Ignite Creation Date:
2025-12-24 @ 7:02 PM
Ignite Modification Date:
2025-12-31 @ 2:48 AM
Study NCT ID:
NCT04685057
Status:
COMPLETED
Last Update Posted:
2022-08-25
First Post:
2020-12-02
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Probiotic Treatment for Prader-Willi Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D011218', 'term': 'Prader-Willi Syndrome'}], 'ancestors': [{'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D025063', 'term': 'Chromosome Disorders'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D000096803', 'term': 'Imprinting Disorders'}, {'id': 'D009765', 'term': 'Obesity'}, {'id': 'D050177', 'term': 'Overweight'}, {'id': 'D044343', 'term': 'Overnutrition'}, {'id': 'D009748', 'term': 'Nutrition Disorders'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D019936', 'term': 'Probiotics'}], 'ancestors': [{'id': 'D019587', 'term': 'Dietary Supplements'}, {'id': 'D005502', 'term': 'Food'}, {'id': 'D000066888', 'term': 'Diet, Food, and Nutrition'}, {'id': 'D010829', 'term': 'Physiological Phenomena'}, {'id': 'D019602', 'term': 'Food and Beverages'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 41}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2021-01-11', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-01', 'completionDateStruct': {'date': '2022-06-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-08-24', 'studyFirstSubmitDate': '2020-12-02', 'studyFirstSubmitQcDate': '2020-12-22', 'lastUpdatePostDateStruct': {'date': '2022-08-25', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-12-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-06-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Change in intestinal microbiota composition', 'timeFrame': 'Baseline, 6, and 12 months', 'description': 'DNA isolated from fecal samples will be analyzed by sequencing the hypervariable region of the bacterial 16S gene.'}, {'measure': 'Change in plasma metabolome', 'timeFrame': 'Baseline, 6, and 12 months', 'description': 'Liquid chromatography coupled to mass spectrometry will be used to obtain a comprehensive metabolic profile of plasma samples'}, {'measure': 'Changes in brain structural anatomy', 'timeFrame': 'Baseline, 6, and 12 months', 'description': '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.'}], 'primaryOutcomes': [{'measure': 'Change in percent body fat content', 'timeFrame': '6 months', 'description': 'Body fat content will be measured by dual energy x-ray absorptiometry (DXA)'}], 'secondaryOutcomes': [{'measure': 'Change in BMI and BMI z-score', 'timeFrame': 'Baseline, 6, and 12 months', 'description': 'Weight and height will be combined to calculate changes in BMI and BMI z scores from all subjects will be assessed'}, {'measure': 'Change in height (cm)', 'timeFrame': 'Baseline, 6, and 12 months', 'description': 'Changes in height from subjects that have not yet reached final height will be measured.'}, {'measure': 'Change in insulin sensitivity', 'timeFrame': 'Baseline, 6, and 12 months', 'description': 'Fasting blood glucose and insulin concentrations will be combined to calculate HOMA-IR (Homeostatic Model Assessment for Insulin Resistance).'}, {'measure': 'Change in lipid profile (triglyceride, cholesterol)', 'timeFrame': 'Baseline, 6, and 12 months', 'description': 'Blood test after overnight fasting'}, {'measure': 'Change in hyperphagia', 'timeFrame': 'Baseline, 6, and 12 months', 'description': '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.'}, {'measure': 'Change in Aberrant Behavior Checklist (ABC) score', 'timeFrame': 'Baseline, 6, and 12 months', 'description': '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.'}, {'measure': 'Change in Repetitive Behavior Scale (RBS) score', 'timeFrame': 'Baseline, 6, and 12 months', 'description': '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.'}, {'measure': 'Change in Social Responsiveness Scale (SRS) score', 'timeFrame': 'Baseline, 6, and 12 months', 'description': '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.'}, {'measure': 'Change in Clinical Global Impression (GCI score) score', 'timeFrame': 'Baseline, 6, and 12 months', 'description': '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.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Prader-Willi Syndrome']}, 'descriptionModule': {'briefSummary': '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).\n\nThe 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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '30 Years', 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosed with Prader-Willi Syndrome with genetic confirmation\n* On a stable diet and medication regimen for at least the last two months before enrollment\n\nExclusion Criteria:\n\n* Current enrollment in or discontinuation within the last 30 days from a clinical trial\n* Patients with bariatric surgery in the last two years\n* Patients with Type 2 Diabetes on insulin therapy\n* Presence of other medical problems that would preclude study participation\n* Unsuitable for inclusion in the study in the opinion of the investigator'}, 'identificationModule': {'nctId': 'NCT04685057', 'briefTitle': 'Probiotic Treatment for Prader-Willi Syndrome', 'organization': {'class': 'OTHER', 'fullName': 'Fundació Sant Joan de Déu'}, 'officialTitle': 'Probiotics as a Long-term Treatment Strategy for Prader-Willi Syndrome', 'orgStudyIdInfo': {'id': 'PIC-194-20'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'This arm will receive placebo for 6 months then probiotic for 6 more months.', 'interventionNames': ['Dietary Supplement: Placebo', 'Dietary Supplement: Follow-up probiotic']}, {'type': 'EXPERIMENTAL', 'label': 'Probiotic', 'description': 'This arm will receive probiotic for 6 months then will keep receiving probiotic for 6 more months.', 'interventionNames': ['Dietary Supplement: Probiotic', 'Dietary Supplement: Follow-up probiotic']}], 'interventions': [{'name': 'Placebo', 'type': 'DIETARY_SUPPLEMENT', 'description': 'Intervention with a daily dose of placebo for 6 months', 'armGroupLabels': ['Placebo']}, {'name': 'Probiotic', 'type': 'DIETARY_SUPPLEMENT', 'description': 'Intervention with a daily dose of probiotic for 6 months', 'armGroupLabels': ['Probiotic']}, {'name': 'Follow-up probiotic', 'type': 'DIETARY_SUPPLEMENT', 'description': 'Intervention with a daily dose of probiotic for 6 months', 'armGroupLabels': ['Placebo', 'Probiotic']}]}, 'contactsLocationsModule': {'locations': [{'zip': '08950', 'city': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Sant Joan de Deu', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}], 'overallOfficials': [{'name': 'Marta Ramon-Krauel', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Hospital Sant Joan de Deu'}, {'name': 'Carles Lerin, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Fundació Sant Joan de Déu'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Fundació Sant Joan de Déu', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}