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NCT Number: NCT07654114

Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome

The purpose of this Phase IIa study is to evaluate the safety, tolerability, and effectiveness of CTH120 in adult males with Fragile X syndrome.

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Key information

Conditions

Fragile X Syndrome (FXS) Central Nervous System Diseases Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disease Attributes Fragile X Syndrome Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Intellectual Disability Mental Disorders Nervous System Diseases Neurobehavioral Manifestations Neurodevelopmental Disorders Neurologic Manifestations Pathologic Processes Pathological Conditions, Signs and Symptoms Rare Diseases Sex Chromosome Disorders Signs and Symptoms X-Linked Intellectual Disability

Age range

18 year–45 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

Hospital del Mar Medical Research Institute, Barcelona, Spain

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About this study

A total of 30 randomized adult participants with Fragile X syndrome will participate in the clinical trial (randomization ratio 1:1 treated vs placebo arms). The expected distribution between sites will be 1:1.

This trial will consist of a Screening period of up to 28 days prior to treatment period. A final follow-up period for safety of 14 days (± 2 days) is planned after the end of treatment. The duration of the study will be approximately 3 months for each participant.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion 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 with study tests.
  • 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 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.
  • Participants are expected to complete all procedures scheduled during the study visits.
  • VCI scaled score >4 on the WISC-V, based on mental age.

Exclusion criteria

  • 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 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.
  • 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 might bias the study results interpretation or could jeopardize the participant's safety.
  • 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.

Treatment and study plan

CTH120

Drug

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

Other

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.

Primary outcomes

  1. Treatment-emergent adverse events (TEAEs).

    Time frame: 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.

  2. Treatment-emergent potentially clinically significant abnormalities (PSCAs) in blood pressure (mmHg).

    Time frame: 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.

  3. Treatment-emergent potentially clinically significant abnormalities (PSCAs) in pulse rate (bpm).

    Time frame: 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.

  4. Treatment-emergent potentially clinically significant abnormalities (PSCAs) in body temperature (ºC).

    Time frame: 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.

  5. Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: heart rate (bpm).

    Time frame: 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).

  6. Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: rhythm.

    Time frame: 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.

  7. Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: PQ/PR interval (ms).

    Time frame: 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).

  8. Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: QRS duration (ms).

    Time frame: 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).

  9. Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: QT (ms).

    Time frame: 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).

  10. Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: QTcF (ms).

    Time frame: 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).

  11. Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: haematology.

    Time frame: 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.

  12. Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: serum chemistry.

    Time frame: 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.

  13. Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: coagulation.

    Time frame: 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.

  14. Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: urinalysis.

    Time frame: 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).

  15. Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: viral serology and detection.

    Time frame: 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.

Secondary outcomes

  1. Observed maximum concentration (Cmax).

    Time frame: On Day 15 and Day 42.

    Secondary Pharmacokinetics endpoint for CTH120 and its main metabolite for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model: Cmax (ng/mL).

  2. Measured concentration at the end of a dosing interval at steady state (Ctrough).

    Time frame: On Day 15, Day 28 and Day 42.

    Secondary Pharmacokinetics endpoint for CTH120 and its main metabolite for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model: Ctrough (ng/mL).

  3. Last analytically quantifiable plasma concentration above LLOQ (Clast).

    Time frame: On Day 15 and Day 42.

    Secondary Pharmacokinetics endpoint CTH120 and its main metabolite for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model: Clast (ng/mL).

  4. Time to reach Cmax (tmax).

    Time frame: On Day 15 and Day 42.

    Secondary Pharmacokinetics endpoint for CTH120 and its main metabolite for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model: tmax (h).

  5. Lag-time (time delay between drug administration and first observed concentration above LOQ in plasma) (tlag).

    Time frame: On Day 15 and Day 42.

    Secondary Pharmacokinetics endpoint for CTH120 and its main metabolite for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model: tlag (h).

  6. Time post administration of the last analytically quantifiable concentration (above LLOQ) (tlast).

    Time frame: On Day 15 and Day 42.

    Secondary Pharmacokinetics endpoint for CTH120 and its main metabolite for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model: tlast (h).

  7. Area under the plasma concentration-time curve (AUC0-t, AUC0-∞, AUCextrap %)

    Time frame: On Day 15 and Day 42.

    Secondary Pharmacokinetics endpoint for CTH120 and its main metabolite for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model: AUC0-t (h * ng/mL), AUC0-∞ (h * ng/mL), AUCextrap % (h * ng/mL).

  8. Elimination rate constant (λz).

    Time frame: On Day 15 and Day 42.

    Secondary Pharmacokinetics endpoint for CTH120 and its main metabolite for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model: λz (1/h).

  9. Terminal half-life (t1/2).

    Time frame: On Day 15 and Day 42.

    Secondary Pharmacokinetics endpoint for CTH120 and its main metabolite for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model: t1/2 (h).

  10. Apparent clearance (CL/F).

    Time frame: On Day 15 and Day 42.

    Secondary Pharmacokinetics endpoint for CTH120 and its main metabolite for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model:CL/F (mL/h * kg).

  11. Apparent volume of distribution during terminal phase after oral administration: (Vz/F).

    Time frame: On Day 15 and Day 42.

    Secondary Pharmacokinetics endpoint for CTH120 for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model: Vz/F (mL/kg).

  12. Accumulation ratio calculated from Cmax after repeated dosing and Cmax after 1st dosing (RAC Cmax).

    Time frame: On Day 42.

    Secondary Pharmacokinetics endpoint for CTH120 and its main metabolite for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model: RAC Cmax.

  13. Accumulation ratio calculated from AUC0-t after repeated dosing and AUC0-t (RAC AUC0-t).

    Time frame: On Day 42.

    Secondary Pharmacokinetics endpoint for CTH120 and its main metabolite for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model: RAC AUC0-t.

  14. Metabolic ratio of metabolite Cmax and parent drug Cmax (MR Cmax).

    Time frame: On Day 15 and Day 42.

    Secondary Pharmacokinetics endpoint for CTH120's main metabolite for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model: MR Cmax (n-fold).

  15. Metabolic ratio of metabolite AUC0-t and parent drug AUC0-t (MR AUC0-t).

    Time frame: On Day 15 and Day 42.

    Secondary Pharmacokinetics endpoint for CTH120's main metabolite for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model: MR AUC0-t (n-fold).

  16. Metabolic ratio of metabolite AUC0-∞ and parent drug AUC0-∞ (MR AUC0-∞).

    Time frame: On Day 15 and Day 42.

    Secondary Pharmacokinetics endpoint for CTH120's main metabolite for FXS-CTH120-01.

    • Plasma PK parameter calculated using a non-compartmental model: MR AUC0-∞ (n-fold).

  17. Global functioning using the Visual Analogue Scale (VAS)

    Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, on Day 42 and on Day 56 (± 2 days) (End-of-study (EOS)).

    Secondary exploratory Efficacy endpoints for FXS-CTH120-01:

    • The VAS consists of a 100 mm horizontal line anchored at each end with descriptors representing opposite extremes of a behavioural or functional domain (Left-end representing the lowest or most impaired level, and the right-end representing the highest or least impaired level). The VAS will be used to evaluate the clinician's perception of the participant's current condition across five specific domains relevant to Fragile X syndrome by placing a mark along the line at the position that best reflects their clinical judgment of the individual's status. This mark is then measured from the left end of the line (0 mm) to the mark (up to 100 mm) to produce a numeric score.

  18. Global severity using the Clinical Global Impression Severity Scale (CGI-S)

    Time frame: On Baseline visit (From Day -14 to Day -1) and on Day 14.

    Secondary exploratory Efficacy endpoints for FXS-CTH120-01. CGI-S establishes the baseline illness status and rates illness severity on a 7-point scale, with anchors ranging from "1 = Normal, not at all sick" to "7 = Among the most extremely sick patients".

  19. Global improvement using the Clinical Global Impression Improvement Scale (CGI- I)

    Time frame: On Day 14, on Day 42 and on Day 56 (± 2 days) (End-of-study (EOS)).

    Secondary exploratory Efficacy endpoints for FXS-CTH120-01. CGI-I rates how much the participant's illness has improved or worsened relative to the baseline state (CGI-S) on a similar 7-point scale: 1 = very much improved; 2 = much improved; 3 = minimally improved; 4 = no change; 5 = minimally worse; 6 = much worse; and 7 = very much worse.

  20. Behaviour troubles using the Aberrant Behaviour Checklist-Community in FXS (ABC-CFXS)

    Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, on Day 42 and on Day 56 (± 2 days) (End-of-study (EOS)).

    Secondary exploratory Efficacy endpoints for FXS-CTH120-01:

    The ABC-CFXS is an adapted version of the Aberrant Behaviour Checklist - Community (ABC-C), specifically tailored for individuals with Fragile X syndrome (FXS). It includes 55 items rated on a 4-point Likert scale ranging from 0 ("not at all a problem") to 3 ("the problem is severe in degree"), assessing six behavioural domains: irritability, hyperactivity, lethargy, social avoidance, stereotypy, and inappropriate speech.

  21. Cognitive functioning using the National Institutes of Health Toolbox Cognition Battery Intellectual disability NIH-TCB-ID

    Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.

    Secondary exploratory Efficacy endpoints for FXS-CTH120-01:

    The Fluid Cognition composite score of the NIH Toolbox cognitive battery combines the scores of five tests assessing the following cognitive domains: Cognitive flexibility, Inhibitory control and visual attention, Episodic memory, Processing speed and Working memory. Higher composite scores indicate better cognitive functioning, whereas lower scores indicate greater cognitive impairment.

  22. Anxiety, Depression, and Mood Scale (ADAMS).

    Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.

    Exploratory Efficacy endpoints for FXS-CTH120-01:

    ADAMS is a scale to assess 28 items, categorized into the following five subscales: Manic/Hyperactive Behaviour, Depressed Mood, Social Avoidance, General Anxiety and Compulsive Behaviour. Each item is evaluated on a 4-point Likert scale rating from 0 (not a problem) to 3 (severe problem). The scores for the 5 subscales will be collected in the eCRF.

  23. Adaptive functioning using the Vineland Adaptive Behaviour Scale 3 (VABS-3)

    Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.

    Exploratory Efficacy endpoints for FXS-CTH120-01:

    Vineland Adaptive Behavior Scales, Third Edition (VABS-3) is a psychometric instrument that assesses adaptive functioning through a standardized interview with the participant or their caregiver. It evaluates activities of daily living across the domains of Communication, Daily Living Skills, and Socialization. Outcomes include raw scores, V-scale scores, Growth Scale Values (GSVs), domain standard scores, and the Adaptive Behavior Composite (ABC).

    The Adaptive Behavior Composite (ABC) standard score ranges from 20 to 123. Higher scores indicate better adaptive functioning, whereas lower scores indicate greater adaptative functioning impairment.

  24. Paediatric Quality of Life Inventory (PedsQL) 2.0

    Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.

    Exploratory Efficacy endpoints for FXS-CTH120-01:

    PedsQL evaluates quality of life involving physical, psychological, social, and cognitive aspects.

    Quality of life using the PedsQL 2.0 Family Impact Module (parent impact for all ages). Items are rated on a 5-point scale from 0 (never a problem) to 4 (almost always a problem). Items are reversed scored and linearly transformed on a scale ranging from 0 to 100. Higher scores indicate better health-related quality of life.

  25. Paediatric Quality of Life Inventory (PedsQL) 3.0

    Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.

    Exploratory Efficacy endpoints for FXS-CTH120-01:

    PedsQL evaluates quality of life involving physical, psychological, social, and cognitive aspects.

    Cognitive functioning using the PedsQL 3.0 Cognitive Functioning Scale (PARENT Reports for Young Adults (ages 18-25) and Adults (ages over 26)). Items are rated on a 5-point scale from 0 (never a problem) to 4 (almost always a problem). Items are reversed scored and linearly transformed on a scale ranging from 0 to 100. Higher scores indicate better health-related quality of life.

  26. Paediatric Quality of Life Inventory (PedsQL) 4.0

    Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.

    Exploratory Efficacy endpoints for FXS-CTH120-01:

    PedsQL evaluates quality of life involving physical, psychological, social, and cognitive aspects.

    Quality of life using the PedsQL 4.0 Generic Core Scales (PARENT Reports for Young Adults (ages 18-25) and Adults (ages over 26). Items are rated on a 5-point scale from 0 (never a problem) to 4 (almost always a problem). Items are reversed scored and linearly transformed on a scale ranging from 0 to 100. Higher scores indicate better health-related quality of life.

  27. Quality of sleep using the Pittsburgh Sleep Quality Index (PSQI)

    Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.

    Exploratory Efficacy endpoints for FXS-CTH120-01:

    Pittsburgh sleep quality index (PSQI) is a self-report questionnaire that assesses sleep quality and quantity. The 19-item self-report questionnaire yields 7 component scores: subjective sleep quality, sleep latency, duration, habitual sleep efficiency, sleep disturbances, use of sleeping medication, and daytime dysfunction. There are five additional questions that are completed by a bed partner if there is one. The sum of the 7 individual scores (from 0 to 3), ranges from 0 to 21. A total score of less or equal than 5 is associated with good sleep quality. A total score of more than 5 is associated with poor sleep quality.

  28. Neural functioning using Electroencephalography (EEG) (auditory oddball, resting-state and auditory steady-state response)

    Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.

    Exploratory Efficacy endpoints for FXS-CTH120-01:

    EEG will be recorded using a mobile wireless helmet for high-precision EEG monitoring (Neuroelectrics Enobio® 20 5G, Starlab technology, Spain).

  29. Social avoidance using eye-tracking

    Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.

    Exploratory Efficacy endpoints for FXS-CTH120-01:

    An eye tracker (Tobii Technology, Sweden) will be used to record X and Y coordinates of eye position and pupil diameter while participants are exposed to stimuli consisting on coloured photographs of adult human faces.

  30. Biorhythm characteristics using Actigraphy

    Time frame: Between Baseline visit (From Day -14 to Day -1) and Day 14, and between Day 28 and Day 42.

    Exploratory Efficacy endpoints for FXS-CTH120-01:

    Sleep-wake rhythms will be assessed using an actigraph (GT3X-BT model) placed on the wrist of a non-dominant hand. The actigraph is a non-invasive device, useful for monitoring circadian rhythms and it records consecutive periods of 60 seconds throughout the 24 hours/day.

    The minimum actigraphy recording will be at least 7 days.

  31. Protein levels of FMRP in peripheral blood

    Time frame: On Baseline visit (From Day -14 to Day -1) and on Day 42.

    Exploratory Efficacy endpoints for FXS-CTH120-01:

    Venous blood samples (9 mL each) will be obtained by extraction of peripheral blood from participants in a EDTA tube.

  32. BDNF concentrations in plasma

    Time frame: On Baseline visit (From Day -14 to Day -1) and on Day 42.

    Exploratory Efficacy endpoints for FXS-CTH120-01:

    Venous blood samples (6 mL each) will be obtained by extraction from a cubital vein from participants. A single venipuncture will be used to obtain three aliquots.

  33. miRNA profile in plasma

    Time frame: On Baseline visit (From Day -14 to Day -1) and on Day 42.

    Exploratory Efficacy endpoints for FXS-CTH120-01:

    Approximately, 200 µL of human plasma obtained from blood samples collected for BDNF measurement will be processed to extract RNA enriched in small RNAs. RNA quality and concentration will be assessed using the prior library preparation. Libraries will be sequenced at CRG using Nextseq500 sequencer. Processed reads will be mapped to miRBase database using STAR software, and count tables will be generated with FeatureCounts (Subread package). For differential expression (DE) analysis, read counts will be transformed to log2-counts-per-million (logCPM), and variance will be modelled using the voom approach in the limma package.

Study contacts

Contact information is provided by the study sponsor or research team.

Josep Prous, PhD

CONTACT

[email protected]

(+34) 934034509

Sponsors and collaborators

Lead sponsor

Connecta Therapeutics, S.L.

Industry

Collaborators

  • Astrum CRO, S.L.
  • Corporacion Parc Tauli
  • Hospital del Mar Research Institute (IMIM)
  • Ministry of Science and Innovation, Spain

Registry information

Official study title

Evaluation of the Safety, Tolerability, and Effectiveness of CTH120 in Adult Males With Fragile X Syndrome

Acronym: FXS-CTH120-01

Important dates

Study start
2026
Primary completion
2027
Study completion
2027
First posted
Jun 17, 2026
Registry last updated
Jul 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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