The Foundation Fighting Blindness has launched a Phase II clinical trial of valproic acid, now underway, to evaluate the drug's effectiveness in slowing vision loss for people with autosomal dominant forms of retinitis pigmentosa (adRP), an inherited retinal degeneration that progressively leads to blindness. Autosomal dominant forms affect multiple generations within families. Valproic acid is already FDA-approved for the treatment of some seizure disorders, and preclinical and clinical research has yielded evidence suggesting the drug may also slow vision loss in people with adRP. The study is the first launched by the National Eye Evaluation and Research (NEER) network, the clinical trial network of the Foundation Fighting Blindness. Recruitment for the 90-participant clinical trial is now underway at two sites: Retina Foundation of the Southwest in Dallas, TX and the University of Utah in Salt Lake City, UT.

"The Foundation Fighting Blindness' NEER network was established to accelerate promising advancements from the lab into the clinic, and to visually impaired patients who need them," said Steven Bramer, Ph.D., Foundation Fighting Blindness Chief Drug Development Officer. "Examining FDA-approved drugs like valproic acid for their effectiveness in treating retinal degenerative diseases is part of our vast portfolio of research to save and restore sight."

Shalesh Kaushal, M.D., Ph.D. is the innovator of the VPA study; David Birch, Ph.D., is the principal investigator at the Retina Foundation of the Southwest; and Paul Bernstein, M.D., Ph.D., is the principal investigator at the University of Utah. Participants are required to visit one of the two clinical trial centers approximately seven times during the trial, including an initial screening, baseline testing, and five follow-up appointments. The valproic acid will be self-administered daily by participants in the form of ingestible capsules. Participants' visual fields will be the primary outcome measured in the double-masked study (i.e. patients will not know if they are being treated with valproic acid or a placebo).

Source:
Foundation Fighting Blindness