Safety and Efficacy Study of OpRegen for Treatment of Advanced Dry-Form Age-Related Macular Degeneration
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|ClinicalTrials.gov Identifier: NCT02286089|
Recruitment Status : Active, not recruiting
First Posted : November 7, 2014
Results First Posted : March 6, 2023
Last Update Posted : October 23, 2023
|Condition or disease||Intervention/treatment||Phase|
|Age-Related Macular Degeneration||Biological: OpRegen||Phase 1 Phase 2|
OpRegen® is a cell-based product composed of retinal pigment epithelial (RPE) cells, derived from human embryonic stem cells (hESC) and administered as a cell suspension either in ophthalmic Balanced Salt Solution Plus (BSS Plus) or in CryoStor® 5 (Thaw-and-Inject, TAI). This is a Phase I/IIa, dose-escalation, evaluating safety and tolerability of OpRegen transplantation to patients with progressive dry-AMD. The study includes also initial exploration of efficacy.
A total of approximately 24 subjects will be enrolled. The subjects should be 50 years of age and older, with non-neovascular (dry) AMD, who have funduscopic findings of GA in the macula, with absence of additional concomitant ocular disorders. The subjects will be divided into four cohorts, according to their best corrected visual acuity (BCVA) and administered OpRegen dose.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||24 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase I/IIa Dose Escalation Safety and Efficacy Study of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelium Cells Transplanted Subretinally in Patients With Advanced Dry-Form Age-Related Macular Degeneration (Geographic Atrophy)|
|Actual Study Start Date :||April 1, 2015|
|Actual Primary Completion Date :||December 31, 2021|
|Estimated Study Completion Date :||July 1, 2025|
Up to 12 legally blind subjects with best corrected visual acuity of 20/200 or less in first three cohorts and 12 subjects with best corrected visual acuity of 20/64 and 20/250 in fourth cohort
Targeted dose of 50,000 - 200,000 cells will be delivered into the subretinal space.
- Percentage of Participants With Treatment Emergent Adverse Events [ Time Frame: From study start till 12 months following last subject dosed, plus up to 90 days (up to approximately 6.5 years) ]An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. The AE's were graded using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v 3.0.
- Change From Baseline in Intraocular Pressure (IOP) [ Time Frame: Baseline, Month 12 ]
- Change From Baseline in Geographic Atrophy (GA) Lesion Area [ Time Frame: Baseline, Month 12 ]The GA lesion area was based on available Fundus Autofluorescence (FAF) imaging data by a central reading center.
- Change From Baseline in Visual Acuity [ Time Frame: Baseline, Month 12 ]Change from baseline in visual acuity was measured by retro illuminated ETDRS chart from 4 meters distance. Visual acuity was reported as the number of letters read correctly.
- Change From Baseline in National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) Quality of Life [ Time Frame: Baseline, Month 12 ]The NEI VFQ-25 is a 25 item patient-reported questionnaire. The composite score ranges from 0-100 with the higher score indicating better visual function.
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): NCT02286089
|Study Director:||Clinical Trials||Hoffmann-La Roche|