Probiotic Treatment for Prader-Willi Syndrome

Not Applicable

Completed

• Conditions: Prader-Willi Syndrome
• Interventions: Dietary Supplement: Placebo; Dietary Supplement: Probiotic; Dietary Supplement: Follow-up probiotic

• Registration Number: NCT04685057
• Lead Sponsor: Fundació Sant Joan de Déu

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-12-02
• Study First Submitted QC: 2020-12-22
• Study First Posted: 2020-12-28
• Study Start: 2021-01-11
• Status Verified: 2022-01
• Primary Completion: 2022-06-30
• Study Completion: 2022-06-30
• Last Update Submitted: 2022-08-24
• Last Update Posted: 2022-08-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 41

Inclusion Criteria

• Diagnosed with Prader-Willi Syndrome with genetic confirmation
• On a stable diet and medication regimen for at least the last two months before enrollment

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Exclusion Criteria

• Current enrollment in or discontinuation within the last 30 days from a clinical trial
• Patients with bariatric surgery in the last two years
• Patients with Type 2 Diabetes on insulin therapy
• Presence of other medical problems that would preclude study participation
• Unsuitable for inclusion in the study in the opinion of the investigator

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Follow-up probiotic	This arm will receive placebo for 6 months then probiotic for 6 more months.
Placebo	Placebo	This arm will receive placebo for 6 months then probiotic for 6 more months.
Probiotic	Probiotic	This arm will receive probiotic for 6 months then will keep receiving probiotic for 6 more months.
Probiotic	Follow-up probiotic	This arm will receive probiotic for 6 months then will keep receiving probiotic for 6 more months.

Primary Outcome Measures

Name	Time	Method
Change in percent body fat content	6 months	Body fat content will be measured by dual energy x-ray absorptiometry (DXA)

Secondary Outcome Measures

Name	Time	Method
Change in height (cm)	Baseline, 6, and 12 months	Changes in height from subjects that have not yet reached final height will be measured.
Change in insulin sensitivity	Baseline, 6, and 12 months	Fasting blood glucose and insulin concentrations will be combined to calculate HOMA-IR (Homeostatic Model Assessment for Insulin Resistance).
Change in Aberrant Behavior Checklist (ABC) score	Baseline, 6, and 12 months	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.
Change in lipid profile (triglyceride, cholesterol)	Baseline, 6, and 12 months	Blood test after overnight fasting
Change in Clinical Global Impression (GCI score) score	Baseline, 6, and 12 months	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.
Change in BMI and BMI z-score	Baseline, 6, and 12 months	Weight and height will be combined to calculate changes in BMI and BMI z scores from all subjects will be assessed
Change in hyperphagia	Baseline, 6, and 12 months	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.
Change in Social Responsiveness Scale (SRS) score	Baseline, 6, and 12 months	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.
Change in Repetitive Behavior Scale (RBS) score	Baseline, 6, and 12 months	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.

Trial Locations

Locations (1)

Hospital Sant Joan de Deu; 🇪🇸 Barcelona, Spain