Connecta Therapeutics has started a new clinical trial (Phase IIa) for a drug called CTH120. This drug is designed to help adults with fragile X syndrome, a genetic condition that causes intellectual disability. Fragile X syndrome affects about 3 out of every 10,000 people and is the most common inherited cause of this type of disability. CTH120 works by targeting TrkB, a protein in the brain that plays a key role in how brain cells adapt and change, a process called neuroplasticity. Problems with neuroplasticity are a major part of neurodevelopmental disorders like fragile X syndrome.
Jordi Fàbrega, the CEO of Connecta Therapeutics, stated that starting this trial is a major step forward. Earlier tests (Phase I) showed that CTH120 was safe, and this new study will further check its safety and how well the body tolerates it. The trial will also look at whether the drug improves symptoms related to brain function in fragile X syndrome.
The study is being conducted as a randomized, double-blind, placebo-controlled trial. This means that 30 adult men aged 18 to 45 will be randomly assigned to receive either CTH120 or a placebo (a sugar pill with no active drug) twice a day. Neither the participants nor the researchers will know who is receiving the real drug until the study ends. The main goal is to check for safety and side effects, but the study will also measure how the drug moves through the body (pharmacokinetics) and whether it improves symptoms.
Dr. Rafael de la Torre Fornell, the lead researcher for the study at Hospital del Mar Research Institute, explained that fragile X syndrome has a serious impact on thinking, behavior, and overall quality of life for those affected and their families. Currently, there are no approved treatments that can change the course of the disease. This trial offers a chance to test a new and promising approach to treatment and help us better understand this lifelong condition.
The trial will take place at two locations in Spain: Hospital del Mar Research Institute in Barcelona and Parc Taulí Research and Innovation Institute Foundation in Sabadell. The study is led by Mara Dierssen from the Centre for Genomic Regulation and will also contribute to research on biomarkers, which are measurable indicators of disease progression.
The trial has been approved by the Spanish Agency of Medicines and Medical Devices and is funded by the Spanish Ministry of Science, Innovation and Universities, along with the EU’s NextGeneration EU programme.