Targeting the Gut Microbiome for Prader-Willi Syndrome Treatment

NCT03548480 | Completed

• 1 other identifier: FSJD-BALPWS-2018
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 1

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.

Conditions

Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (1)

• Change in percent body fat

  Measured by DXA scan

  3 months

Secondary Outcomes (5)

• Change in lipid profile (triglyceride, cholesterol)

  3 months

• Change in glucose metabolic parameters (glucose, insulin, HbA1c)

  3 months

• Change in circulating cytokine levels

  3 months

• Change in hyperphagia

  3 months

• Change in thermoregulation

  3 months

Other Outcomes (3)

• Change in plasma metabolome

  3 months

• Change in urine metabolome

  3 months

• Change in intestinal microbiome

  3 months

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Dietary Supplement: Placebo

Probiotic

EXPERIMENTAL

Dietary Supplement: Probiotic

Interventions

Placebo DIETARY_SUPPLEMENT

Intervention with a daily dose of placebo

Placebo

Probiotic DIETARY_SUPPLEMENT

Intervention with a daily dose of probiotic

Probiotic

Eligibility Criteria

• Age: 2 Years - 19 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosed with Prader-Willi Syndrome with genetic confirmation
• On a stable diet and medication regimen for at least the last two months before enrollment

You may not qualify if:

• 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

Sponsors & Collaborators

• Fundació Sant Joan de Déu: lead

Study Sites (1)

Hospital Sant Joan de Deu

Barcelona, 08950, Spain

Location

Related Publications (1)

• Amat-Bou M, Garcia-Ribera S, Climent E, Piquer-Garcia I, Corripio R, Sanchez-Infantes D, Villalta L, Elias M, Jimenez-Chillaron JC, Chenoll E, Ramon D, Ibanez L, Ramon-Krauel M, Lerin C. Effects of Bifidobacterium animalis Subsp. lactis (BPL1) Supplementation in Children and Adolescents with Prader-Willi Syndrome: A Randomized Crossover Trial. Nutrients. 2020 Oct 13;12(10):3123. doi: 10.3390/nu12103123.

  PMID: 33066107 DERIVED

MeSH Terms

Conditions

Prader-Willi Syndrome

Interventions

Probiotics

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Dietary Supplements; Food; Diet, Food, and Nutrition; Physiological Phenomena; Food and Beverages

Study Officials

• Carles Lerin, PhD

  Fundació Sant Joan de Déu

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 2, 2018
• First Posted: June 7, 2018
• Study Start: January 1, 2018
• Primary Completion: January 31, 2019
• Study Completion: January 31, 2019
• Last Updated: March 5, 2020

Record last verified: 2020-03

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1)