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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.
 
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

Tracking Information
First Submitted Date  ICMJE May 2, 2018
First Posted Date  ICMJE June 7, 2018
Last Update Posted Date March 5, 2020
Actual Study Start Date  ICMJE January 1, 2018
Actual Primary Completion Date January 31, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 6, 2018)
Change in percent body fat [ Time Frame: 3 months ]
Measured by DXA scan
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 6, 2018)
  • Change in lipid profile (triglyceride, cholesterol) [ Time Frame: 3 months ]
    Blood test after overnight fasting
  • Change in glucose metabolic parameters (glucose, insulin, HbA1c) [ Time Frame: 3 months ]
    Blood test after overnight fasting
  • Change in circulating cytokine levels [ Time Frame: 3 months ]
    Quantified in plasma samples
  • Change in hyperphagia [ Time Frame: 3 months ]
    Measured by validated questionnaire (HQ-CT)
  • Change in thermoregulation [ Time Frame: 3 months ]
    Measured by thermal imaging
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: June 6, 2018)
  • 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
  • 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
  • Change in intestinal microbiome [ Time Frame: 3 months ]
    DNA isolated from fecal samples will be analyzed by sequencing.
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Targeting the Gut Microbiome for Prader-Willi Syndrome Treatment
Official Title  ICMJE Targeting the Gut Microbiome for Prader-Willi Syndrome Treatment
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Prader-Willi Syndrome
Intervention  ICMJE
  • Dietary Supplement: Placebo
    Intervention with a daily dose of placebo
  • Dietary Supplement: Probiotic
    Intervention with a daily dose of probiotic
Study Arms  ICMJE
  • Placebo Comparator: Placebo
    Intervention: Dietary Supplement: Placebo
  • Experimental: Probiotic
    Intervention: Dietary Supplement: Probiotic
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 4, 2020)
39
Original Estimated Enrollment  ICMJE
 (submitted: June 6, 2018)
40
Actual Study Completion Date  ICMJE January 31, 2019
Actual Primary Completion Date January 31, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 19 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Spain
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03548480
Other Study ID Numbers  ICMJE FSJD-BALPWS-2018
Has Data Monitoring Committee No
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Fundació Sant Joan de Déu
Study Sponsor  ICMJE Fundació Sant Joan de Déu
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Carles Lerin, PhD Fundació Sant Joan de Déu
PRS Account Fundació Sant Joan de Déu
Verification Date March 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP