Targeting the Gut Microbiome for Prader-Willi Syndrome Treatment

Status Completed

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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT03548480
Study type	Interventional
Source	Fundació Sant Joan de Déu
Contact
Status	Completed
Phase	N/A
Start date	January 1, 2018
Completion date	January 31, 2019

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT05032326` - Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial	Phase 3
Completed	`NCT04526379` - Study of Emotion and Cognition Abilities of Children With PWS and Proposition of an Innovative Remediation	N/A
Terminated	`NCT03458416` - A Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Participants With Prader-Willi Syndrome	Phase 2
Completed	`NCT03718416` - Natural History Study of Serious Medical Events in PWS
Completed	`NCT05322096` - Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome	Phase 2
Terminated	`NCT02179151` - Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Beloranib) in Obese Subjects With Prader-Willi Syndrome	Phase 3
Completed	`NCT02205450` - Growth Hormone in Children Under 2 Years With Prader-Willi in Hospital of Sabadell
Completed	`NCT00375089` - Characteristics of Prader-Willi Syndrome and Early-onset Morbid Obesity	N/A
Completed	`NCT00004351` - Study of Phenotype and Genotype Correlations in Patients With Contiguous Gene Deletion Syndromes	N/A
Recruiting	`NCT05938543` - Cerebellar TMS and Satiety in Prader-Willi Syndrome	N/A
Suspended	`NCT05879614` - An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)	Phase 2
Recruiting	`NCT03031626` - Oxygen Versus Medical Air for Treatment of CSA in Prader Will Syndrome	Phase 4
Withdrawn	`NCT04086810` - An Open-Label Study of DCCR Tablet in Patients With PWS	Phase 3
Completed	`NCT02629991` - Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome	Phase 2
Recruiting	`NCT02297022` - Deep Brain Stimulation for the Treatment of Obesity in Patients With Prader-Willi Syndrome	Phase 1
Not yet recruiting	`NCT02263781` - PREPL in Health and Disease	N/A
Completed	`NCT00551343` - Gut Derived Hormones, Body Composition and Metabolism in Prader-Willi Syndrome	N/A
Recruiting	`NCT06448871` - Ultrasound to Assess Sarcopenia in Prader Willi Syndrome
Enrolling by invitation	`NCT03655223` - Early Check: Expanded Screening in Newborns
Recruiting	`NCT05939453` - Impact of Bright Light Therapy on Prader-Willi Syndrome	N/A

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

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

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms