Could a liver drug help people see color? tiny trial launched

NCT ID NCT04041232

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This early-phase trial tests whether an FDA-approved drug called glycerol phenylbutyrate (PBA) can improve vision in people with a rare form of color blindness caused by mutations in the ATF6 gene. Only 2 participants are enrolled, and they will take PBA three times daily while their vision is monitored with various tests. The study is currently suspended, so results are not yet available.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

glycerol phenylbutyrate (PBA)

What this could lead to

If it works, this could point toward a treatment to improve vision in people with a rare form of color blindness caused by ATF6 mutations.

What could go wrong

This is a very early, tiny trial (only 2 participants) that is currently suspended. Even if results are positive, much larger studies would be needed to confirm any benefit.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for ACHROMATOPSIA 7 are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

achromatopsia achromatopsia 7 Color Vision Defects

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Columbia University Irving Medical Center

    New York, New York, 10032, United States