CTH120: 15 participants will be randomized to receive CTH120 75 mg hard capsules. Capsules of CTH120 will be administered by the oral route to participants twice a day, one capsule in the morning and one capsule in the evening, at approximately the same times each day, after breakfast (morning dose) and after dinner (evening dose), with a glass of water. The evening dose will be taken approximately 12 h from the morning dose, and preferably before midnight. The evening dose must not be taken before at least 8 hours from the morning dose.
CTH120 placebo hard capsules: 15 participants will be randomized to receive CTH120 placebo hard capsules. Capsules of CTH120 matching placebo will be administered by the oral route to participants twice a day, one capsule in the morning and one capsule in the evening, at approximately the same times each day, after breakfast (morning dose) and after dinner (evening dose), with a glass of water. The evening dose will be taken approximately 12 h from the morning dose, and preferably before midnight. The evening dose must not be taken before at least 8 hours from the morning dose.
Study summary
The purpose of this Phase IIa study is to evaluate the safety, tolerability, and effectiveness of CTH120 in adult males with Fragile X syndrome.
Eligibility
Sex
MALE
Min age
18 Years
Max age
45 Years
Healthy volunteers
No
Inclusion Criteria:
1. Adult male participants.
2. Aged ≥ 18 and ≤ 45 years.
3. Weight ≥ 50 kg and ≤ 100 kg.
4. Body mass index (BMI) ≥ 18.5 and ≤ 32.
5. Clinical and molecular diagnosis of Fragile X syndrome (\> 200 CGG repeats in the promoter region of the FMR1 gene).
6. 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 with study tests.
7. 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.
8. Participant assenting and/or willing to participate.
9. Signed informed consent by legal representative prior to any study-mandated procedure.
10. 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 be understood most of the time and must not depend upon other forms of communication, signs, symbol boards or devices as their primary form of communication.
11. Participants are expected to complete all procedures scheduled during the study visits.
12. VCI scaled score \>4 on the WISC-V, based on mental age.
Exclusion Criteria:
1. Personal history of infantile spasms/convulsions/epilepsy, severe head trauma or CNS infections (e.g. meningitis), except for infantile febrile seizures.
2. 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 are secondary, such as attention deficit hyperactivity disorder, depressive disorders, anxiety disorders and conduct disorders are allowed if they are considered to not interfere with study conduct and are stable during the 8 weeks prior to screening. Related allowed treatments must be on stable dosing for the last 3 months.
3. Substance use disorder according to the DSM-5 criteria.
4. Epileptiform abnormalities on EEG (excluding isolated sharp waves and beyond those expected for age).
5. Any life-threatening medical disease.
6. 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 might bias the study results interpretation or could jeopardize the participant's safety.
7. Any clinically significant findings on physical examination including clinically significant vital sign abnormalities, from the perspective of the investigator.
8. 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.
9. Known hypersensitivity or intolerance to any component of the investigational medicinal product or its excipients.
10. 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.
11. More than 3 psychotropic medications simultaneously in the 8 weeks prior to Screening and also during the study.
12. Any new prescription or over the counter drug (except occasional use of paracetamol) in the last 2 weeks before Day 1.
13. Participation in a clinical study with investigational treatments in the last 8 weeks prior to screening.
14. Auditory or visual impairments that cannot be corrected.
15. Positive EtG/EtS test in urine.
16. Positive drug test in urine.
Primary outcome measure(s)
Treatment-emergent adverse events (TEAEs). — From Day 1 to End-of-study: EOS will be on Day 56 (±2 days). Primary Safety and Tolerability endpoint for FXS-CTH120-01. AEs will be described in terms of result, frequency, intensity, medical decision, relation with study drug, as well as treatment received and subject retirement, duration, and time elapsed. AEs will also be listed and coded using the MedDRA Dictionary for the term's codification.
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in blood pressure (mmHg). — From Day 1 to End-of-study (EOS): on Day 56 (±2 days). Primary Safety and Tolerability endpoint for FXS-CTH120-01. Blood pressure (mmHg) will be measured in the supine position following a 5 min rest.
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in pulse rate (bpm). — From Day 1 to End-of-study (EOS): on Day 56 (±2 days). Primary Safety and Tolerability endpoint for FXS-CTH120-01. Pulse rate (bpm) will be measured in the supine position following a 5 min rest.
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in body temperature (ºC). — From Day 1 to End-of-study (EOS): on Day 56 (±2 days). Primary Safety and Tolerability endpoint for FXS-CTH120-01. Body temperature will be measured using an automated vital sign monitor device.
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: heart rate (bpm). — From Day 1 to End-of-study (EOS): on Day 56 (±2 days). Primary Safety and Tolerability endpoint for FXS-CTH120-01. Triplicate 12-lead ECGs will be recorded in a supine position after at least 10-minute rest. Each lead will be recorded for at least 3 beats at a speed of 25 mm/s.CGs will be recorded using a machine that automatically calculates the following parameter: heart rate (bpm).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: rhythm. — From Day 1 to End-of-study (EOS): on Day 56 (±2 days). Primary Safety and Tolerability endpoint for FXS-CTH120-01. Triplicate 12-lead ECGs will be recorded in a supine position after at least 10-minute rest. Each lead will be recorded for at least 3 beats at a speed of 25 mm/s.CGs will be recorded using a machine that automatically calculates the following parameter: rhythm.
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: PQ/PR interval (ms). — From Day 1 to End-of-study (EOS): on Day 56 (±2 days). Primary Safety and Tolerability endpoint for FXS-CTH120-01. Triplicate 12-lead ECGs will be recorded in a supine position after at least 10-minute rest. Each lead will be recorded for at least 3 beats at a speed of 25 mm/s. ECGs will be recorded using a machine that automatically calculates the following parameter: PQ/PR interval (ms).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: QRS duration (ms). — From Day 1 to End-of-study (EOS): on Day 56 (±2 days). Primary Safety and Tolerability endpoint for FXS-CTH120-01. Triplicate 12-lead ECGs will be recorded in a supine position after at least 10-minute rest. Each lead will be recorded for at least 3 beats at a speed of 25 mm/s. ECGs will be recorded using a machine that automatically calculates the following parameter: QRS duration (ms).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: QT (ms). — From Day 1 to End-of-study (EOS): on Day 56 (±2 days). Primary Safety and Tolerability endpoint for FXS-CTH120-01. Triplicate 12-lead ECGs will be recorded in a supine position after at least 10-minute rest. Each lead will be recorded for at least 3 beats at a speed of 25 mm/s. ECGs will be recorded using a machine that automatically calculates the following parameter: QT (ms).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: QTcF (ms). — From Day 1 to End-of-study (EOS): on Day 56 (±2 days). Primary Safety and Tolerability endpoint for FXS-CTH120-01. Triplicate 12-lead ECGs will be recorded in a supine position after at least 10-minute rest. Each lead will be recorded for at least 3 beats at a speed of 25 mm/s. ECGs will be recorded using a machine that automatically calculates the following parameter: QTcF (ms).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: haematology. — Screening visit (From Day -28 to Day -1), on Day 15, on Day 28, on Day 42 and Day 56 (± 2 days) (End-of-study (EOS)). Primary Safety and Tolerability endpoint for FXS-CTH120-01. The following tests will be performed: • Haematology: haemoglobin, haematocrit, red blood cell count, mean corpuscular volume, mean corpuscular haemoglobin, white blood cell count (absolute and %), platelets.
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: serum chemistry. — Screening visit (From Day -28 to Day -1), on Day 15, on Day 28, on Day 42 and Day 56 (± 2 days) (End-of-study (EOS)). Primary Safety and Tolerability endpoint for FXS-CTH120-01. The following tests will be performed: • Serum chemistry: sodium, potassium, urea, creatinine, albumin, calcium, phosphate, glucose, total cholesterol, LDL, HDL, TG, ALT, AST, GGT, total bilirubin, CPK, LDH.
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: coagulation. — Screening visit (From Day -28 to Day -1), on Day 15, on Day 28, on Day 42 and Day 56 (± 2 days) (End-of-study (EOS)). Primary Safety and Tolerability endpoint for FXS-CTH120-01. The following tests will be performed: • Coagulation parameters: INR, aPTT, PT.
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: urinalysis. — Screening visit (from Day -28 to Day -1). Primary Safety and Tolerability endpoint for FXS-CTH120-01. The following tests will be performed: • Urinalysis parameters: leucocytes, protein, bilirubin, urobilinogen, ketones, red blood cells, pH, nitrite, glucose (only at Screening visit).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: viral serology and detection. — Screening visit (from day -28 to Day -1). Primary Safety and Tolerability endpoint for FXS-CTH120-01. The following tests will be performed: • Viral serology and detection: Hepatitis B (HBsAg), Hepatitis C (Ac IgG VHC) and HIV antibody.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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