Status:
SUSPENDED
PBA Use for Treatment of ATF6-/- Patients
Lead Sponsor:
Columbia University
Conditions:
ACHROMATOPSIA 7
Achromatopsia
Eligibility:
All Genders
18+ years
Phase:
EARLY_PHASE1
Brief Summary
Some patients with achromatopsia, an inherited disorder characterized by partial or complete loss of color vision, carry mutations in ATF6. ATF6 is a gene that is responsible for coding a protein that...
Detailed Description
ATF6 has been described as an endoplasmic reticulum (ER) stress-regulated transmembrane protein that activates the transcription of molecular chaperones in response to ER stress. Patients harboring mu...
Eligibility Criteria
Inclusion
- Patients harboring mutations in ATF6 present with decreased retinal function
Exclusion
- Patients who are minors
- Patients who are pregnant
Key Trial Info
Start Date :
April 8 2025
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 1 2027
Estimated Enrollment :
2 Patients enrolled
Trial Details
Trial ID
NCT04041232
Start Date
April 8 2025
End Date
August 1 2027
Last Update
September 12 2025
Active Locations (1)
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1
Columbia University Irving Medical Center
New York, New York, United States, 10032