A Phase 2 interventional study of CTH120 and CTH120 placebo hard capsules in Fragile X Syndrome (FXS) and Fragile X Syndrome, sponsored by Connecta Therapeutics, S.L.. Recruiting at 2 sites in Spain. Open to male participants aged 18 Years to 45 Years. Per ClinicalTrials.gov, last updated 2026-07-17.
Sponsored by Connecta Therapeutics, S.L. · Phase 2, Interventional, and Treatment
The purpose of this Phase IIa study is to evaluate the safety, tolerability, and effectiveness of CTH120 in adult males with Fragile X syndrome.
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.
114 studies on the registry are indexed under Fragile X Syndrome; 24 are open to participants now.
This study's planned enrollment of 30 is below the median of 50 across 91 interventional studies indexed under Fragile X Syndrome.
Browse Fragile X Syndrome studies →Connecta Therapeutics, S.L. is the lead sponsor of 3 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The American Psychiatric Association’s manual of mental health diagnoses. DSM-5 (2013, revised as DSM-5-TR in 2022) replaced DSM-IV, and some studies still name the older edition. “Meets DSM criteria for …” means diagnosed by that edition’s diagnostic criteria, usually by a clinician in an interview.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
DSM diagnostic criteria
The American Psychiatric Association’s manual of mental health diagnoses. DSM-5 (2013, revised as DSM-5-TR in 2022) replaced DSM-IV, and some studies still name the older edition. “Meets DSM criteria for …” means diagnosed by that edition’s diagnostic criteria, usually by a clinician in an interview.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CTH120 75 mg hard capsules will be provided by CONNECTA Therapeutics, S.L. as hard capsules for oral administration. CTH120 75 mg hard capsules contain 75 mg CTH120 drug substance (dose expressed as active moiety) and compendial excipients to be orally administered after breakfast and after dinner with a glass of water. The recommended dosage of CTH120 is 75 mg orally twice daily (BID) in fed conditions after breakfast and after dinner.
Drug: CTH120
CTH120 placebo hard capsules will be provided by CONNECTA Therapeutics, S.L. as hard capsules for oral administration. CTH120 matching placebo hard capsules (that contain the same compendial excipients than and are identical in appearance to CTH120 75 mg hard capsules) will be orally administered twice daily (BID) in fed conditions after breakfast and after dinner with a glass of water.
Other: CTH120 placebo hard capsules
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.
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.
Treatment-emergent adverse events (TEAEs).
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.
Time frame: From Day 1 to End-of-study: EOS will be on Day 56 (±2 days).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in blood pressure (mmHg).
Primary Safety and Tolerability endpoint for FXS-CTH120-01. Blood pressure (mmHg) will be measured in the supine position following a 5 min rest.
Time frame: From Day 1 to End-of-study (EOS): on Day 56 (±2 days).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in pulse rate (bpm).
Primary Safety and Tolerability endpoint for FXS-CTH120-01. Pulse rate (bpm) will be measured in the supine position following a 5 min rest.
Time frame: From Day 1 to End-of-study (EOS): on Day 56 (±2 days).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in body temperature (ºC).
Primary Safety and Tolerability endpoint for FXS-CTH120-01. Body temperature will be measured using an automated vital sign monitor device.
Time frame: From Day 1 to End-of-study (EOS): on Day 56 (±2 days).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: heart rate (bpm).
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).
Time frame: From Day 1 to End-of-study (EOS): on Day 56 (±2 days).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: rhythm.
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.
Time frame: From Day 1 to End-of-study (EOS): on Day 56 (±2 days).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: PQ/PR interval (ms).
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).
Time frame: From Day 1 to End-of-study (EOS): on Day 56 (±2 days).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: QRS duration (ms).
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).
Time frame: From Day 1 to End-of-study (EOS): on Day 56 (±2 days).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: QT (ms).
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).
Time frame: From Day 1 to End-of-study (EOS): on Day 56 (±2 days).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in electrocardiogram (ECG) values: QTcF (ms).
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).
Time frame: From Day 1 to End-of-study (EOS): on Day 56 (±2 days).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: haematology.
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.
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)).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: serum chemistry.
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.
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)).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: coagulation.
Primary Safety and Tolerability endpoint for FXS-CTH120-01. The following tests will be performed: • Coagulation parameters: INR, aPTT, PT.
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)).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: urinalysis.
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).
Time frame: Screening visit (from Day -28 to Day -1).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: viral serology and detection.
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.
Time frame: Screening visit (from day -28 to Day -1).
Observed maximum concentration (Cmax).
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).
Time frame: On Day 15 and Day 42.
Measured concentration at the end of a dosing interval at steady state (Ctrough).
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).
Time frame: On Day 15, Day 28 and Day 42.
Last analytically quantifiable plasma concentration above LLOQ (Clast).
Secondary Pharmacokinetics endpoint CTH120 and its main metabolite for FXS-CTH120-01. • Plasma PK parameter calculated using a non-compartmental model: Clast (ng/mL).
Time frame: On Day 15 and Day 42.
Time to reach Cmax (tmax).
Secondary Pharmacokinetics endpoint for CTH120 and its main metabolite for FXS-CTH120-01. • Plasma PK parameter calculated using a non-compartmental model: tmax (h).
Time frame: On Day 15 and Day 42.
Lag-time (time delay between drug administration and first observed concentration above LOQ in plasma) (tlag).
Secondary Pharmacokinetics endpoint for CTH120 and its main metabolite for FXS-CTH120-01. • Plasma PK parameter calculated using a non-compartmental model: tlag (h).
Time frame: On Day 15 and Day 42.
Time post administration of the last analytically quantifiable concentration (above LLOQ) (tlast).
Secondary Pharmacokinetics endpoint for CTH120 and its main metabolite for FXS-CTH120-01. • Plasma PK parameter calculated using a non-compartmental model: tlast (h).
Time frame: On Day 15 and Day 42.
Area under the plasma concentration-time curve (AUC0-t, AUC0-∞, AUCextrap %)
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).
Time frame: On Day 15 and Day 42.
Elimination rate constant (λz).
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).
Time frame: On Day 15 and Day 42.
Terminal half-life (t1/2).
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).
Time frame: On Day 15 and Day 42.
Apparent clearance (CL/F).
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).
Time frame: On Day 15 and Day 42.
Apparent volume of distribution during terminal phase after oral administration: (Vz/F).
Secondary Pharmacokinetics endpoint for CTH120 for FXS-CTH120-01. • Plasma PK parameter calculated using a non-compartmental model: Vz/F (mL/kg).
Time frame: On Day 15 and Day 42.
Accumulation ratio calculated from Cmax after repeated dosing and Cmax after 1st dosing (RAC Cmax).
Secondary Pharmacokinetics endpoint for CTH120 and its main metabolite for FXS-CTH120-01. • Plasma PK parameter calculated using a non-compartmental model: RAC Cmax.
Time frame: On Day 42.
Accumulation ratio calculated from AUC0-t after repeated dosing and AUC0-t (RAC AUC0-t).
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.
Time frame: On Day 42.
Metabolic ratio of metabolite Cmax and parent drug Cmax (MR Cmax).
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).
Time frame: On Day 15 and Day 42.
Metabolic ratio of metabolite AUC0-t and parent drug AUC0-t (MR AUC0-t).
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).
Time frame: On Day 15 and Day 42.
Metabolic ratio of metabolite AUC0-∞ and parent drug AUC0-∞ (MR AUC0-∞).
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).
Time frame: On Day 15 and Day 42.
Global functioning using the Visual Analogue Scale (VAS)
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.
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)).
Global severity using the Clinical Global Impression Severity Scale (CGI-S)
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".
Time frame: On Baseline visit (From Day -14 to Day -1) and on Day 14.
Global improvement using the Clinical Global Impression Improvement Scale (CGI- I)
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.
Time frame: On Day 14, on Day 42 and on Day 56 (± 2 days) (End-of-study (EOS)).
Behaviour troubles using the Aberrant Behaviour Checklist-Community in FXS (ABC-CFXS)
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.
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)).
Cognitive functioning using the National Institutes of Health Toolbox Cognition Battery Intellectual disability NIH-TCB-ID
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.
Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.
Anxiety, Depression, and Mood Scale (ADAMS).
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.
Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.
Adaptive functioning using the Vineland Adaptive Behaviour Scale 3 (VABS-3)
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.
Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.
Paediatric Quality of Life Inventory (PedsQL) 2.0
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.
Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.
Paediatric Quality of Life Inventory (PedsQL) 3.0
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.
Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.
Paediatric Quality of Life Inventory (PedsQL) 4.0
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.
Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.
Quality of sleep using the Pittsburgh Sleep Quality Index (PSQI)
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.
Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.
Neural functioning using Electroencephalography (EEG) (auditory oddball, resting-state and auditory steady-state response)
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).
Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.
Social avoidance using eye-tracking
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.
Time frame: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.
Biorhythm characteristics using Actigraphy
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.
Time frame: Between Baseline visit (From Day -14 to Day -1) and Day 14, and between Day 28 and Day 42.
Protein levels of FMRP in peripheral blood
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.
Time frame: On Baseline visit (From Day -14 to Day -1) and on Day 42.
BDNF concentrations in plasma
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.
Time frame: On Baseline visit (From Day -14 to Day -1) and on Day 42.
miRNA profile in plasma
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.
Time frame: On Baseline visit (From Day -14 to Day -1) and on Day 42.
Plan to share: No
Started May 2026 (actual). Primary completion is estimated for May 2027, 12 months after the start.
Of 15 phase 2 interventional studies under Fragile X Syndrome, half reached primary completion within 24 months of starting (the median), counting those still running; a quarter within 15 and three quarters within 37. This study's planned 12 months is below that.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Connecta Therapeutics, S.L.