Choroideremia Gene Therapy Clinical Trial
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|ClinicalTrials.gov Identifier: NCT02553135|
Recruitment Status : Completed
First Posted : September 17, 2015
Results First Posted : July 29, 2019
Last Update Posted : July 29, 2019
|Condition or disease||Intervention/treatment||Phase|
|Choroideremia||Biological: Injection of AAV2-REP1 (10e11 vg)||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open Label Phase 2 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)|
|Actual Study Start Date :||September 2015|
|Actual Primary Completion Date :||February 2018|
|Actual Study Completion Date :||February 2018|
Experimental: Injection of AAV2-REP1
Injection of AAV-REP1, 1.00x10e11 vg, subretinal injection of total volume of 100 μL.
Biological: Injection of AAV2-REP1 (10e11 vg)
Single Group: single arm study
- Change in Best Corrected Visual Acuity From Baseline [ Time Frame: Baseline, 24 Months ]Patients will have an assessment of visual acuity using the Early Treatment of Diabetic Retinopathy Study (ETDRS) vision charts in both eyes. Low Luminance BCVA was measured by placing a 2.0 log unit neutral density filter over the best correction for that eye and having the participant read the normally illuminated ETDRS chart and was reported as the number of letters read correctly (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). The study eye was defined as the eye that met inclusion/exclusion criteria with the worst standard BCVA. If the BCVA in both eyes was similar the Patient determines the worse eye be selected as the study eye. A positive change from baseline indicates an improvement and a negative change from baseline indicates a worsening.
- Change in Retinal Macular Autofluorescence From Baseline [ Time Frame: 12 and 24 months ]Measured in mm^2 by Fundus Autofluorescence and shows changes in the integrity and metabolism of retinal cells. A negative change from baseline indicates a decrease in size of the retained retina (worsening; disease progression).
- Changes in Microperimetry From Baseline [ Time Frame: Baseline to 24 months ]Microperimetry assessments. A negative change from baseline indicates disease worsening.
- Number of Participants Who Experience an Adverse Event [ Time Frame: 24 months ]Adverse events during treatment and follow-up period
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02553135
|United States, Florida|
|Bascom Palmer Eye Institute, University of Miami|
|Miami, Florida, United States, 33136|
|Principal Investigator:||BYRON LAM, MD||UNIVERSITY OF MIAMI, BASCOM PALMER EYE INSTITUTE|