Roatan

Researchers are evaluating the safety and effectiveness of a gene therapy for the Klotho gene in healthy adults. This pilot phase 1 study uses a nonviral plasmid to deliver the Klotho gene, aiming to explore potential benefits in cognitive function, kidney health, and overall healthspan and lifespan. The investigational product is given by subcutaneous injection into abdominal fat deposits. The study is notable for being conducted partly outside the U.S., with outcome assessments like cognitive tests and blood samples performed at the U.S. site. Participants receive a single injection of the plasmid-delivered Klotho gene therapy. Before and after treatment, they undergo various evaluations including blood draws, health screenings, questionnaires, and brain perfusion and cognitive function tests. Measurements are taken at multiple time points: one month and seven days before injection, then three days, seven days, one month, three months, and six months after treatment to monitor serum markers and treatment effects. Throughout the study, participants complete cognitive and health assessments to evaluate changes following gene therapy. Researchers measure serum levels of Klotho, fibroblast growth factor 23, parathyroid hormone, vitamin D, phosphorus, cystatin C, and urinary phosphate excretion. Adverse events are tracked through patient-reported outcomes at several intervals post-treatment. The total follow-up period extends to six months after therapy to assess safety and biological responses.

This research aims to assess the safety and effectiveness of a combined klotho and follistatin gene therapy delivered through a nonviral plasmid in healthy adults aged 50 to 80 years. The study also explores potential cognitive and health benefits of this gene therapy in participants who are generally healthy. This is an early phase 1 pilot study without a placebo control. Participants will receive the investigational gene therapy by subcutaneous injection into abdominal fat deposits. The gene therapy involves plasmid delivery of klotho and follistatin genes, which may influence physical and cognitive functions, kidney health, body composition, biological aging, and overall well-being. The gene therapy administration occurs at a site outside the U.S., while pre- and post-treatment assessments take place at the U.S. site. During the study, participants will undergo various cognitive and health tests before and after treatment. Blood samples will be collected to measure serum levels of alpha-Klotho and follistatin at five timepoints from one month prior to treatment up to three months after. Researchers will also monitor and record any treatment-related adverse events reported by participants up to three months post-treatment. Overall participation involves multiple visits and assessments over a period of about four months.