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Tests treatment safety and results for Fragile X Syndrome (FXS)
Official title Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome
ClinicalTrials.gov ID: NCT07654114
What this study is testing
What is CTH120?
CTH120 is an investigational medicine, given as a twice-daily pill taken by mouth, being studied as a potential treatment for fragile x syndrome (fxs).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The purpose of this Phase IIa study is to evaluate the safety, tolerability, and effectiveness of CTH120 in adult males with Fragile X syndrome.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 45, men only
You may be able to join if
- Adult male participants.
- Aged ≥ 18 and ≤ 45 years.
- Weight ≥ 50 kg and ≤ 100 kg.
- Body mass index (BMI) ≥ 18.5 and ≤ 32.
- Clinical and molecular diagnosis of Fragile X syndrome (\> 200 CGG repeats in the promoter region of the FMR1 gene).
You likely can't join if
- Personal history of infantile spasms/convulsions/epilepsy, severe head trauma or CNS infections (e.g. meningitis), except for infantile febrile...
- Participants with a current diagnosis of severe (Level 3) autism spectrum disorder or any primary psychiatric diagnosis according to DSM-5...
- Substance use disorder according to the DSM-5 criteria.
- Epileptiform abnormalities on EEG (excluding isolated sharp waves and beyond those expected for age).
- Any life-threatening medical disease.
- Any other clinically relevant concomitant disease or condition or finding at screening that in the judgment of the investigator could interfere with...
See the full eligibility criteria
- Adult male participants.
- Aged ≥ 18 and ≤ 45 years.
- Weight ≥ 50 kg and ≤ 100 kg.
- Body mass index (BMI) ≥ 18.5 and ≤ 32.
- Clinical and molecular diagnosis of Fragile X syndrome (\> 200 CGG repeats in the promoter region of the FMR1 gene).
- Participants must have a parent, or other reliable caregiver, who agrees to accompany the participant to all study visits, provide information about the participant as required by the protocol, and ensure compliance...
- Legal representative understands and accepts the study procedures. If only one parent signs, he/she should confirm that the other parent does not object to the patient's participation in the study.
- Participant assenting and/or willing to participate.
- Signed informed consent by legal representative prior to any study-mandated procedure.
- Participant with a CGI-S score ≥ 3 evaluated by a clinician with experience on Fragile X syndrome, independently mobile and having sufficient vision and hearing to participate in study evaluations. They must be able to...
- Participants are expected to complete all procedures scheduled during the study visits.
- VCI scaled score \>4 on the WISC-V, based on mental age.
- Personal history of infantile spasms/convulsions/epilepsy, severe head trauma or CNS infections (e.g. meningitis), except for infantile febrile seizures.
- Participants with a current diagnosis of severe (Level 3) autism spectrum disorder or any primary psychiatric diagnosis according to DSM-5 (Diagnostic and Statistical Manual of Mental Disorders-DSM-5). Diagnoses that...
- Substance use disorder according to the DSM-5 criteria.
- Epileptiform abnormalities on EEG (excluding isolated sharp waves and beyond those expected for age).
- Any life-threatening medical disease.
- Any other clinically relevant concomitant disease or condition or finding at screening that in the judgment of the investigator could interfere with the treatment, the conduct of the study and related procedures and/or...
- Any clinically significant findings on physical examination including clinically significant vital sign abnormalities, from the perspective of the investigator.
- Any clinically significant laboratory or ECG abnormalities, from the perspective of the investigator, at Screening and/or prior to the initiation of the study medication.
- Known hypersensitivity or intolerance to any component of the investigational medicinal product or its excipients.
- Neuroleptic or antidepressant (SSRI) drugs within the 8 weeks prior to screening, except for sertraline at maximum 100 mg/day, and with no changes in the 8 weeks prior the initiation of the study.
- More than 3 psychotropic medications simultaneously in the 8 weeks prior to Screening and also during the study.
- Any new prescription or over the counter drug (except occasional use of paracetamol) in the last 2 weeks before Day 1.
- Participation in a clinical study with investigational treatments in the last 8 weeks prior to screening.
- Auditory or visual impairments that cannot be corrected.
- Positive EtG/EtS test in urine.
- Positive drug test in urine.
The study team makes the final eligibility decision.
Where it's taking place
- Barcelona, Barcelona, Spain
- Sabadell, Barcelona, Spain
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 18 years to 45 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Barcelona, Barcelona, Spain; Sabadell, Barcelona, Spain. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.