Prader-Willi SyndromeNCT04685057
Probiotic Treatment for Prader-Willi Syndrome
A study testing Probiotic and Follow-up probiotic in people with Prader-Willi Syndrome, run by Fundació Sant Joan de Déu at 1 site in Spain.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 to 30 years
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- Probiotic and Follow-up probiotic
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 41 participants
- Where
- 1 siteSpain
- Study dates
- 11 Jan 2021to 30 Jun 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosed with Prader-Willi Syndrome with genetic confirmation
- On a stable diet and medication regimen for at least the last two months before enrollment
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Probiotics as a Long-term Treatment Strategy 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.
- Conditions
- Prader-Willi Syndrome
- Being tested
- Placebo, Probiotic, Follow-up probiotic
- Medical subject headings
- Prader-Willi Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDietary supplement
Intervention with a daily dose of placebo for 6 months
ProbioticDietary supplement
Intervention with a daily dose of probiotic for 6 months
Follow-up probioticDietary supplement
Intervention with a daily dose of probiotic for 6 months
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | This arm will receive placebo for 6 months then probiotic for 6 more months. Receives: 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. Receives: Dietary Supplement: Probiotic; Dietary Supplement: Follow-up probiotic |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in percent body fat content
Body fat content will be measured by dual energy x-ray absorptiometry (DXA)
Measured: 6 months
Secondary outcomes (9)
Change in BMI and BMI z-score
Weight and height will be combined to calculate changes in BMI and BMI z scores from all subjects will be assessed
Measured: Baseline, 6, and 12 months
Change in height (cm)
Changes in height from subjects that have not yet reached final height will be measured.
Measured: Baseline, 6, and 12 months
Change in insulin sensitivity
Fasting blood glucose and insulin concentrations will be combined to calculate HOMA-IR (Homeostatic Model Assessment for Insulin Resistance).
Measured: Baseline, 6, and 12 months
Change in lipid profile (triglyceride, cholesterol)
Blood test after overnight fasting
Measured: Baseline, 6, and 12 months
Change in hyperphagia
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.
Measured: Baseline, 6, and 12 months
Change in Aberrant Behavior Checklist (ABC) score
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.
Measured: Baseline, 6, and 12 months
Change in Repetitive Behavior Scale (RBS) score
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.
Measured: Baseline, 6, and 12 months
Change in Social Responsiveness Scale (SRS) score
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.
Measured: Baseline, 6, and 12 months
Change in Clinical Global Impression (GCI score) score
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.
Measured: Baseline, 6, and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Dec 2020
- Study start11 Jan 2021
- Primary completion30 Jun 2022
- Study completion30 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fundació Sant Joan de Déu
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Marta Ramon-Krauel, Principal investigator, Hospital Sant Joan de DeuCarles Lerin, PhD, Principal investigator, Fundació Sant Joan de Déu
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04685057
- Sponsor's protocol ID
- PIC-194-20
- Status last verified
- Jan 2022
- First submitted
- 2 Dec 2020
- Last updated
- 25 Aug 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Probiotics as a Long-term Treatment Strategy for Prader-Willi Syndrome
- ClinicalTrials.gov ID
- NCT04685057
- Acronym / sponsor ID
- PIC-194-20
- Conditions
- Prader-Willi Syndrome
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Fundació Sant Joan de DéuUniversity, hospital or other
- Study start
- 11 Jan 2021 (actual)
- Primary completion
- 30 Jun 2022 (actual)
- Study completion
- 30 Jun 2022 (actual)
- First posted
- 28 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Dietary Supplement: Placebo, Dietary Supplement: Follow-up probioticThis arm will receive placebo for 6 months then probiotic for 6 more months. |
| Probiotic | Experimental | Dietary Supplement: Probiotic, Dietary Supplement: Follow-up probioticThis arm will receive probiotic for 6 months then will keep receiving probiotic for 6 more months. |
Who may qualify
- Ages
- 6 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Diagnosed with Prader-Willi Syndrome with genetic confirmation
- On a stable diet and medication regimen for at least the last two months before enrollment
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PlaceboIntervention with a daily dose of placebo for 6 months | Dietary supplement | Placebo |
| ProbioticIntervention with a daily dose of probiotic for 6 months | Dietary supplement | Probiotic |
| Follow-up probioticIntervention with a daily dose of probiotic for 6 months | Dietary supplement | Placebo, Probiotic |
Primary outcome
Change in percent body fat content
Time frame: 6 months
Body fat content will be measured by dual energy x-ray absorptiometry (DXA)
9 secondary outcomes
Change in BMI and BMI z-score
Time frame: Baseline, 6, and 12 months
Change in height (cm)
Time frame: Baseline, 6, and 12 months
Change in insulin sensitivity
Time frame: Baseline, 6, and 12 months
Change in lipid profile (triglyceride, cholesterol)
Time frame: Baseline, 6, and 12 months
Change in hyperphagia
Time frame: Baseline, 6, and 12 months
Change in Aberrant Behavior Checklist (ABC) score
Time frame: Baseline, 6, and 12 months
Change in Repetitive Behavior Scale (RBS) score
Time frame: Baseline, 6, and 12 months
Change in Social Responsiveness Scale (SRS) score
Time frame: Baseline, 6, and 12 months
Change in Clinical Global Impression (GCI score) score
Time frame: Baseline, 6, and 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Aug 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Marta Ramon-Krauel principal investigator
Hospital Sant Joan de Deu
Carles Lerin, PhD principal investigator
Fundació Sant Joan de Déu
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Probiotic and Follow-up probiotic in people with Prader-Willi Syndrome, run by Fundació Sant Joan de Déu at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in percent body fat content (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Aug 2022; AzTrials last refreshed its copy on 2 Nov 2026.