Probiotic treatment for children with Fragile X Syndrome

Probiotic Intervention for Microbiome Modifications and Consequential Clinical Improvements in Children With Fragile X Syndrome: Pilot Study

This study is testing if a special mix of probiotics can help improve behavior and brain function in children with Fragile X Syndrome.

Quick facts

• Phase: NA
• Study type: Interventional
• Enrollment: 15 (estimated)
• Ages: 3 Years to 18 Years
• Sex: All
• Sponsor: Specila hospital for cerebral palsy and developmental neurology (other)
• Locations: 1 site (Belgrade)
• Trial ID: NCT06279858 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the efficacy of a probiotic mixture containing Lactobacillus casei, Lactobacillus salivarius, and Bifidobacterium breve in children aged 3-18 years with Fragile X Syndrome. The study aims to assess the relationship between microbiome modifications induced by the probiotics and improvements in behavioral manifestations and brain processing, using tools like eye tracking and EEG analysis. Participants will receive the probiotic for a duration of 12 weeks, and the trial will include 15 children over a one-year period, with a focus on understanding which patients may benefit from this treatment.

Who should consider this trial

Good fit: Ideal candidates are children aged 3-18 years with a confirmed diagnosis of Fragile X Syndrome.

Not a fit: Patients who do not have Fragile X Syndrome or those who cannot participate due to language barriers or unstable behavioral treatments may not benefit from this study.

Why it matters

Potential benefit: If successful, this intervention could lead to significant improvements in behavioral and cognitive outcomes for children with Fragile X Syndrome.

How similar studies have performed: While the gut-brain axis is a growing area of research, this specific probiotic approach in Fragile X Syndrome is relatively novel and has not been extensively tested in prior studies.

Eligibility criteria

Inclusion Criteria:

1. Subject has Fragile X syndrome with a molecular genetic confirmation of the full FMR1 mutation or mosaicism.
2. Subject is a male or female age 3 to 18 years.
3. Subject must have a parent or caretaker who is willing to participate in the whole study.
4. Subject and caregiver are able to attend the clinic regularly and reliably.
5. Subject and/or subject's parent/legal authorized representative is able to understand, read, write and speak Serbian fluently to complete study-related materials.
6. Behavioral and other non-pharmacological treatments/interventions must be stable for 4 weeks before screening and must remain stable during the period between screening and the commencement of study probiotic, and throughout the study. Minor changes in hours or times of therapy that are not considered clinically significant will not be exclusionary. Changes in therapies provided through a school program, due to school vacations, are allowed.
7. The use of concomitant medications must be stable, in terms of dose and dosing regimen, for at least 4 weeks prior to Screening and must remain stable during the period between Screening and the commencement of the study; every effort should be made to maintain stable regimens of allowed concomitant medications from the time of commencement of double-blind study medication until the last study assessment.
8. Patient's parent(s), legal authorized guardian(s), or consistent caregiver(s) can understand and sign an informed consent form to participate in the study. For subjects who are not their own legal guardian, subject's parent/legal authorized representative is able to understand and sign an informed consent to participate in the study.
9. Subject and/or subject's parent/legal authorized representative is able to understand, read, write, and speak Serbian fluently to complete study-related materials.

Exclusion Criteria:

1. Families that are not cooperative and will not follow through with the demands of this study;
2. Antibiotic use in the last two months (not counting topical antibiotics);
3. Currently taking antibiotics;
4. Any changes in medications, nutritional supplements, therapies, in the last two months, or any plans to change them during the first 3 months of probiotic treatment;
5. Diagnosis of severe gastrointestinal diseases, such as Crohn's Disease, or Ulcerative Colitis;
6. Subject has a life-threatening medical problem or other major systemic illness that compromises health or safety and/or would interfere with this study;
7. Age younger than 3 or older than 18 years.

Where this trial is running

Belgrade

• Special Hospital for Cerebral Palsy and Developmental Neurology — Belgrade, Serbia (RECRUITING)

Study contacts

• Principal investigator: Dragana Protic, Prof — University od Belgrade
• Study coordinator: Dragana Protic, Prof.

Conditions: Fragile X Syndrome, Fragile X Sindrome, Gut-Brain Axis, Probiotics