Methodological Insights for Randomized Clinical Trials of Retinitis Pigmentosa

There are no approved drug treatments for autosomal-dominant retinitis pigmentosa.
 
A study of oral Valproic acid (VPA) does not support the use of VPA in the treatment of autosomal-dominant retinitis pigmentosa.
 
The lessons learned from a trial of Valproic acid are:

• Rigorous evaluation of drug treatment for retinitis pigmentosa is necessary.
• FDA-approved drugs for other uses can have major adverse effects.
• Patients vary greatly in their rates of progression of retinitis pigmentosa with identical gene mutations.

“Methodological Insights for Randomized Clinical Trials of Retinitis Pigmentosa”
Lessons Learned from a Trial of Valproic Acid

JAMA Ophthalmol 136:857-858, August 2018
Brian P. Brooks, MD, PhD; Brett Jeffrey, PhD

Full Paper: Methodological Insights for Randomized Clinical Trials of Retinitis Pigmentosa_08-13-18

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