 12 months
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