CONNECTA Therapeutics has commenced a Phase IIa trial to assess its lead programme, CTH120, a neuroplasticity modulator, in male adults living with fragile X syndrome (FXS), an inherited cause of intellectual disability.
CTH120 is a new small molecule designed to target the tropomyosin receptor kinase B (TrkB), which regulates neuroplasticity, the brain’s capacity to form and adapt neural pathways.
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Impaired neuroplasticity is central to neurodevelopmental disorders and is a known contributor to clinical symptoms in conditions such as FXS.
The double-blind, randomised, parallel-group, multi-centre, placebo-controlled trial will compare CTH120, administered twice daily, with a placebo.
Adult males aged 18–45 years, the population most severely affected due to the X-linked nature of FXS, will participate.
The primary endpoint is safety and tolerability, with secondary endpoints including pharmacokinetics and clinical efficacy. A total of 30 patients will be randomised in a 1:1 ratio.
The trial will take place at Hospital del Mar Research Institute and Parc Taulí Research and Innovation Institute Foundation in Spain, led by Dr Rafael de la Torre Fornell, Ana Aldea Perona, and Ana Roche Martínez.
The Centre for Genomic Regulation, under Mara Dierssen, will contribute to the identification of disease-specific biomarkers and to understanding treatment responses.
The Spanish Agency of Medicines and Medical Devices (AEMPS) has authorised the study, with relevant ethics committee approvals.
Funding comes from the Spanish Ministry of Science, Innovation and Universities and the European Union’s NextGeneration EU programme.
CONNECTA Therapeutics co-founder and CEO Jordi Fàbrega said: “Building on encouraging Phase I safety data, this trial is designed to further evaluate the safety and tolerability of CTH120 and to generate critical insights into its potential therapeutic benefit by modulating pathways implicated in disrupted neuronal function in FXS.
“We believe this study could support the development of a novel, disease-modifying approach for FXS and serve as an important proof point for our broader neuroplasticity modulation platform.”
