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Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome (FXS-CTH120-01)

12 giugno 2026 aggiornato da: Connecta Therapeutics, S.L.

Evaluation of the Safety, Tolerability, and Effectiveness of CTH120 in Adult Males With 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.

Panoramica dello studio

Stato

Reclutamento

Condizioni

Intervento / Trattamento

Descrizione dettagliata

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.

Tipo di studio

Interventistico

Iscrizione (Stimato)

30

Fase

  • Fase 2

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Contatto studio

Luoghi di studio

  • Spagna
    • Barcelona
      • Barcelona, Barcelona, Spagna, 08003
        • Reclutamento
        • Hospital del Mar Medical Research Institute
        • Contatto:
        • Contatto:
      • Sabadell, Barcelona, Spagna, 08208
        • Reclutamento
        • Consorci Corporació Sanitaria Parc Taulí. Institut Investigació i Innovació Parc Taulí (I3PT)
        • Contatto:
          • Ana Roche Martínez, MD, PhD
          • Numero di telefono: (+34) 93 723 10 10
          • Email: aroche@tauli.cat

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

  • Adulto

Accetta volontari sani

No

Descrizione

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.

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: Randomizzato
  • Modello interventistico: Assegnazione parallela
  • Mascheramento: Quadruplicare

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: CTH120

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.
Droga: 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.
Comparatore placebo: CTH120 placebo hard capsules
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.
Altro: 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.

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Treatment-emergent adverse events (TEAEs).
Lasso di tempo: 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.
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).
Lasso di tempo: 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.
From Day 1 to End-of-study (EOS): on Day 56 (±2 days).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in pulse rate (bpm).
Lasso di tempo: 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.
From Day 1 to End-of-study (EOS): on Day 56 (±2 days).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in body temperature (ºC).
Lasso di tempo: 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.
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).
Lasso di tempo: 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).
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.
Lasso di tempo: 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.
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).
Lasso di tempo: 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).
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).
Lasso di tempo: 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).
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).
Lasso di tempo: 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).
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).
Lasso di tempo: 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).
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.
Lasso di tempo: 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.
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.
Lasso di tempo: 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.
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.
Lasso di tempo: 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.
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.
Lasso di tempo: 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).
Screening visit (from Day -28 to Day -1).
Treatment-emergent potentially clinically significant abnormalities (PSCAs) in safety laboratory parameters: viral serology and detection.
Lasso di tempo: 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.
Screening visit (from day -28 to Day -1).

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
Observed maximum concentration (Cmax).
Lasso di tempo: 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).
On Day 15 and Day 42.
Measured concentration at the end of a dosing interval at steady state (Ctrough).
Lasso di tempo: 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).
On Day 15, Day 28 and Day 42.
Last analytically quantifiable plasma concentration above LLOQ (Clast).
Lasso di tempo: 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).
On Day 15 and Day 42.
Time to reach Cmax (tmax).
Lasso di tempo: 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).
On Day 15 and Day 42.
Lag-time (time delay between drug administration and first observed concentration above LOQ in plasma) (tlag).
Lasso di tempo: 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).
On Day 15 and Day 42.
Time post administration of the last analytically quantifiable concentration (above LLOQ) (tlast).
Lasso di tempo: 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).
On Day 15 and Day 42.
Area under the plasma concentraction-time curve (AUC0-t, AUC0-∞, AUCextrap %)
Lasso di tempo: 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).
On Day 15 and Day 42.
Elimination rate constant (λz).
Lasso di tempo: 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).
On Day 15 and Day 42.
Terminal half-life (t1/2).
Lasso di tempo: 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).
On Day 15 and Day 42.
Apparent clearance (CL/F).
Lasso di tempo: 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).
On Day 15 and Day 42.
Apparent volume of distribution during terminal phase after oral administration: (Vz/F).
Lasso di tempo: 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).
On Day 15 and Day 42.
Accumulation ratio calculated from Cmax after repeated dosing and Cmax after 1st dosing (RAC Cmax).
Lasso di tempo: 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.
On Day 42.
Accumulation ratio calculated from AUC0-t after repeated dosing and AUC0-t (RAC AUC0-t).
Lasso di tempo: 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.
On Day 42.
Metabolic ratio of metabolite Cmax and parent drug Cmax (MR Cmax).
Lasso di tempo: 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).
On Day 15 and Day 42.
Metabolic ratio of metabolite AUC0-t and parent drug AUC0-t (MR AUC0-t).
Lasso di tempo: 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).
On Day 15 and Day 42.
Metabolic ratio of metabolite AUC0-∞ and parent drug AUC0-∞ (MR AUC0-∞).
Lasso di tempo: 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).
On Day 15 and Day 42.
Global functioning using the Visual Analogue Scale (VAS)
Lasso di tempo: 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. 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.
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)
Lasso di tempo: 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".
On Baseline visit (From Day -14 to Day -1) and on Day 14.
Global improvement using the Clinical Global Impression Improvement Scale (CGI- I)
Lasso di tempo: 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.
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)
Lasso di tempo: 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.
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 NIH-TCB-ID
Lasso di tempo: 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.
On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.
Anxiety, Depression, and Mood Scale (ADAMS).
Lasso di tempo: 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.
On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.
Adaptive functioning using the Vineland Adaptive Behaviour Scale (VABS-3)
Lasso di tempo: On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.

Exploratory Efficacy endpoints for FXS-CTH120-01:

VABS-3 is a psychometric instrument that assesses the adaptive level of functioning by standardized interview of the person or their caregiver through their activities of daily living such as walking, talking, getting dressed, going to school, preparing a meal, etc.
On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.
Paediatric Quality of Life Inventory PedsQL
Lasso di tempo: 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)
  • Quality of life using the PedsQL 4.0 Generic Core Scales (PARENT Reports for Young Adults (ages 18-25) and Adults (ages over 26)
  • Quality of life using the PedsQL 2.0 Family Impact Module (parent impact for all ages)
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)
Lasso di tempo: 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.
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)
Lasso di tempo: 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).
On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.
Social avoidance using eye-tracking
Lasso di tempo: 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 participanta are exposed to stimuli consisting on coloured photographs of adult human faces.
On Baseline visit (From Day -14 to Day -1), on Day 14, and on Day 42.
Biorhythm characteristics using Actigraphy
Lasso di tempo: 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.
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
Lasso di tempo: 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.
On Baseline visit (From Day -14 to Day -1) and on Day 42.
BDNF concentrations in plasma
Lasso di tempo: 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.
On Baseline visit (From Day -14 to Day -1) and on Day 42.
miRNA profile in plasma
Lasso di tempo: 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.
On Baseline visit (From Day -14 to Day -1) and on Day 42.

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Connecta Therapeutics, S.L.

Collaboratori

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

Investigatori

  • Investigatore principale: Ana Roche Martínez, MD, PhD, Consorci Corporació Sanitària Parc Taulí. Institut d'Investigació i Innovació Parc Taulí (I3PT)
  • Investigatore principale: Rafael De la Torre, Pharm, PhD, Hospital del Mar Medical Research Institute
  • Investigatore principale: Ana María Aldea, MD, PhD, Hospital del Mar Medical Research Institute

Pubblicazioni e link utili

La persona responsabile dell'inserimento delle informazioni sullo studio fornisce volontariamente queste pubblicazioni. Questi possono riguardare qualsiasi cosa relativa allo studio.

Pubblicazioni generali

Collegamenti utili

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Effettivo)

21 maggio 2026

Completamento primario (Stimato)

1 maggio 2027

Completamento dello studio (Stimato)

1 maggio 2027

Date di iscrizione allo studio

Primo inviato

12 giugno 2026

Primo inviato che soddisfa i criteri di controllo qualità

12 giugno 2026

Primo Inserito (Effettivo)

17 giugno 2026

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

17 giugno 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

12 giugno 2026

Ultimo verificato

1 giugno 2026

Maggiori informazioni

Termini relativi a questo studio

Parole chiave

Termini MeSH pertinenti aggiuntivi

Altri numeri di identificazione dello studio

  • CTH-CTH120-FXS-01
  • 2025-522972-97-00 (Ctis)

Piano per i dati dei singoli partecipanti (IPD)

Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?

NO

Informazioni su farmaci e dispositivi, documenti di studio

Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti

No

Studia un dispositivo regolamentato dalla FDA degli Stati Uniti

No

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .

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Sponsor e collaboratori

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CROs by country

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Condizioni

Malattie rare

Interventi farmacologici

Supplementi dietetici

Sponsor / Collaboratori

Sedi

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