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
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Sponsor:
Fundació Sant Joan de Déu
Information provided by (Responsible Party):
Fundació Sant Joan de Déu
Tracking Information | |||||||
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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 |
Change in percent body fat [ Time Frame: 3 months ] Measured by DXA scan
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Original Primary Outcome Measures ICMJE | Same as current | ||||||
Change History | |||||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE | Same as current | ||||||
Current Other Pre-specified Outcome Measures |
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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 |
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Condition ICMJE | Prader-Willi Syndrome | ||||||
Intervention ICMJE |
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Study Arms ICMJE |
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Publications * | 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. | ||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||
Recruitment Status ICMJE | Completed | ||||||
Actual Enrollment ICMJE |
39 | ||||||
Original Estimated Enrollment ICMJE |
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:
Exclusion Criteria:
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Sex/Gender ICMJE |
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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 |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Fundació Sant Joan de Déu | ||||||
Original Responsible Party | Same as current | ||||||
Current Study Sponsor ICMJE | Fundació Sant Joan de Déu | ||||||
Original Study Sponsor ICMJE | Same as current | ||||||
Collaborators ICMJE | Not Provided | ||||||
Investigators ICMJE |
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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 |