Connecta Therapeutics has begun a phase IIa clinical trial of CTH120, its first‑in‑class neuroplasticity modulator, in adult males with fragile X syndrome.
CTH120 is a novel small molecule that targets TrkB, a key regulator of neuroplasticity. Impaired neuroplasticity is a core feature of neurodevelopmental disorders, including fragile X syndrome, the most common inherited cause of intellectual disability, affecting around 3 in 10,000 people.
Jordi Fàbrega, Co Founder and CEO of Connecta Therapeutics, said: “Initiating this phase 2a study represents a significant milestone for CONNECTA. Building on encouraging phase 1 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.”
The randomized, double blind, placebo controlled, parallel group study will assess twice‑daily dosing of CTH120 versus placebo in 30 adult males aged 18–45. Safety and tolerability form the primary endpoint, with pharmacokinetics and clinical efficacy as secondary measures. Participants will be enrolled in a 1:1 ratio.
Dr Rafael de la Torre Fornell, Principal Investigator and Study Coordinator at Hospital del Mar Research Institute, said: “FXS has a profound impact on cognitive and behavioral function, severely affecting the quality of life of individuals living with the condition and their families.
“It remains a condition with significant unmet medical need, with no approved disease modifying treatments. This Phase IIa trial provides an important opportunity to evaluate a promising new mechanism of action and advance clinical understanding of this lifelong condition.”
The study will run at Hospital del Mar Research Institute in Barcelona and the Parc Taulí Research and Innovation Institute Foundation in Sabadell. Under the leadership of Mara Dierssen at the Centre for Genomic Regulation, it will also contribute to biomarker research.
The trial has been authorised by the Spanish Agency of Medicines and Medical Devices and is funded by the Spanish Ministry of Science, Innovation and Universities and the EU’s NextGeneration EU programme.
