2-[4-[3-(METHYLAMINO)-1-PHENYLPROPOXY]PHENYL]ETHANOL HYDROCHLORIDE for Fragile X syndrome

Inclusion criteria

• {"criterion_text":"- Adult male participants\n- Aged ≥ 18 and ≤ 45 years\n- Weight ≥ 50 kg and ≤ 100 kg\n- Body mass index (BMI) ≥ 18.5 and ≤ 32\n- Clinical and molecular diagnosis of Fragile X syndrome (> 200 CGG repeats in the promoter region of the FMR1 gene)\n- 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\n- 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.\n- Participant assenting and/or willing to participate\n- Signed informed consent by legal representative prior to any study-mandated procedure\n- 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\n- Participants are expected to complete all procedures scheduled during the study visits\n- VCI scaled score >4 on the WISC-V, based on mental age"}

Exclusion criteria

• {"criterion_text":"- Personal history of infantile spasms/convulsions/epilepsy, severe head trauma or CNS infections (e.g. meningitis), except for infantile febrile seizures\n- Not more than 3 psychotropic medications simultaneously in the 8 weeks prior to screening and also during the study.\n- Any new prescription or over the counter drug (except occasional use of paracetamol) in the last 2 weeks before Day 1\n- Participation in a clinical study with investigational treatments in the last 8 weeks prior to screening\n- Auditory or visual impairments that cannot be corrected\n- Positive EtG/EtS test in urine\n- Positive drug test in urine\n- 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\n- Substance use disorder according to the DSM-5 criteria\n- Epileptiform abnormalities on EEG (excluding isolated sharp waves and beyond those expected for age)\n- Any life-threatening medical disease\n- 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\n- Any clinically significant findings on physical examination including clinically significant vital sign abnormalities, from the perspective of the investigator\n- 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\n- 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\n- Known hypersensitivity or intolerance to any component of the investigational medicinal product or its excipients."}

Primary endpoints

• {"endpoint_text":"- Treatment-emergent adverse events (TEAEs) from Day 1 to the End-of-study (EOS)","definition_or_measurement_approach":"Collection and recording of treatment-emergent adverse events occurring from Day 1 through end of study (EOS); standard AE reporting per protocol."}
• {"endpoint_text":"- Treatment-emergent potentially clinically significant abnormalities (PSCAs) in vital signs, 12-lead ECG and safety laboratory parameters from Day 1 to EOS","definition_or_measurement_approach":"Assessment of vital signs, 12-lead ECG and safety laboratory parameters from Day 1 to EOS to identify potentially clinically significant abnormalities emergent after treatment."}

Secondary endpoints

• {"endpoint_text":"- Exploratory plasma pharmacokinetics of CTH120 (Cmax, Ctrough, Clast, tmax, tlag, tlast, AUC0-t, AUC0-∞, AUCextrap %, λz, t1/2, CL/F, Vz/F, RAC AUC0-t and RAC Cmax)","definition_or_measurement_approach":"Plasma PK sampling and analysis to derive listed PK parameters for CTH120."}
• {"endpoint_text":"- Exploratory plasma pharmacokinetics of metabolite M4 (Cmax, Ctrough, Clast, tmax, tlag, tlast, AUC0-t, AUC0-∞, AUCextrap %, λz, t1/2, MR Cmax, MR AUC0-t, and MR AUC0-∞, RAC AUC0-t and RAC Cmax)","definition_or_measurement_approach":"Plasma PK sampling and analysis to derive listed PK parameters for metabolite M4."}
• {"endpoint_text":"- Exploratory PK values assessment considering the different CYP2D6 metaboliser phenotypes","definition_or_measurement_approach":"Comparison of PK parameters across CYP2D6 metaboliser phenotype groups."}
• {"endpoint_text":"- Global functioning using the Visual Analogue Scale (VAS)","definition_or_measurement_approach":"Assessment of global functioning via VAS score."}
• {"endpoint_text":"- Global severity and improvement using the Clinical Global Impression Improvement Scale (CGI-S and CGI- I)","definition_or_measurement_approach":"Assessment of severity and improvement using CGI-S and CGI-I scales."}
• {"endpoint_text":"- Behaviour troubles using the Aberrant Behaviour Checklist-Community in FXS (ABC-CFXS)","definition_or_measurement_approach":"Assessment of behavioural problems using ABC-CFXS instrument."}
• {"endpoint_text":"- Cognitive functioning using the NIH-TCB-ID","definition_or_measurement_approach":"Cognitive assessment using NIH-TCB-ID battery."}
• {"endpoint_text":"- Anxiety using the Anxiety, Depression, and Mood Scale (ADAMS)","definition_or_measurement_approach":"Assessment of anxiety via ADAMS scale."}
• {"endpoint_text":"- Adaptive functioning using the Vineland Adaptive Behaviour Scale (VABS-3)","definition_or_measurement_approach":"Assessment of adaptive functioning via VABS-3."}
• {"endpoint_text":"- Cognitive functioning using the PedsQL 3.0 Cognitive Functioning Scale (PARENT Reports for Young Adults (ages 18-25) and Adults (ages over 26)","definition_or_measurement_approach":"Parent-reported cognitive functioning using PedsQL 3.0 cognitive module for specified age groups."}
• {"endpoint_text":"- Quality of life using the PedsQL 4.0 Generic Core Scales (PARENT Reports for Young Adults (ages 18-25) and Adults (ages over 26)","definition_or_measurement_approach":"Parent-reported quality of life using PedsQL 4.0 Generic Core Scales for specified age groups."}
• {"endpoint_text":"- Quality of life using the PedsQL 2.0 Family Impact Module (parent impact for all ages)","definition_or_measurement_approach":"Assessment of family impact using PedsQL 2.0 Family Impact Module (parent report)."}
• {"endpoint_text":"- Quality of sleep using the Pittsburgh sleep quality index (PSQI)","definition_or_measurement_approach":"Assessment of sleep quality using PSQI questionnaire."}
• {"endpoint_text":"- Neural functioning using Electroencephalography (EEG) (auditory oddball, resting-state and auditory steady-state response)","definition_or_measurement_approach":"EEG recordings using specified paradigms to assess neural functioning."}
• {"endpoint_text":"- Social attention using eye-tracking","definition_or_measurement_approach":"Eye-tracking measures to assess social attention."}
• {"endpoint_text":"- Biorhythm characteristics using Actigraphy","definition_or_measurement_approach":"Actigraphy monitoring to assess biorhythm characteristics."}
• {"endpoint_text":"- Protein levels of FMRP in peripheral blood","definition_or_measurement_approach":"Laboratory measurement of peripheral blood FMRP protein levels."}
• {"endpoint_text":"- BDNF concentrations in serum","definition_or_measurement_approach":"Laboratory measurement of serum BDNF concentrations."}

Spain

Earliest CTIS Part Ii Submission Date

22-10-2025

Latest Decision Or Authorization Date

01-12-2025

Processing Time Days

40

Number Of Sites

3

Number Of Participants

30

Primary sponsor

Full Name

Connecta Therapeutics S.L.

Organisation Type

Pharmaceutical company

Country Of Registered Address

Spain

Contract research organisations

Name

Astrum Cro Spain S.L.

Responsibilities

Regulatory and pharmacovigilance support and other sponsor duties (codes 1,10,12,15 as listed); contact regulatory@astrumcro.com

Third parties

• {"country":"Spain","full_name":"Astrum Cro Spain S.L.","duties_or_roles":"Codes: 1, 10, 12, 15 (Pharmacovigilance)","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Spain","full_name":"Parc Tauli Hospital Universitari","duties_or_roles":"Code: 15 (Actigraph analyses)","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Spain","full_name":"Fundacio Privada Mon Clinic Barcelona","duties_or_roles":"Code: 12","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Spain","full_name":"Parc Tauli Hospital Universitari","duties_or_roles":"Code: 4","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Spain","full_name":"Consorci Mar Parc De Salut De Barcelona","duties_or_roles":"Code: 4","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Spain","full_name":"Parc Tauli Hospital Universitari","duties_or_roles":"Code: 4","organisation_type":"Hospital/Clinic/Other health care facility"}