Oral Valproic Acid Treatment for Retinitis Pigmentosa

A Phase II Multiple Site, Randomized, Placebo-Controlled Trial of Oral Valproic Acid for Retinitis Pigmentosa Protocol #H-13371

The purpose of this study is to learn about a drug therapy being studied in patients with Retinitis Pigmentosa (RP). Valproic Acid (VPA) has been approved for the treatment of seizures, certain psychiatric disorders, and migraine headaches. The study investigators want to find out if VPA can help people with RP.VPA is taken by mouth.

Eligibility criteria:

  • Adults 18 years and older
  • Must have clinical diagnosis of RP as determined by decreased visual field, night blindness, reduced ERG responses, and other clinical signs.
  • Must have autosomal dominant RP determined by a disease causing mutation in one of the following genes: RHO, PRPH2, IMPDH1, PRPF31, PRPF3, RP1, and PRPF8
  • Must have visual acuity of greater than 20/200 in at least one eye.
  • For women, not pregnant
  • Willing to use more than one contraceptive method during the course of the study
  • Good general health

Interested participants will come to the Casey Eye Institute in Portland, OR, for a screening visit that will last 1-3 days.If the participant is eligible, the subject will return to Casey Eye Institute for a base-line visit where they will be randomly assigned to either the VPA treatment group or the placebo group.Neither the subject nor the doctors and study staff at Casey will know which group the subject is in. The subject will take VPA (or placebo) for 12 months. More information is available at https://clinicaltrials.gov.

During the study, subjects will have 6 more visits at the Casey Eye Institute. The subjects will also check in with a study coordinator by phone between study visits. Participation will last for 15 months. Each study visit will last about 4-8 hours and may include vision function tests, dilated eye exams, blood draws, and urine collection.

For more information, please call the study coordinator at 503 494-0020.

Principal Investigator:

Mark Pennesi, MD, PhD
Casey Eye Institute
3375 SW Terwilliger Blvd.
Portland, OR97239
eIRB# 8253