Phase 2 Rare Disease Trial, Recruiting

Written by an AI model from the ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• You must be a male adult.
• You must be between 18 and 45 years old.
• You must weigh between 50 kg (about 110 lbs) and 100 kg (about 220 lbs).
• Your Body Mass Index (BMI) must be between 18.5 and 32 (a measure of body weight relative to height).
• You must have a confirmed diagnosis of Fragile X syndrome, proven through both clinical assessment and a genetic test showing more than 200 CGG repeats in the FMR1 gene.
• You must have a parent or reliable caregiver who agrees to attend all study visits with you, provide information about you as needed, and help ensure you follow study requirements.
• A legal representative must understand and agree to the study procedures, and if only one parent signs, they must confirm the other parent does not object to your participation.
• You must be willing to participate and agree to take part.
• A legal representative must sign a consent form before any study procedures begin.
• A clinician experienced in Fragile X syndrome must rate your condition at a certain level of severity (CGI-S score of 3 or higher); you must also be able to move around independently, have adequate vision and hearing, be understandable most of the time, and use spoken language as your main way of communicating.
• You must be expected to attend and complete all scheduled study visits and procedures.
• You must score above 4 on a specific thinking and reasoning test (VCI scaled score on the WISC-V), based on your mental age (confirm with trial site).

Who may not be able to join:

• You have a history of infantile spasms, seizures, or epilepsy, serious head injury, or infections of the brain or nervous system (such as meningitis) — though a history of simple childhood febrile (fever-related) seizures may be acceptable.
• You currently have a severe form of autism spectrum disorder (Level 3), or a primary psychiatric condition as defined by medical diagnostic guidelines (DSM-5); some secondary conditions like ADHD, depression, anxiety, or conduct disorders may be allowed if they are stable and not expected to interfere with the study (confirm with trial site).
• You have a current diagnosis of substance use disorder according to DSM-5 guidelines.
• A brain wave test (EEG) shows certain abnormal patterns beyond what would normally be expected for your age.
• You have any life-threatening medical condition.
• You have any other significant health condition or finding that, in the doctor's opinion, could interfere with the study or put your safety at risk.
• You have any significant findings on a physical examination, including abnormal vital signs, as judged by the doctor.
• You have any significant abnormal results on blood tests or a heart tracing (ECG) at the screening stage or before starting the study medication.
• You have a known allergy or sensitivity to any ingredient in the study medication.
• You have taken certain antipsychotic or antidepressant medications (including SSRIs) within the 8 weeks before screening — though sertraline at a maximum dose of 100 mg/day with no changes in the 8 weeks before the study starts may be acceptable (confirm with trial site).
• You are currently taking more than 3 medications that affect the mind or mood at the same time, or have been in the 8 weeks before screening.
• You have started taking any new prescription or over-the-counter medication (other than occasional use of paracetamol/acetaminophen) in the 2 weeks before the study starts.
• You have taken part in another clinical trial using an experimental treatment within the 8 weeks before screening.
• You have hearing or vision problems that cannot be corrected with glasses, hearing aids, or similar devices.
• A urine test shows the presence of alcohol (EtG/EtS markers).
• A urine test shows the presence of recreational drugs.

Important: Always verify eligibility with the trial site directly before applying.