According to a story from drugs.com, the biopharmaceutical company Reata Pharmaceuticals, Inc., recently announced that is had recently completed a end of Phase 2 meeting with officials from the US Food and Drug Administration (FDA). In this interaction, the company and the agency came to a final agreement on the design of the Phase 3 trial testing bardoxolone as a treatment for autosomal dominant polycystic kidney disease (ADPKD).
About Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Autosomal dominant polycystic kidney disease (ADPKD) is the most widespread monogenic human disorder that can be potentially fatal. However, it is still generally rare. It is characterized by the development of cysts in the kidneys and sometimes other organs as well. ADPKD is caused by mutations affecting the PKD1 gene in 85 percent of cases; in the remainder, the mutation affects the PKD2 gene. The presentation of the disease varies considerably, even within families. Signs of kidney problems often do not appear until middle age, although cysts may begin formation very early in life. Symptoms include anemia, bloody urine, acute loin pain, uremia, liver cysts, berry aneurysm, and high blood pressure. Treatment options are varied; aquaretics can slow disease progression temporarily. Other options include a number of surgical procedures, dialysis, and, when the kidneys begin to fail, kidney transplant. To learn more about ADPKD, click here.
Clinical Trial Design
The clinical trial will be international in scope; it will be placebo controlled and double-blind. The company intends to recruit 300 patients with ADPKD to participate. The participants may range in age from 18 years to 70 years. In order to assess the effectiveness of bardoxolone, the trial will measure changes to the estimated glomerular filtration rate. Patients will be treated for a period of 48 weeks and will have the final filtration rate measured after a one month withdrawal period.
The data from this trial could, if it is encouraging enough, warrant accelerated approval, which could speed up the process of getting bardoxolone approved as a treatment for ADPKD. The patients will also be monitored for a second year in order to document the effect of the treatment in the longer term.
The company is confident that the drug will display strong results in this trial, if the findings from the previous Phase 2 trial are anything to go by. Bardoxolone is also being tested in an ongoing Phase 3 trial as a treatment for Alport syndrome, another rare disease that affects the kidneys.