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

NCT ID NCT04041232

Symptom relief Sponsor: Columbia University Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused This study
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Sep 11, 2026 · Updated 1 time

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Early phase 1

The earliest testing in people: a first look at safety, in a very small group.

Participants

2 people

The number who actually took part.

Started

Apr 2025

Expected to finish

Aug 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Patients harboring mutations in ATF6 present with decreased retinal function Exclusion Criteria: * Patients who are minors * Patients who are pregnant

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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.

Achromatopsia Achromatopsia 7

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Columbia University Irving Medical Center

    New York, New York, 10032, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.