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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 : Recruiting
First Posted : November 7, 2014
Last Update Posted : November 13, 2019
Sponsor:
Collaborator:
CellCure Neurosciences Ltd.
Information provided by (Responsible Party):
Lineage Cell Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE November 2, 2014
First Posted Date  ICMJE November 7, 2014
Last Update Posted Date November 13, 2019
Study Start Date  ICMJE April 2015
Estimated Primary Completion Date December 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 14, 2018)
  • Incidence and frequency of treatment emergent adverse events [ Time Frame: 12 months post transplantation ]
    The AE's will be graded using NCI's CTCAE v 3.0
  • Treatment emergent changes of clinical and ophthalmological parameters [ Time Frame: 12 months post transplantation ]
    The parameters will be measured via different modalities, such as vital signs and ocular imaging and captured as adverse events
Original Primary Outcome Measures  ICMJE
 (submitted: November 5, 2014)
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.
Change History Complete list of historical versions of study NCT02286089 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 14, 2018)
  • Change in GA lesion area [ Time Frame: 12 months post transplantation ]
    Measurement of change in GA lesion area will be performed based on available imaging data by a central reading center.
  • Change in visual acuity [ Time Frame: 12 months post transplantation ]
    Change in visual acuity will be measured by ETDRS chart
  • Change in Quality of Life [ Time Frame: 12 months post transplantation ]
    Change in NEI VFQ-25 Quality of Life score will be measured from baseline
Original Secondary Outcome Measures  ICMJE
 (submitted: November 5, 2014)
  • Duration of graft survival [ Time Frame: 12 months post transplantation ]
  • Efficacy of the graft [ Time Frame: 12 months post transplantation ]
    Efficacy of the graft will be evaluated by decreased rate of GA progression and changes in visual acuity. Retinal sensitivity to light in engrafted regions, extent and depth of scotomata will be evaluated as well.
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
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
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 14, 2018)
24
Original Estimated Enrollment  ICMJE
 (submitted: November 5, 2014)
15
Estimated Study Completion Date  ICMJE December 2024
Estimated Primary Completion Date December 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Age 50 and older;
  2. Diagnosis of dry (non-neovascular) age related macular degeneration in both eyes;
  3. Funduscopic findings of dry AMD with progressive geographic atrophy in the macula;
  4. 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;
  5. Vision in the non-operated eye must be better than or equal to that in the operated eye;
  6. Subjects with sufficiently good health to allow participation in all study-related procedures and complete the study follow up period (medical records);
  7. Ability to undergo a vitreoretinal surgical procedure under monitored anesthesia care;
  8. Blood counts, blood chemistry, coagulation and urinalysis without abnormal significance;
  9. Negative for TB (cohort 4), HIV, HBC, and HCV, negative for CMV IgM and EBV IgM;
  10. Patients with 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;
  11. Willing to defer all future blood and tissue donation;
  12. Able to understand and willing to sign informed consent.

Exclusion Criteria:

  1. 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;
  2. History or presence of diabetic retinopathy, vascular occlusions, uveitis, Coat's disease, 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;
  3. History of retinal detachment repair in the study eye;
  4. Axial myopia greater than -6 diopters;
  5. 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;
  6. History of cognitive impairments or dementia;
  7. Contraindication for systemic immunosuppression;
  8. History of any condition other than AMD associated with choroidal neovascularization in the study eye (e.g. pathologic myopia or presumed ocular histoplasmosis);
  9. 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 patient to a significant degree or put the patient at special risk.
  10. Female; pregnancy or breastfeeding;
  11. Current participation in another clinical study. Past participation (within 6 months) in any clinical study of a drug administered systemically or to the eye.
  12. Currently receiving aspirin, aspirin containing products and/or any other coagulation modifying drugs which cannot be discontinued 7 days prior to surgery;
  13. History of cancer (other than a non-melanoma skin cancer). For cancers cured more than five years ago, enrollment is allowed with concurred documented approval of principal investigator and oncologist prior to enrollment.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 50 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Diana Angelini +1 510 7750472 dangelini@lineagecell.com
Contact: Avi Ben-Shabat, MD 972-73-326-3657 avi@cellcure.co.il
Listed Location Countries  ICMJE Israel,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02286089
Other Study ID Numbers  ICMJE CCN_CT02
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Lineage Cell Therapeutics, Inc.
Study Sponsor  ICMJE Lineage Cell Therapeutics, Inc.
Collaborators  ICMJE CellCure Neurosciences Ltd.
Investigators  ICMJE
Principal Investigator: Tareq Jaouni, MD Hadassah Ein Kerem University Hospital, Israel
Principal Investigator: Rita Ehrlich, MD Rabin Medical Center, Israel
Principal Investigator: Adiel Barak, MD, Prof. Tel Aviv Souraski Medical Center, Israel
Principal Investigator: Richard McDonald, MD West Coast Retina Medical Group, Inc, USA
Principal Investigator: David Boyer, MD Retina Vitreous Associates Medical Group, USA
Principal Investigator: Diana Do, MD, Prof. Byers Eye Institute, Stanford, USA
Principal Investigator: David Telander, MD Retinal Consultants Medical Group, USA
PRS Account Lineage Cell Therapeutics, Inc.
Verification Date November 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP