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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | This arm will receive placebo for 6 months then probiotic for 6 more months. |
|
| Probiotic | Experimental | This arm will receive probiotic for 6 months then will keep receiving probiotic for 6 more months. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Placebo | Dietary Supplement | Intervention with a daily dose of placebo for 6 months |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Change in percent body fat content | Body fat content will be measured by dual energy x-ray absorptiometry (DXA) | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | Baseline, 6, and 12 months |
| Change in height (cm) |
| Measure | Description | Time Frame |
|---|---|---|
| Change in intestinal microbiota composition | DNA isolated from fecal samples will be analyzed by sequencing the hypervariable region of the bacterial 16S gene. | Baseline, 6, and 12 months |
| Change in plasma metabolome |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Marta Ramon-Krauel | Hospital Sant Joan de Deu | Principal Investigator |
| Carles Lerin, PhD | Fundació Sant Joan de Déu | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hospital Sant Joan de Deu | Barcelona | 08950 | Spain |
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| ID | Term |
|---|---|
| D011218 | Prader-Willi Syndrome |
| ID | Term |
|---|---|
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| D019936 | Probiotics |
| ID | Term |
|---|---|
| D019587 | Dietary Supplements |
| D005502 | Food |
| D000066888 | Diet, Food, and Nutrition |
| D010829 | Physiological Phenomena |
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| Probiotic |
| Dietary Supplement |
Intervention with a daily dose of probiotic for 6 months |
|
| Follow-up probiotic | Dietary Supplement | Intervention with a daily dose of probiotic for 6 months |
|
Changes in height from subjects that have not yet reached final height will be measured.
| 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). | Baseline, 6, and 12 months |
| Change in lipid profile (triglyceride, cholesterol) | Blood test after overnight fasting | 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. | 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. | 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. | 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. | 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. | Baseline, 6, and 12 months |
Liquid chromatography coupled to mass spectrometry will be used to obtain a comprehensive metabolic profile of plasma samples
| Baseline, 6, and 12 months |
| Changes in brain structural anatomy | Subjects older that 12 years of age will undergo brain magnetic resonance imaging (MRI). Changes in orbitofrontal cortex, amygdala, hypothalamus, and total gray matter volume will be evaluated. | Baseline, 6, and 12 months |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| D000096803 | Imprinting Disorders |
| D009765 | Obesity |
| D050177 | Overweight |
| D044343 | Overnutrition |
| D009748 | Nutrition Disorders |
| D009750 | Nutritional and Metabolic Diseases |
| D019602 |
| Food and Beverages |