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PBA in ATF6-/- Achromatopsia
NCT04041232Phase 1Suspended
- Sponsor
- Columbia University
- Start
- Apr 2025
- Primary completion
- Aug 2027
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of glycerol phenylbutyrate in achromatopsia (ACHM), sponsored by Columbia University. Primary endpoints: Best Corrected Visual Acuity at 6 months, Contrast Sensitivity at 6 months and 4 more. Started Apr 2025; status: suspended.
02
Drugs
glycerol phenylbutyrate
03
Conditions
achromatopsia (ACHM)
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