Age-Related Macular Degeneration
Conditions
Brief summary
The main objective of the study is evaluation of the safety and tolerability of OpRegen - Human embryonic stem cell-derived retinal pigment epithelial (RPE) cells. The study will also include initial exploration of the ability of transplanted OpRegen cells to engraft, survive, and moderate disease progression.
Detailed description
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.
Interventions
BIOLOGICALOpRegen
Targeted dose of 50,000 - 200,000 cells will be delivered into the subretinal space.
Sponsors
Hoffmann-La Roche
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
50 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Age 50 and older; * Diagnosis of dry (non-neovascular) age related macular degeneration in both eyes; * Funduscopic findings of dry age-related macular degeneration (AMD) with progressive geographic atrophy in the macula; * Best corrected central visual acuity equal or less than 20/200 in cohorts 1-3 and 20/64-20/250 in cohort 4 in the study eye by ETDRS vision testing; * Vision in the non-operated eye must be better than or equal to that in the operated eye; * Subjects with sufficiently good health to allow participation in all study-related procedures and complete the study follow up period (based on medical records); * Ability to undergo a vitreoretinal surgical procedure under monitored anesthesia care; * Blood counts, blood chemistry, coagulation and urinalysis without abnormal significance; * Negative for tuberculosis (TB) (cohort 4), human immunodeficiency virus (HIV), hepatitis B (HBC), and hepatitis C virus (HCV), negative for cytomegalovirus (CMV) Immunoglobulin (IgM) and Epstein-Barr Virus (EBV) IgM or asymptomatic in the opinion of the investigator (cohort 4); * No history of malignancy (other than a non-melanoma skin cancer). For cancers in remission for more then 5 years enrollment is allowed with concurred documented approval of principal investigator and oncologist prior to enrollment; * Willing to defer all future blood and tissue donation; * Able to understand study procedures and willing to sign informed consent.
Exclusion criteria
* Evidence of neovascular AMD by history, as well as by clinical exam, fluorescein angiography (FA), or ocular coherence tomography (OCT) at baseline in either eye; * History or presence of diabetic retinopathy, vascular occlusions, uveitis, Coat's disease, uncontrolled glaucoma, cataract or media opacity preventing posterior pole visualization or any significant ocular disease other than AMD that has compromised or could compromise vision in the study eye and confound analysis of the primary outcome; * History of retinal detachment repair in the study eye; * Axial myopia greater than -6 diopters; * At least 2 months following cataract removal in the study eye and Yttrium Aluminum Garnet (YAG) laser capsulotomy in the study eye in the past 4 weeks and any other ocular surgery in the study eye in the past 3 months prior to implantation; * History of cognitive impairments or dementia; * Contraindication for systemic immunosuppression; * History of any condition other than AMD associated with choroidal neovascularization in the study eye (e.g. pathologic myopia or presumed ocular histoplasmosis); * Any type of systemic disease or its treatment, in the opinion of the Investigator, including any medical condition (controlled or uncontrolled) that could be expected to progress, recur, or change to such an extent that it may bias the assessment of the clinical status of the participant to a significant degree or put the patient at special risk. * Pregnancy or breastfeeding; * Current participation in another clinical study. Past participation (within 6 months) in any clinical study of a drug administered systemically or to the eye. * Currently receiving aspirin, aspirin containing products and/or any other coagulation modifying drugs which cannot be discontinued 7 days prior to surgery; * History of cancer (other than a non-melanoma skin cancer). For cancers in remission more than five years ago, enrollment is allowed with concurred documented approval of principal investigator and oncologist prior to enrollment.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Treatment Emergent Adverse Events | 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) | Baseline, Month 12 | — |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Geographic Atrophy (GA) Lesion Area | 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 | 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 | 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. |
Countries
Israel, United States
Contacts
STUDY_DIRECTORClinical Trials
Hoffmann-La Roche
Participant flow
Pre-assignment details
The enrollment into the study was staggered with 4 week intervals between the first 3 cohorts to allow for data safety monitory board (DSMB) review. Accumulated data of participants was reviewed by the DSMB before continuation to Cohort 4. All four cohorts were then followed in parallel.
Participants by arm
| Arm | Count |
|---|---|
| Cohort 1 Participants in cohort 1 with Best Corrected Visual Acuity (BCVA) of 20/200 or less received a single subretinal delivery of the low dose of OpRegen on Day 0. | 3 |
| Cohort 2 Participants in cohort 2 with BCVA of 20/200 or less received a single subretinal delivery of the high dose of OpRegen on Day 0. | 3 |
| Cohort 3 Additional participants in cohort 3 with BCVA of 20/200 or less received a single subretinal delivery of the high dose of OpRegen on Day 0. | 6 |
| Cohort 4 Participants in cohort 4 with BCVA of 20/64 to 20/250 received a single subretinal delivery of the high dose of OpRegen on Day 0. | 12 |
| Total | 24 |
Baseline characteristics
| Characteristic | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Total |
|---|---|---|---|---|---|
| Age, Continuous | 77.4 Years STANDARD_DEVIATION 2.7 | 74.4 Years STANDARD_DEVIATION 8.7 | 80.4 Years STANDARD_DEVIATION 9.9 | 78.1 Years STANDARD_DEVIATION 8.2 | 76.9 Years STANDARD_DEVIATION 8 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 3 Participants | 3 Participants | 6 Participants | 12 Participants | 24 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 3 Participants | 3 Participants | 6 Participants | 12 Participants | 24 Participants |
| Sex: Female, Male Female | 2 Participants | 3 Participants | 2 Participants | 6 Participants | 13 Participants |
| Sex: Female, Male Male | 1 Participants | 0 Participants | 4 Participants | 6 Participants | 11 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 3 | 0 / 3 | 0 / 6 | 0 / 12 |
| other Total, other adverse events | 3 / 3 | 3 / 3 | 6 / 6 | 12 / 12 |
| serious Total, serious adverse events | 1 / 3 | 2 / 3 | 2 / 6 | 4 / 12 |
Outcome results
Primary
Change From Baseline in Intraocular Pressure (IOP)
Time frame: Baseline, Month 12
Population: Safety Population included all participants who received OpRegen in the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort 1 | Change From Baseline in Intraocular Pressure (IOP) | Baseline | 13.3 mmHg | Standard Error 2.3 |
| Cohort 1 | Change From Baseline in Intraocular Pressure (IOP) | Change from Baseline at Month 12 | 1.0 mmHg | Standard Error 2.6 |
| Cohort 2 | Change From Baseline in Intraocular Pressure (IOP) | Change from Baseline at Month 12 | 1.0 mmHg | Standard Error 1.7 |
| Cohort 2 | Change From Baseline in Intraocular Pressure (IOP) | Baseline | 14.0 mmHg | Standard Error 0 |
| Cohort 3 | Change From Baseline in Intraocular Pressure (IOP) | Baseline | 13.7 mmHg | Standard Error 3.7 |
| Cohort 3 | Change From Baseline in Intraocular Pressure (IOP) | Change from Baseline at Month 12 | -0.4 mmHg | Standard Error 2.5 |
| Cohort 4 | Change From Baseline in Intraocular Pressure (IOP) | Change from Baseline at Month 12 | -0.6 mmHg | Standard Error 1.4 |
| Cohort 4 | Change From Baseline in Intraocular Pressure (IOP) | Baseline | 14.6 mmHg | Standard Error 3.1 |
Primary
Percentage of Participants With Treatment Emergent Adverse Events
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.
Time frame: From study start till 12 months following last subject dosed, plus up to 90 days (up to approximately 6.5 years)
Population: Safety Population included all participants who received OpRegen in the study.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Investigations | 100 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Injury, poisoning and procedural complications | 66.7 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Eye disorders | 100 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Skin and subcutaneous tissue disorders | 33.3 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Infections and infestations | 100 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | General disorders and administration site conditions | 66.7 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Gastrointestinal disorders | 66.7 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Surgical and medical procedures | 100 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Blood and lymphatic system disorders | 33.3 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Respiratory, thoracic and mediastinal disorders | 66.7 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Psychiatric disorders | 0 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Nervous system disorders | 0 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Cardiac disorders | 33.3 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Renal and urinary disorders | 0 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 0 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Musculoskeletal and connective tissue disorders | 66.7 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Ear and labyrinth disorders | 66.7 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Product issues | 66.7 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Metabolism and nutrition disorders | 0 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Endocrine disorders | 0 percentage of participants |
| Cohort 1 | Percentage of Participants With Treatment Emergent Adverse Events | Vascular disorders | 0 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Surgical and medical procedures | 66.7 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Blood and lymphatic system disorders | 33.3 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Product issues | 0 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Cardiac disorders | 0 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Ear and labyrinth disorders | 33.3 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Endocrine disorders | 0 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Eye disorders | 100 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Gastrointestinal disorders | 66.7 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | General disorders and administration site conditions | 100 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Infections and infestations | 66.7 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Injury, poisoning and procedural complications | 100 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Investigations | 66.7 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Metabolism and nutrition disorders | 0 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Musculoskeletal and connective tissue disorders | 66.7 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 33.3 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Nervous system disorders | 33.3 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Psychiatric disorders | 0 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Skin and subcutaneous tissue disorders | 33.3 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Vascular disorders | 0 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Renal and urinary disorders | 0 percentage of participants |
| Cohort 2 | Percentage of Participants With Treatment Emergent Adverse Events | Respiratory, thoracic and mediastinal disorders | 66.7 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Skin and subcutaneous tissue disorders | 33.3 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Surgical and medical procedures | 0 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Respiratory, thoracic and mediastinal disorders | 16.7 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Nervous system disorders | 50.0 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 50.0 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Metabolism and nutrition disorders | 16.7 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | General disorders and administration site conditions | 50.0 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Renal and urinary disorders | 33.3 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Gastrointestinal disorders | 0 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Musculoskeletal and connective tissue disorders | 0 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Blood and lymphatic system disorders | 0 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Vascular disorders | 16.7 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Eye disorders | 100 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Ear and labyrinth disorders | 16.7 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Infections and infestations | 50.0 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Investigations | 33.3 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Psychiatric disorders | 0 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Injury, poisoning and procedural complications | 50.0 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Cardiac disorders | 0 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Endocrine disorders | 0 percentage of participants |
| Cohort 3 | Percentage of Participants With Treatment Emergent Adverse Events | Product issues | 0 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Endocrine disorders | 8.3 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Investigations | 75.0 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Product issues | 0 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Metabolism and nutrition disorders | 33.3 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Cardiac disorders | 8.3 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Musculoskeletal and connective tissue disorders | 33.3 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 25.0 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Blood and lymphatic system disorders | 8.3 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Nervous system disorders | 33.3 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Renal and urinary disorders | 0 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Psychiatric disorders | 8.3 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Surgical and medical procedures | 16.7 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Ear and labyrinth disorders | 8.3 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Skin and subcutaneous tissue disorders | 8.3 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Infections and infestations | 33.3 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Respiratory, thoracic and mediastinal disorders | 0 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Gastrointestinal disorders | 33.3 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Eye disorders | 100 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Vascular disorders | 16.7 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | General disorders and administration site conditions | 16.7 percentage of participants |
| Cohort 4 | Percentage of Participants With Treatment Emergent Adverse Events | Injury, poisoning and procedural complications | 16.7 percentage of participants |
Secondary
Change From Baseline in Geographic Atrophy (GA) Lesion Area
The GA lesion area was based on available Fundus Autofluorescence (FAF) imaging data by a central reading center.
Time frame: Baseline, Month 12
Population: Efficacy Population included all participants who received OpRegen in the study and for whom imaging data were available.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort 1 | Change From Baseline in Geographic Atrophy (GA) Lesion Area | Change from Baseline at Month 12 | 2.3 mm^2 | Standard Deviation 1.9 |
| Cohort 1 | Change From Baseline in Geographic Atrophy (GA) Lesion Area | Baseline | 13.5 mm^2 | Standard Deviation 6.4 |
| Cohort 2 | Change From Baseline in Geographic Atrophy (GA) Lesion Area | Change from Baseline at Month 12 | 1.6 mm^2 | — |
| Cohort 2 | Change From Baseline in Geographic Atrophy (GA) Lesion Area | Baseline | 18.5 mm^2 | Standard Deviation 10.6 |
| Cohort 3 | Change From Baseline in Geographic Atrophy (GA) Lesion Area | Baseline | 9.4 mm^2 | Standard Deviation 2 |
| Cohort 3 | Change From Baseline in Geographic Atrophy (GA) Lesion Area | Change from Baseline at Month 12 | 2.5 mm^2 | Standard Deviation 4 |
| Cohort 4 | Change From Baseline in Geographic Atrophy (GA) Lesion Area | Change from Baseline at Month 12 | 1.8 mm^2 | Standard Deviation 0.8 |
| Cohort 4 | Change From Baseline in Geographic Atrophy (GA) Lesion Area | Baseline | 7.4 mm^2 | Standard Deviation 2.9 |
Secondary
Change From Baseline in National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) Quality of Life
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.
Time frame: Baseline, Month 12
Population: Efficacy Population included all participants who received OpRegen in the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort 1 | Change From Baseline in National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) Quality of Life | Baseline | 50.3 score on a scale | Standard Deviation 15.3 |
| Cohort 1 | Change From Baseline in National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) Quality of Life | Change from Baseline at Month 12 | 5.1 score on a scale | Standard Deviation 10.8 |
| Cohort 2 | Change From Baseline in National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) Quality of Life | Baseline | 42.9 score on a scale | Standard Deviation 6 |
| Cohort 2 | Change From Baseline in National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) Quality of Life | Change from Baseline at Month 12 | -5.7 score on a scale | Standard Deviation 17.6 |
| Cohort 3 | Change From Baseline in National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) Quality of Life | Baseline | 46.9 score on a scale | Standard Deviation 18.8 |
| Cohort 3 | Change From Baseline in National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) Quality of Life | Change from Baseline at Month 12 | 3.1 score on a scale | Standard Deviation 18.2 |
| Cohort 4 | Change From Baseline in National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) Quality of Life | Change from Baseline at Month 12 | 3.7 score on a scale | Standard Deviation 11.4 |
| Cohort 4 | Change From Baseline in National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) Quality of Life | Baseline | 62.2 score on a scale | Standard Deviation 13.8 |
Secondary
Change From Baseline in Visual Acuity
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.
Time frame: Baseline, Month 12
Population: Efficacy Population included all participants who received OpRegen in the study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Cohort 1 | Change From Baseline in Visual Acuity | Baseline | 20.0 number of letters | Standard Deviation 18 |
| Cohort 1 | Change From Baseline in Visual Acuity | Change from Baseline at Month 12 | 15.7 number of letters | Standard Deviation 34.2 |
| Cohort 2 | Change From Baseline in Visual Acuity | Change from Baseline at Month 12 | 3.0 number of letters | Standard Deviation 8.7 |
| Cohort 2 | Change From Baseline in Visual Acuity | Baseline | 14.3 number of letters | Standard Deviation 7.2 |
| Cohort 3 | Change From Baseline in Visual Acuity | Baseline | 29.8 number of letters | Standard Deviation 6.8 |
| Cohort 3 | Change From Baseline in Visual Acuity | Change from Baseline at Month 12 | -1.4 number of letters | Standard Deviation 17.8 |
| Cohort 4 | Change From Baseline in Visual Acuity | Baseline | 44.8 number of letters | Standard Deviation 7.5 |
| Cohort 4 | Change From Baseline in Visual Acuity | Change from Baseline at Month 12 | 7.6 number of letters | Standard Deviation 8.7 |