Prevail Therapeutics is recruiting patients for PROPEL, a clinical trial testing an investigational gene therapy called PR001 to treat Parkinson’s disease in people with GBA mutations. About 7 to 10 percent of people with Parkinson’s have mutations in the GBA gene, making it the most common genetic cause of the disease. The GBA gene is responsible for making an enzyme called glucocerebrosidase, which is low in people who carry a GBA mutation.
PR001 was designed to slow down the progression of the symptoms of Parkinson’s disease specifically in patients who carry GBA mutations. In animal models, PR001 provided brain cells a working copy of the GBA gene, resulting in an increase in the enzyme glucocerebrosidase and improvements in other types of pathology associated with low glucocerebrosidase levels. The investigational treatment requires only a single, one-time injection.
This past July (2019), the FDA granted Fast Track Designation for this AAV9-based gene therapy.
Sixteen study participants are sought for this Phase I/II trial that will be conducted at several clinical sites in the United States. More sites in other countries will be announced as and when approval is received.
Eligible participants must have Parkinson's disease with at least one GBA mutation. Patients with both Gaucher disease and Parkinson’s disease may also be eligible. Don’t know if you have a mutation? Prevail offers programs for genetic testing at PROPEL clinical sites.
To learn more about the clinical trial visit https://clinicaltrials.gov/ct2/show/NCT04127578?term=GBA&cond=Parkinson&draw=2&rank=1 or contact the Prevail PROPEL recruitment team with questions or to express interest in study participation on firstname.lastname@example.org