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PBA for Color Blindness

Phase < 1
Waitlist Available
Led By Stephen Tsang, MD
Research Sponsored by Columbia University
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients harboring mutations in ATF6 present with decreased retinal function
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, 1 month, 3 months, 6 months post-pba use
Awards & highlights

Study Summary

This trial will study whether the drug glycerol phenylbutyrate can improve retinal function in patients with achromatopsia caused by ATF6 mutations.

Who is the study for?
This trial is for adults with achromatopsia, a form of color blindness due to ATF6 gene mutations. Participants must have decreased retinal function but cannot be minors or pregnant.Check my eligibility
What is being tested?
The study tests if glycerol phenylbutyrate (PBA), an FDA-approved drug, can improve vision in patients with color blindness caused by ATF6 mutations. Patients will take PBA three times daily and undergo regular eye exams.See study design
What are the potential side effects?
Potential side effects of PBA may include digestive issues like nausea or stomach pain, headaches, dizziness, fatigue, and skin rash. These are not specific to the trial but known from general use.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My vision is impaired due to a genetic mutation.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, 1 month, 3 months, 6 months post-pba use
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, 1 month, 3 months, 6 months post-pba use for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Changes in Full-field Electroretinogram (ffERG) X
Changes in best corrected visual acuity (BCVA)
Changes in color vision
+3 more
Secondary outcome measures
Changes in anterior segment
Changes in intraocular pressure
Changes observed in posterior segment (slit lamp and binocular fundus examination)

Side effects data

From 2015 Phase 4 trial • 80 Patients • NCT02117687
5%
Dry Eye
5%
Eye Discharge
5%
Eye Irritation
3%
Pericarditis
100%
80%
60%
40%
20%
0%
Study treatment Arm
VISMED® Multi
OPTIVE FUSION™

Trial Design

1Treatment groups
Experimental Treatment
Group I: PBA treatment of ATF6-/- AchromatopsiaExperimental Treatment1 Intervention
Patients will be monitored at the baseline visit, followed by a second and third visit that will be 1 and 3 months after the initial visit. Patients will complete a standard visual functioning questionnaire and undergo a complete ophthalmic evaluation at each visit. Other visual assessments will consist of color vision testing, contrast sensitivity, retinal imaging, and macular sensitivity testing using microperimetry. Full-field electroretinogram will also be performed at the baseline visit and after 1 and 3 months of PBA use. If improvement in retinal function is observed, an additional ophthalmic evaluation will be conducted after 6 months of PBA use. A blood draw will be performed at each visit to test for any indications of adverse effects from drug use.

Find a Location

Who is running the clinical trial?

Columbia UniversityLead Sponsor
1,423 Previous Clinical Trials
2,473,040 Total Patients Enrolled
Stephen Tsang, MDPrincipal InvestigatorColumbia University

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any openings in this trial that new participants can join?

"At present, the trial is not taking on any new candidates. It was initially posted in January 1st of 2023 and most recently updated on 4th April 2022. If you are looking for alternative trials to participate in, 7 studies related to achromatopsia have open recruitment and 3 medical interventions targeting PBA are currently onboarding participants."

Answered by AI

Is this the inaugural attempt at such a clinical trial?

"Presently, there are 3 ongoing studies regarding PBA that span 11 cities and 5 countries. The initial investigation into the drug was initiated in 2018 by Horizon Therapeutics, LLC as a Phase 4 drug approval trial with 18 patients contributing data. Since then, 12 more trials have been successfully concluded."

Answered by AI

How many participants is the clinical trial currently seeking?

"This research is no longer recruiting participants; the trial's original posting on 1/1/2023 was last modified on 4/4/2022. For those looking for alternative studies, 7 achromatopsia trials and 3 PBA trials are now open to new patients."

Answered by AI

Are there any precedential studies which have included PBA in their research?

"Currently, there are 3 studies involving PBA. Notably, none of these trials have reached Phase 3 status. These medical investigations are primarily located in Padua, Veneto but can also be found across 17 additional sites."

Answered by AI

Who else is applying?

What state do they live in?
Illinois
What site did they apply to?
Edward S. Harkness Eye Institute
What portion of applicants met pre-screening criteria?
Met criteria
How many prior treatments have patients received?
0

Why did patients apply to this trial?

I want to see a realistic cure for achromotopsia within my lifetime. As I have moved further into adulthood I have longed more and more to have better vision. I want to be involved in the steps that are taken to get there.
PatientReceived no prior treatments
~1 spots leftby Mar 2025