| Autor: |
Erickson CA; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.; Department of Psychiatry and Behavioral Neuroscience, University of Cincinnati, Cincinnati, OH 45229, USA., Perez-Cano L; Discovery and Data Science (DDS) Unit, STALICLA SL, Moll de Barcelona, s/n, Edif Este, 08039 Barcelona, Spain., Pedapati EV; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.; Department of Psychiatry and Behavioral Neuroscience, University of Cincinnati, Cincinnati, OH 45229, USA.; Division of Child Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA., Painbeni E; Drug Development Unit (DDU), STALICLA SA, Campus Biotech Innovation Park, Avenue de Sécheron 15, 1202 Geneva, Switzerland., Bonfils G; Drug Development Unit (DDU), STALICLA SA, Campus Biotech Innovation Park, Avenue de Sécheron 15, 1202 Geneva, Switzerland., Schmitt LM; Division of Behavioral Medicine and Clinical Psychology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA.; Department of Pediatrics, College of Medicine, University of Cincinnati, Cincinnati, OH 45229, USA., Sachs H; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA., Nelson M; Division of Behavioral Medicine and Clinical Psychology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA., De Stefano L; Division of Behavioral Medicine and Clinical Psychology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA., Westerkamp G; Division of Child and Adolescent Psychiatry, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA., de Souza ALS; Drug Development Unit (DDU), STALICLA SA, Campus Biotech Innovation Park, Avenue de Sécheron 15, 1202 Geneva, Switzerland., Pohl O; Drug Development Unit (DDU), STALICLA SA, Campus Biotech Innovation Park, Avenue de Sécheron 15, 1202 Geneva, Switzerland., Laufer O; Firefly Neuroscience, Herzliya 4672501, Israel., Issachar G; Firefly Neuroscience, Herzliya 4672501, Israel., Blaettler T; Drug Development Unit (DDU), STALICLA SA, Campus Biotech Innovation Park, Avenue de Sécheron 15, 1202 Geneva, Switzerland., Hyvelin JM; Drug Development Unit (DDU), STALICLA SA, Campus Biotech Innovation Park, Avenue de Sécheron 15, 1202 Geneva, Switzerland., Durham LA; Drug Development Unit (DDU), STALICLA SA, Campus Biotech Innovation Park, Avenue de Sécheron 15, 1202 Geneva, Switzerland. |
| Abstrakt: |
This study aimed to evaluate the safety and tolerability of STP1, a combination of ibudilast and bumetanide, tailored for the treatment of a clinically and biologically defined subgroup of patients with Autism Spectrum Disorder (ASD), namely ASD Phenotype 1 (ASD-Phen1). We conducted a randomized, double-blind, placebo-controlled, parallel-group phase 1b study with two 14-day treatment phases (registered at clinicaltrials.gov as NCT04644003). Nine ASD-Phen1 patients were administered STP1, while three received a placebo. We assessed safety and tolerability, along with electrophysiological markers, such as EEG, Auditory Habituation, and Auditory Chirp Synchronization, to better understand STP1's mechanism of action. Additionally, we used several clinical scales to measure treatment outcomes. The results showed that STP1 was well-tolerated, with electrophysiological markers indicating a significant and dose-related reduction of gamma power in the whole brain and in brain areas associated with executive function and memory. Treatment with STP1 also increased alpha 2 power in frontal and occipital regions and improved habituation and neural synchronization to auditory chirps. Although numerical improvements were observed in several clinical scales, they did not reach statistical significance. Overall, this study suggests that STP1 is well-tolerated in ASD-Phen1 patients and shows indirect target engagement in ASD brain regions of interest. |
