Clinical trial NCT04685057: Probiotic Treatment for Prader-Willi Syndrome
Official title: Probiotics as a Long-term Treatment Strategy for Prader-Willi Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Prader-Willi Syndrome
Lead sponsor: Fundació Sant Joan de Déu
Sponsor class: OTHER
First posted: 2020-12-28
Enrollment: 41
Sex: ALL
Minimum age (years): 6
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Change in percent body fat content
timeFrame: 6 months
Secondary outcomes:
measure: Change in BMI and BMI z-score
timeFrame: Baseline, 6, and 12 months
measure: Change in height (cm)
timeFrame: Baseline, 6, and 12 months
measure: Change in insulin sensitivity
timeFrame: Baseline, 6, and 12 months
measure: Change in lipid profile (triglyceride, cholesterol)
timeFrame: Baseline, 6, and 12 months
measure: Change in hyperphagia
timeFrame: Baseline, 6, and 12 months
measure: Change in Aberrant Behavior Checklist (ABC) score
timeFrame: Baseline, 6, and 12 months
measure: Change in Repetitive Behavior Scale (RBS) score
timeFrame: Baseline, 6, and 12 months
measure: Change in Social Responsiveness Scale (SRS) score
timeFrame: Baseline, 6, and 12 months
measure: Change in Clinical Global Impression (GCI score) score
timeFrame: Baseline, 6, and 12 months
Other outcomes:
measure: Change in intestinal microbiota composition
timeFrame: Baseline, 6, and 12 months
measure: Change in plasma metabolome
timeFrame: Baseline, 6, and 12 months
measure: Changes in brain structural anatomy
timeFrame: Baseline, 6, and 12 months
Locations:
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Deu
Study officials:
affiliation: Hospital Sant Joan de Deu
name: Marta Ramon-Krauel
role: PRINCIPAL_INVESTIGATOR
affiliation: Fundació Sant Joan de Déu
name: Carles Lerin, PhD
role: PRINCIPAL_INVESTIGATOR
References: