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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03548480
Recruitment Status : Enrolling by invitation
First Posted : June 7, 2018
Last Update Posted : June 7, 2018
Information provided by (Responsible Party):
Fundació Sant Joan de Déu

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 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
Estimated Primary Completion Date : June 2019
Estimated Study Completion Date : July 2019

Resource links provided by the National Library of Medicine

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

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.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   2 Years to 19 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

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 identifier (NCT number): NCT03548480

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Hospital Sant Joan de Deu
Barcelona, Spain, 08950
Sponsors and Collaborators
Fundació Sant Joan de Déu
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Principal Investigator: Carles Lerin, PhD Fundació Sant Joan de Déu

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Responsible Party: Fundació Sant Joan de Déu Identifier: NCT03548480     History of Changes
Other Study ID Numbers: FSJD-BALPWS-2018
First Posted: June 7, 2018    Key Record Dates
Last Update Posted: June 7, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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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
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Nutrition Disorders