Age-Related Macular DegenerationPhase 1/2NCT02286089
Safety and Efficacy Study of OpRegen for Treatment of Advanced Dry-Form Age-Related Macular Degeneration
A Phase 1/2 study testing OpRegen in people with Age-Related Macular Degeneration, run by Hoffmann-La Roche at 7 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Age-Related Macular Degeneration
- What is being tested?
- OpRegen
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 1 Apr 2015to 31 Jan 2031
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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);
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- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
7 sites in 2 countries.
7 sites in 2 countries
Israel4 sites
Hadassah Medical Center
Jerusalem, 9112001
Rabin Medical Center
Petah Tikva, 4941492
Kaplan Medical Center
Rehovot, 76100
Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906
United States3 sites
Cincinnati Eye Institute
Cincinnati, Ohio, 45242
Mid Atlantic Retina
Philadelphia, Pennsylvania, 19107
West Coast Retina Medical Group
San Francisco, California, 94109
About this study
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)
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.
Read the 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.
- Conditions
- Age-Related Macular Degeneration
- Being tested
- OpRegen
- Medical subject headings
- Macular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OpRegenBiological
Targeted dose of 50,000 - 200,000 cells will be delivered into the subretinal space.
| Study group | Role | What they receive |
|---|---|---|
| OpRegen | Experimental | 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 Receives: Biological: OpRegen |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From study start till 12 months following last subject dosed, plus up to 90 days (up to approximately 6.5 years)
Change From Baseline in Intraocular Pressure (IOP)
Measured: Baseline, Month 12
Secondary outcomes (3)
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.
Measured: Baseline, Month 12
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.
Measured: Baseline, Month 12
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.
Measured: Baseline, Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2014 (estimated)
- Study start1 Apr 2015
- Primary completion31 Dec 2021
- Study completion31 Jan 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Age-Related Macular DegenerationStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02286089
- Sponsor's protocol ID
- GR44172
- Status last verified
- Oct 2026
- First submitted
- 2 Nov 2014
- Last updated
- 23 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data\_sharing
Publications
Aweidah H, Matsevich C, Khaner H, Idelson M, Ejzenberg A, Reubinoff B, Banin E, Obolensky A. Survival of Neural Progenitors Derived from Human Embryonic Stem Cells Following Subretinal Transplantation in Rodents. J Ocul Pharmacol Ther. 2023 Jun;39(5):347-358. doi: 10.1089/jop.2022.0161. Epub 2023 May 4.
PubMed 37140896