Probiotic Treatment for Prader-Willi Syndrome

NCT04685057 | Completed

• 1 other identifier: PIC-194-20
• Study Type: interventional
• Target: 41
• Locations: 1 country
• Sites: 1

Brief Summary

A whole new research area studying the function of intestinal microorganisms, also known as gut microbiota, has emerged during the last decade. As a result, dietary supplementation with specific bacteria (or probiotics) holds great promise as a therapeutic strategy for a wide range of diseases, from obesity to anxiety and depression, all of which are major characteristics of Prader-Willi syndrome (PWS). The main objective of the current proposal is to determine the effects of Bifidobacterium animalis subsp. lactis (strain BPL1) supplementation in children and young adults with PWS. Specifically, participants will receive placebo or BPL1 for 6 months, and then this phase will be followed by a 6-month extension period in which all participants will receive BPL1. This study will allow us to 1) determine the effects on fat mass and glucose metabolism; and 2) explore the effects on mental health symptoms by studying potential structural changes in the brain by magnetic resonance imaging (MRI) as well as using a number of psychiatric questionnaires.

Conditions

Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (1)

• Change in percent body fat content

  Body fat content will be measured by dual energy x-ray absorptiometry (DXA)

  6 months

Secondary Outcomes (9)

• Change in BMI and BMI z-score

  Baseline, 6, and 12 months

• Change in height (cm)

  Baseline, 6, and 12 months

• Change in insulin sensitivity

  Baseline, 6, and 12 months

• Change in lipid profile (triglyceride, cholesterol)

  Baseline, 6, and 12 months

• Change in hyperphagia

  Baseline, 6, and 12 months

Other Outcomes (3)

• Change in intestinal microbiota composition

  Baseline, 6, and 12 months

• Change in plasma metabolome

  Baseline, 6, and 12 months

• Changes in brain structural anatomy

  Baseline, 6, and 12 months

Study Arms (2)

Placebo

PLACEBO COMPARATOR

This arm will receive placebo for 6 months then probiotic for 6 more months.

Dietary Supplement: Placebo; Dietary Supplement: Follow-up probiotic

Probiotic

EXPERIMENTAL

This arm will receive probiotic for 6 months then will keep receiving probiotic for 6 more months.

Dietary Supplement: Probiotic; Dietary Supplement: Follow-up probiotic

Interventions

Placebo DIETARY_SUPPLEMENT

Intervention with a daily dose of placebo for 6 months

Placebo

Probiotic DIETARY_SUPPLEMENT

Intervention with a daily dose of probiotic for 6 months

Probiotic

Follow-up probiotic DIETARY_SUPPLEMENT

Intervention with a daily dose of probiotic for 6 months

Placebo; Probiotic

Eligibility Criteria

• Age: 6 Years - 30 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
• Patients with bariatric surgery in the last two years
• Patients with Type 2 Diabetes on insulin therapy
• Presence of other medical problems that would preclude study participation

Sponsors & Collaborators

• Fundació Sant Joan de Déu: lead

Study Sites (1)

Hospital Sant Joan de Deu

Barcelona, 08950, Spain

Location

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

• Marta Ramon-Krauel

  Hospital Sant Joan de Deu

  PRINCIPAL INVESTIGATOR

• 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: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 2, 2020
• First Posted: December 28, 2020
• Study Start: January 11, 2021
• Primary Completion: June 30, 2022
• Study Completion: June 30, 2022
• Last Updated: August 25, 2022

Record last verified: 2022-01

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1)