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 : April 17, 2024
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Tracking Information | |||||
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First Submitted Date ICMJE | November 2, 2014 | ||||
First Posted Date ICMJE | November 7, 2014 | ||||
Results First Submitted Date ICMJE | December 19, 2022 | ||||
Results First Posted Date ICMJE | March 6, 2023 | ||||
Last Update Posted Date | April 17, 2024 | ||||
Actual Study Start Date ICMJE | April 1, 2015 | ||||
Actual Primary Completion Date | December 31, 2021 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
Safety and tolerability of OpRegen [ Time Frame: 12 months post transplantation ] Safety and tolerability of the graft and of the surgical procedure will be evaluated by the absence of adverse events. Severity of the adverse events will be graded according to the National Cancer Institute (NCI) grading system.
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Change History | |||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Safety and Efficacy Study of OpRegen for Treatment of Advanced Dry-Form Age-Related Macular Degeneration | ||||
Official Title ICMJE | 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) | ||||
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. |
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Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 1 Phase 2 |
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Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Age-Related Macular Degeneration | ||||
Intervention ICMJE | Biological: OpRegen
Targeted dose of 50,000 - 200,000 cells will be delivered into the subretinal space.
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Study Arms ICMJE | Experimental: OpRegen
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
Intervention: Biological: OpRegen
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Active, not recruiting | ||||
Actual Enrollment ICMJE |
24 | ||||
Original Estimated Enrollment ICMJE |
15 | ||||
Estimated Study Completion Date ICMJE | July 1, 2025 | ||||
Actual Primary Completion Date | December 31, 2021 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 50 Years and older (Adult, Older Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | Not Provided | ||||
Removed Location Countries | Israel, United States | ||||
Administrative Information | |||||
NCT Number ICMJE | NCT02286089 | ||||
Other Study ID Numbers ICMJE | GR44172 | ||||
Has Data Monitoring Committee | Not Provided | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE | Not Provided | ||||
Current Responsible Party | Hoffmann-La Roche | ||||
Original Responsible Party | Lineage Cell Therapeutics, Inc. | ||||
Current Study Sponsor ICMJE | Hoffmann-La Roche | ||||
Original Study Sponsor ICMJE | Lineage Cell Therapeutics, Inc. | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE |
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PRS Account | Hoffmann-La Roche | ||||
Verification Date | April 2024 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |