Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexMale
Age18-45
SponsorConnecta Therapeutics, S.L.
About this trial
The purpose of this Phase IIa study is to evaluate the safety, tolerability, and effectiveness of CTH120 in adult males with Fragile X syndrome.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Adult male participants.
Aged ≥ 18 and ≤ 45 years.
Weight ≥ 50 kg and ≤ 100 kg.
Body mass index (BMI) ≥ 18.5 and ≤ 32.
Disqualifiers
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).
Trial design
Design model
Parallel
Treatments tested in this trial
CTH120
Drug15 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 intervention15 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 groups
30 Participants
are divided into 2 treatment groupsGroup A: CTH120Experimental treatment 1 intervention
Group B: CTH120 placebo hard capsulesPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
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).2
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).3
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).4
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).Secondary outcomes
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 42.2
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 42.3
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 42.4
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.Sponsors and contacts
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Connecta Therapeutics, S.L.
Lead sponsor
Hospital del Mar Research Institute (IMIM)
Collaborator
Corporacion Parc Tauli
Collaborator
Ministry of Science and Innovation, Spain
Collaborator
Astrum CRO, S.L.
Collaborator
Source ClinicalTrials.gov