Clinical trials on LGMDR9

  • CT-EU-00117800

    Gene Therapy Trial for Limb-Girdle Muscular Dystrophy Type R9

    This here clinical trial is called the “GNT0006 Gene Therapy Trial in Patients With LGMDR9“. It’s a study to test the safety and effectiveness of a new gene therapy treatment for a rare muscle disease called LGMDR9, also known as limb-girdle muscular dystrophy type R9.

    The study has two main parts. In the first part, called Stage 1, a small group of patients will receive the gene therapy treatment at different dose levels to make sure it’s safe and to find the best dose. In the second part, called Stage 2, a larger group of 33 patients who can still walk will be split into two groups. One group will get the gene therapy treatment at the best dose found in Stage 1, and the other group will get a placebo, which is like a fake treatment. After one year, the groups will switch treatments.

    The main thing the researchers are looking at is how the gene therapy affects the patients’ lung function, measured by something called Forced Vital Capacity. They’ll be checking this at different time points up to one year after the treatment. The study will also look at whether the treatment is safe and how long the effects last, with patients being followed for up to 5 years after receiving the real gene therapy treatment.

    So in a nutshell, this is a clinical trial testing a new gene therapy for a rare muscle disease, with the main goal being to see if it can improve lung function and whether it’s safe for patients to take. The study will take several years to complete, but could lead to an important new treatment option for people with this condition.