PBA Use for Treatment of ATF6-/- Patients

Phase 1

Not yet recruiting

• Conditions: ACHROMATOPSIA 7; Achromatopsia

• Registration Number: NCT04041232
• Lead Sponsor: Columbia University

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2019-7-31
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: Not yet recruiting
• Sex: All
• Target Recruitment: 2

Inclusion Criteria

Inclusion Criteria:<br><br> - Patients harboring mutations in ATF6 present with decreased retinal function<br><br>Exclusion Criteria:<br><br> - Patients who are minors<br><br> - Patients who are pregnant

Exclusion Criteria

Not provided

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Changes in best corrected visual acuity (BCVA);Changes in contrast sensitivity;Changes in color vision;Changes in macular sensitivity;Changes in retinal imaging;Changes in Full-field Electroretinogram (ffERG) X

Secondary Outcome Measures

Name	Time	Method
Changes in intraocular pressure;Changes in anterior segment;Changes observed in posterior segment (slit lamp and binocular fundus examination)