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Completed

NCT Number: NCT04685057

Probiotic Treatment for Prader-Willi Syndrome

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.

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Key information

Conditions

Prader-Willi Syndrome Abnormalities, Multiple Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Imprinting Disorders Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutrition Disorders Nutritional and Metabolic Diseases Obesity Overnutrition Overweight

Age range

6 year–30 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Hospital Sant Joan de Deu

Barcelona, 08950, Spain

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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
  • 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
  • Unsuitable for inclusion in the study in the opinion of the investigator

Treatment and study plan

Placebo

Dietary Supplement

Intervention with a daily dose of placebo for 6 months

Probiotic

Dietary Supplement

Intervention with a daily dose of probiotic for 6 months

Follow-up probiotic

Dietary Supplement

Intervention with a daily dose of probiotic for 6 months

Primary outcomes

  1. Change in percent body fat content

    Time frame: 6 months

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

Secondary outcomes

  1. Change in BMI and BMI z-score

    Time frame: Baseline, 6, and 12 months

    Weight and height will be combined to calculate changes in BMI and BMI z scores from all subjects will be assessed

  2. Change in height (cm)

    Time frame: Baseline, 6, and 12 months

    Changes in height from subjects that have not yet reached final height will be measured.

  3. Change in insulin sensitivity

    Time frame: Baseline, 6, and 12 months

    Fasting blood glucose and insulin concentrations will be combined to calculate HOMA-IR (Homeostatic Model Assessment for Insulin Resistance).

  4. Change in lipid profile (triglyceride, cholesterol)

    Time frame: Baseline, 6, and 12 months

    Blood test after overnight fasting

  5. Change in hyperphagia

    Time frame: Baseline, 6, and 12 months

    Measured by the validated Hyperphagia Questionnaire for Clinical Trials (HQ-CT), specific for subjects with Prader-Willi syndrome (scale 0-36, higher values indicate higher degree of hiperphagia). Parental or caregiver reported.

  6. Change in Aberrant Behavior Checklist (ABC) score

    Time frame: Baseline, 6, and 12 months

    The Aberrant Behavior Checklist (ABC) is a 58-item questionnaire. It consists of five subscales: hyperactivity (0-48), lethargy (0-48), stereotypical behavior (0-21), irritability (0-45), and inappropriate speech (0-12). Higher scores indicate worse outcome. Parental or caregiver reported.

  7. Change in Repetitive Behavior Scale (RBS) score

    Time frame: Baseline, 6, and 12 months

    The Repetitive Behavior Scale (RBS) measures repetitive behaviors that are related to autism. It consists of 43-items grouped in two different scores: higher-order (ritualistic, sameness, compulsive and restricted subscales; score 0-87) and lower-order (stereotypy and self-injury; score 0-42) repetitive behaviors. Higher scores indicate worse outcome. Parental or caregiver reported.

  8. Change in Social Responsiveness Scale (SRS) score

    Time frame: Baseline, 6, and 12 months

    Measured by the validated Social Responsiveness Scale (SRS-2) which assesses autism-related symptoms focusing on social function. Higher values indicate worse outcome. A total score of 76 or higher is considered severe; a score between 66 and 75 is considered moderate; a score between 60 and 65 is considered mild; a score of 59 or lower is considered within normal limits. Parental or caregiver reported.

  9. Change in Clinical Global Impression (GCI score) score

    Time frame: Baseline, 6, and 12 months

    Measured by the validated Clinical Global Impression Scale (CGI, scale 0-14, higher values indicate worse outcome). This toll will be completed by the clinician.

Other outcomes

  1. Change in intestinal microbiota composition

    Time frame: Baseline, 6, and 12 months

    DNA isolated from fecal samples will be analyzed by sequencing the hypervariable region of the bacterial 16S gene.

  2. Change in plasma metabolome

    Time frame: Baseline, 6, and 12 months

    Liquid chromatography coupled to mass spectrometry will be used to obtain a comprehensive metabolic profile of plasma samples

  3. Changes in brain structural anatomy

    Time frame: Baseline, 6, and 12 months

    Subjects older that 12 years of age will undergo brain magnetic resonance imaging (MRI). Changes in orbitofrontal cortex, amygdala, hypothalamus, and total gray matter volume will be evaluated.

Sponsors and collaborators

Lead sponsor

Fundació Sant Joan de Déu

Other

Registry information

Official study title

Probiotics as a Long-term Treatment Strategy for Prader-Willi Syndrome

Important dates

Study start
2021
Primary completion
2022
Study completion
2022
First posted
Dec 28, 2020
Registry last updated
Aug 25, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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