BALPWS: Targeting the Gut Microbiome for Prader-Willi Syndrome Treatment

Sponsor

Fundació Sant Joan de Déu (Other)

Overall Status

Completed

CT.gov ID

NCT03548480

Collaborator

(none)

Enrollment

Location

Arms

Actual Duration (Months)

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The gut microbiome has recently emerged as a major contributor to obesity, systemic inflammation, and metabolic disease. Furthermore, intestinal bacteria are crucial players in the gut-brain axis, regulating a broad range of central nervous system processes, from satiety mechanisms to anxiety and social behavior. Thus, targeting the microbiome is being actively investigated as a therapeutic strategy for a wide array of diseases, including obesity, anxiety, depression, and autism. Among all intestinal bacteria, Bifidobacterium animalis spp. lactis (BAL) has shown promise for obesity treatment in experimental animal models and human subjects, improving body composition and metabolic health, and reducing energy intake. Moreover, tryptophan metabolism, a crucial regulator of satiety mechanisms and anxiety, is a main target of BAL. Given that clinical manifestations of Prader-Willi syndrome (PWS) include hyperphagia, anxiety, altered body composition, and metabolic dysregulation, the aforementioned effects of BAL might prove highly beneficial for children with PWS. Here, the investigators will test this hypothesis by performing a randomized double-blinded placebo-controlled crossover clinical study to assess the effects of BAL supplementation on an array of clinical manifestations of PWS. Children with PWS will undergo a 3-month placebo/probiotic treatment period, a 3-month washout period, followed by a 3-month probiotic/placebo supplementation. Anthropometric, biochemical, and psychological data as well as biological samples will be obtained at the beginning of the study, and after each of the study periods, with a total of four time-points. Specifically, the investigators will determine body composition by DXA analysis; metabolic health by assessing glucose and lipid metabolic parameters as well as circulating hormonal and cytokine levels; thermoregulation by non-invasive thermal imaging; and hyperphagia and emotional and behavioral problems by applying parental-rated validated questionnaires.

Condition or Disease	Intervention/Treatment	Phase
Prader-Willi Syndrome	Dietary Supplement: Placebo Dietary Supplement: Probiotic	N/A

Study Design

Study Type:

Interventional

Actual Enrollment :

39 participants

Allocation:

Randomized

Intervention Model:

Crossover Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official Title:

Targeting the Gut Microbiome for Prader-Willi Syndrome Treatment

Actual Study Start Date :

Jan 1, 2018

Actual Primary Completion Date :

Jan 31, 2019

Actual Study Completion Date :

Jan 31, 2019

Arms and Interventions

Arm	Intervention/Treatment
Placebo Comparator: Placebo	Dietary Supplement: Placebo Intervention with a daily dose of placebo
Experimental: Probiotic	Dietary Supplement: Probiotic Intervention with a daily dose of probiotic

Arm

Intervention/Treatment

Placebo Comparator: Placebo

Dietary Supplement: Placebo

Intervention with a daily dose of placebo

Experimental: Probiotic

Dietary Supplement: Probiotic

Intervention with a daily dose of probiotic

Outcome Measures

Primary Outcome Measures

Change in percent body fat [3 months]
Measured by DXA scan

Secondary Outcome Measures

Change in lipid profile (triglyceride, cholesterol) [3 months]
Blood test after overnight fasting
Change in glucose metabolic parameters (glucose, insulin, HbA1c) [3 months]
Blood test after overnight fasting
Change in circulating cytokine levels [3 months]
Quantified in plasma samples
Change in hyperphagia [3 months]
Measured by validated questionnaire (HQ-CT)
Change in thermoregulation [3 months]
Measured by thermal imaging

Other Outcome Measures

Change in plasma metabolome [3 months]
Liquid chromatography coupled to mass spectrometry will be used to obtain a comprehensive metabolic profile of plasma samples
Change in urine metabolome [3 months]
Liquid chromatography coupled to mass spectrometry will be used to obtain a comprehensive metabolic profile of urine samples
Change in intestinal microbiome [3 months]
DNA isolated from fecal samples will be analyzed by sequencing.

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years to 19 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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
Presence of other medical problems that would preclude study participation
Patients with a history of bariatric surgery
Unsuitable for inclusion in the study in the opinion of the investigator

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Hospital Sant Joan de Deu	Barcelona		Spain	08950

Sponsors and Collaborators

Fundació Sant Joan de Déu

Investigators

Principal Investigator: Carles Lerin, PhD, Fundació Sant Joan de Déu

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Fundació Sant Joan de Déu

ClinicalTrials.gov Identifier:

NCT03548480

Other Study ID Numbers:

FSJD-BALPWS-2018

First Posted:

Jun 7, 2018

Last Update Posted:

Mar 5, 2020

Last Verified:

Mar 1, 2020

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Product Manufactured in and Exported from the U.S.:

Additional relevant MeSH terms:

Prader-Willi Syndrome

Syndrome

Study Results

No Results Posted as of Mar 5, 2020