Phase 2 Evaluation of Oral CTH120 Capsules for Safety and Effectiveness in Adult Males with Fragile X Syndrome Aged 18 to 45

What this Trial Is About

Researchers are evaluating CTH120 in adult males aged 18 to 45 with Fragile X syndrome to assess its safety, tolerability, and preliminary effectiveness. This Phase IIa clinical trial includes 30 participants randomized equally to receive either CTH120 or a placebo. The study is sponsored by Connecta Therapeutics, S.L., aiming to better understand potential treatment effects in this specific population. Participants will receive either CTH120 75 mg capsules or placebo capsules twice daily after meals for approximately 8 weeks. The drug and placebo are taken orally with water after breakfast and dinner, spaced about 12 hours apart. The trial includes a screening period of up to 28 days before treatment and a safety follow-up lasting about 14 days after treatment ends, with a total participation duration of approximately 3 months per participant. During the study, participants will undergo multiple assessments including monitoring for adverse events, vital signs, ECGs, and laboratory tests at several time points. Cognitive and behavioral evaluations, quality of life questionnaires, and biomarker analyses will be conducted at baseline and during treatment. Researchers will monitor treatment-emergent adverse events and clinically significant abnormalities throughout, with a final safety follow-up visit to ensure participant well-being.

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