Safety and Efficacy Study of OpRegen for Treatment of Advanced Dry-Form Age-Related Macular Degeneration

NCT02286089 | Active Not Recruiting Phase 1 Results FDA Drug

• 1 other identifier: GR44172
• Study Type: interventional
• Target: 24
• Locations: 2 countries
• Sites: 7

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.

Conditions

Age-Related Macular Degeneration

Outcome Measures

Primary Outcomes (2)

• 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.

  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)

  Baseline, Month 12

Secondary Outcomes (3)

• Change From Baseline in Geographic Atrophy (GA) Lesion Area

  Baseline, Month 12

• Change From Baseline in Visual Acuity

  Baseline, Month 12

• Change From Baseline in National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) Quality of Life

  Baseline, Month 12

Study Arms (1)

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

Biological: OpRegen

Interventions

OpRegen BIOLOGICAL

Targeted dose of 50,000 - 200,000 cells will be delivered into the subretinal space.

OpRegen

Eligibility Criteria

• Age: 50 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (7)

West Coast Retina Medical Group

San Francisco, California, 94109, United States

Location

Cincinnati Eye Institute

Cincinnati, Ohio, 45242, United States

Location

Mid Atlantic Retina

Philadelphia, Pennsylvania, 19107, United States

Location

Hadassah Medical Center

Jerusalem, 9112001, Israel

Location

Rabin Medical Center

Petah Tikva, 4941492, Israel

Location

Kaplan Medical Center

Rehovot, 76100, Israel

Location

Tel Aviv Sourasky Medical Center

Tel Aviv, 6423906, Israel

Location

Related Publications (1)

• 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.

  PMID: 37140896 DERIVED

MeSH Terms

Conditions

Macular Degeneration

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases

Results Point of Contact

• Title: Medical Communications
• Organization: Hoffmann-La Roche

genentech@druginfo.com

800 821-8590

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 2, 2014
• First Posted: November 7, 2014
• Study Start: April 1, 2015
• Primary Completion: December 31, 2021
• Study Completion (Estimated): January 31, 2031
• Last Updated: April 27, 2026
• Results First Posted: March 6, 2023

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

US (3); IL (4)