Probiotic Intervention for Microbiome Modifications and Clinical Improvements in Fragile X Syndrome

Launched by SPECILA HOSPITAL FOR CEREBRAL PALSY AND DEVELOPMENTAL NEUROLOGY · Feb 22, 2024

Keywords

ClinConnect Summary

This clinical trial is looking at how a mixture of probiotics—helpful bacteria found in some foods and supplements—can affect children with Fragile X Syndrome, a genetic condition that can impact behavior and learning. Specifically, researchers want to see if these probiotics can change the gut microbiome (the balance of bacteria in the digestive system) and how these changes might improve behaviors and brain functions in kids aged 3 to 18 years. The study will last for three months, during which participants will receive the probiotic mixture and attend three visits to a clinic in Serbia for assessments.

To participate, children must have a confirmed diagnosis of Fragile X Syndrome and be between the ages of 3 and 18. They should also have a parent or caregiver who can commit to attending all study visits and completing necessary questionnaires. The study is currently recruiting up to 15 participants, and during the trial, researchers will measure various aspects of behavior and brain activity to see if the probiotics make a difference. It's important for families to know that this study is open-label, meaning everyone knows they are receiving the probiotics, and there will be no placebo involved.

Gender

ALL

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.