Clinical Trials at the Penn FTD Center

Clinical research is the heart of all medical advances by looking at new ways to prevent, detect, or treat disease. The goal of clinical research is to help doctors and researchers learn more about disease and improve health care for people in the future. Clinical trials are an essential way to determine whether new drugs, diagnostics, or treatments are both safe and effective. These trials are critically important for developing cures for conditions like FTD. Over the last 20 years, we’ve seen incredible advancements in FTD research, including a number of clinical trials. 

The Penn FTD Center has been a site for multiple clinical trials over the years and is proud to be home to many active trials today. Currently active trials include the PROCLAIM trial, the upliFT-D trial, the DNLI-H-0001 trial, and the Veri-T-001 trial. 

The PROCLAIM, upliFT-D, and DNLI-H-0001 trials are for individuals who are confirmed carriers of a GRN mutation causative of FTD (also known as FTD-GRN). Individuals with FTD-GRN have a mutation in the GRN gene so they are making about 50% less of a protein called progranulin. Progranulin deficiency is a known cause of FTD, so it is a good therapeutic target for clinical trials.

The PROCLAIM and upliFT-D trials are gene therapy trials. Gene therapy is a therapeutic technique that aims to fix a faulty gene or replace it with a healthy gene to try and stop disease caused by the faulty gene. Both trials involve a one-time dose of the study drug into the fluid area at the base of the skill (called the cisterna magna). Injecting the study drug into the cisterna magna allows for the study drug to efficiently get into cells throughout the brain delivering a healthy copy of the gene into those cells. The PROCLAIM and upliFT-D trials are both early phase trials, meaning that the goal of these trials is to evaluate the safety, tolerability, and effects of the product rather than focusing on clinical efficacy. 

The DNLI-H-0001 trial is a protein replacement therapy for individuals with a mutation in the GRN gene (FTD-GRN). Protein replacement is a therapeutic technique that aims to restore or supplement the missing or deficient protein. This trial is placebo-controlled with an option to continue into an open-label period, meaning that the first 6 months both the study team and patient won’t know if they are receiving the active study drug or a placebo and after that time, the patient has the option to begin receiving the study drug for the next 18 months. The study drug (or placebo) is delivered via IV infusions every 4 weeks. This trial is a phase 1/2, meaning that the goal of these trials is to evaluate the safety, tolerability, and effects of the product rather than focusing on clinical efficacy.

The Veri-T-001 trial is an oral medication trial for individuals with svPPA. The medication, called Verdiperstat, inhibits myeloperoxidase which is an enzyme involved in inflammation. This may reduce oxidative stress and neuroinflammation that is seen in svPPA patients. This trial is placebo-controlled, meaning that some participants will receive the active drug and some will receive a placebo. The study period is 24-weeks with visits occurring every 4 weeks on site. Patients are asked to take the study drug twice a day. This trial is a phase 1 study, meaning that the goal is to evaluate the safety, tolerability, pharmacology, and preliminary efficacy of the medication. 

The Penn FTD Center is excited to be involved in so many promising clinical trials for FTD. If you are interested in learning more about clinical trials at the Penn FTD Center, please check out our website or reach out to our clinical trials team senior coordinator, Quinn Hlava at quinn.hlava@pennmedicine.upenn.edu. 

~ Quinn Hlava, Senior Clinical Research Coordinator