Badanie nad terapią genową SBT101 u dorosłych pacjentów z adrenomieloneuropatią

Adrenomyeloneuropathy is a rare genetic disorder that affects the nervous system, leading to problems with movement and coordination. This study is focused on evaluating a new treatment called SBT101, which is a type of gene therapy. Gene therapy involves introducing new genetic material into the body to help treat or prevent diseases. In this case, SBT101 uses a special viral vector, known as adeno-associated viral vector serotype 9, to deliver a healthy version of the ABCD1 gene to patients. This gene is important for the proper functioning of nerve cells.

The purpose of the study is to assess the safety and effectiveness of a one-time administration of SBT101 in adults diagnosed with adrenomyeloneuropathy. The treatment is given through a procedure called intrathecal administration, which involves delivering the therapy directly into the spinal fluid. This method helps the treatment reach the nervous system more effectively. The study is designed to monitor how well patients tolerate the treatment and to observe any changes in their condition over time.

Participants in the study will receive either the SBT101 treatment or a placebo. The study will follow a randomized and blinded approach, meaning neither the participants nor the researchers will know who receives the actual treatment or the placebo. The study will track various health indicators, such as walking ability and balance, over a period of time to determine the treatment's impact. The main goal is to ensure the treatment is safe and to gather information on its potential benefits for people with adrenomyeloneuropathy.