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Targeting the Gut Microbiome for Prader-Willi Syndrome Treatment (BALPWS)

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ClinicalTrials.gov Identifier: NCT03548480
Recruitment Status : Completed
First Posted : June 7, 2018
Last Update Posted : March 5, 2020
Sponsor:
Information provided by (Responsible Party):
Fundació Sant Joan de Déu

Study Description
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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 Not Applicable

Study Design
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Study Type : Interventional  (Clinical Trial)
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 : January 1, 2018
Actual Primary Completion Date : January 31, 2019
Actual Study Completion Date : January 31, 2019

Resource links provided by the National Library of Medicine


Arms and Interventions
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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
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Primary Outcome Measures :
  1. Change in percent body fat [ Time Frame: 3 months ]
    Measured by DXA scan


Secondary Outcome Measures :
  1. Change in lipid profile (triglyceride, cholesterol) [ Time Frame: 3 months ]
    Blood test after overnight fasting

  2. Change in glucose metabolic parameters (glucose, insulin, HbA1c) [ Time Frame: 3 months ]
    Blood test after overnight fasting

  3. Change in circulating cytokine levels [ Time Frame: 3 months ]
    Quantified in plasma samples

  4. Change in hyperphagia [ Time Frame: 3 months ]
    Measured by validated questionnaire (HQ-CT)

  5. Change in thermoregulation [ Time Frame: 3 months ]
    Measured by thermal imaging


Other Outcome Measures:
  1. Change in plasma metabolome [ Time Frame: 3 months ]
    Liquid chromatography coupled to mass spectrometry will be used to obtain a comprehensive metabolic profile of plasma samples

  2. Change in urine metabolome [ Time Frame: 3 months ]
    Liquid chromatography coupled to mass spectrometry will be used to obtain a comprehensive metabolic profile of urine samples

  3. Change in intestinal microbiome [ Time Frame: 3 months ]
    DNA isolated from fecal samples will be analyzed by sequencing.


Eligibility Criteria
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Ages Eligible for Study:   2 Years to 19 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  • 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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03548480


Locations
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Spain
Hospital Sant Joan de Deu
Barcelona, Spain, 08950
Sponsors and Collaborators
Fundació Sant Joan de Déu
Investigators
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Principal Investigator: Carles Lerin, PhD Fundació Sant Joan de Déu
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Fundació Sant Joan de Déu
ClinicalTrials.gov Identifier: NCT03548480    
Other Study ID Numbers: FSJD-BALPWS-2018
First Posted: June 7, 2018    Key Record Dates
Last Update Posted: March 5, 2020
Last Verified: March 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Prader-Willi Syndrome
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
 Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders