Probiotics as a Long-term Treatment Strategy for Prader-Willi Syndrome
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Fundació Sant Joan de Déu
- Enrollment
- 41
- Locations
- 2
- Primary Endpoint
- Change in percent body fat content
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 6 Years to 30 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
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
Outcomes
Primary Outcomes
Change in percent body fat content
Time Frame: 6 months
Body fat content will be measured by dual energy x-ray absorptiometry (DXA)
Secondary Outcomes
- Change in height (cm)(Baseline, 6, and 12 months)
- Change in insulin sensitivity(Baseline, 6, and 12 months)
- Change in Aberrant Behavior Checklist (ABC) score(Baseline, 6, and 12 months)
- Change in lipid profile (triglyceride, cholesterol)(Baseline, 6, and 12 months)
- Change in Clinical Global Impression (GCI score) score(Baseline, 6, and 12 months)
- Change in BMI and BMI z-score(Baseline, 6, and 12 months)
- Change in hyperphagia(Baseline, 6, and 12 months)
- Change in Social Responsiveness Scale (SRS) score(Baseline, 6, and 12 months)
- Change in Repetitive Behavior Scale (RBS) score(Baseline, 6, and 12 months)