Targeting the Gut Microbiome for Prader-Willi Syndrome Treatment

Prader-Willi Syndrome Clinical Trial

— BALPWS  

Official title:

Targeting the Gut Microbiome for Prader-Willi Syndrome Treatment

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03548480
• Other study ID #: FSJD-BALPWS-2018
• Status: Completed
• Phase: N/A
• Start date: January 1, 2018
• Est. completion date: January 31, 2019

Study information

• Verified date: March 2020
• Source: Fundació Sant Joan de Déu
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 39
• Est. completion date: January 31, 2019
• Est. primary completion date: January 31, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 19 Years

Eligibility

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

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Intervention

Dietary Supplement:
• Placebo
Intervention with a daily dose of placebo
• Probiotic
Intervention with a daily dose of probiotic

Locations

Country	Name	City	State
Spain	Hospital Sant Joan de Deu	Barcelona

Sponsors (1)

Lead Sponsor	Collaborator
Fundació Sant Joan de Déu

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Change in plasma metabolome	Liquid chromatography coupled to mass spectrometry will be used to obtain a comprehensive metabolic profile of plasma samples	3 months
Other	Change in urine metabolome	Liquid chromatography coupled to mass spectrometry will be used to obtain a comprehensive metabolic profile of urine samples	3 months
Other	Change in intestinal microbiome	DNA isolated from fecal samples will be analyzed by sequencing.	3 months
Primary	Change in percent body fat	Measured by DXA scan	3 months
Secondary	Change in lipid profile (triglyceride, cholesterol)	Blood test after overnight fasting	3 months
Secondary	Change in glucose metabolic parameters (glucose, insulin, HbA1c)	Blood test after overnight fasting	3 months
Secondary	Change in circulating cytokine levels	Quantified in plasma samples	3 months
Secondary	Change in hyperphagia	Measured by validated questionnaire (HQ-CT)	3 months
Secondary	Change in thermoregulation	Measured by thermal imaging	3 months