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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 : May 17, 2018
Sponsor:
Information provided by (Responsible Party):
Cell Cure Neurosciences Ltd.

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.

Condition or disease Intervention/treatment Phase
Age-related Macular Degeneration Biological: OpRegen Phase 1 Phase 2

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 in ophthalmic Balanced Salt Solution Plus (BSS Plus).

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 : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Study Start Date : April 2015
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
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
Biological: OpRegen
Targeted dose of 50,000 - 200,000 cells will be delivered into the subretinal space following vitrectomy procedure



Primary Outcome Measures :
  1. 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

  2. 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


Secondary Outcome Measures :
  1. 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.

  2. Change in visual acuity [ Time Frame: 12 months post transplantation ]
    Change in visual acuity will be measured by ETDRS chart

  3. 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



Information from the National Library of Medicine

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Ages Eligible for Study:   50 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02286089


Contacts
Contact: Maria Gurevich +972-733263657 maria@cellcure.co.il
Contact: Ghesal Razag +1 510 8714125 grazag@biotimeinc.com

Locations
United States, California
Retina Vitreous Associates Medical Group Recruiting
Los Angeles, California, United States, 900211
Contact: Janet Kurokouchi, B.Sc.    310-289-2478 ext 1243    JKurokouchi@laretina.com   
Contact: Gerard Aquino, B.Sc.    310-289-2478 ext 1225    GAquino@laretina.com   
Principal Investigator: David Boyer, MD         
Byers Eye Institute, Stanford School of Medicine Recruiting
Palo Alto, California, United States, 94303
Contact: Lisa Greer, MBA    650-725-9184    lgreer7@stanford.edu   
Principal Investigator: Diana Do, Prof.         
Retinal Consultants Medical Group Recruiting
Sacramento, California, United States, 95819
Contact: Erin Nickerman    916-453-5429    nickermane@retinalMD.com   
Contact: Whitney Lewis    916-453-5429    lewisw@retinalmd.com   
Principal Investigator: David Telander, MD         
West Coast Retina Medical Group, Inc Recruiting
San Francisco, California, United States, 94109
Contact: Kaitlin E. Miani, BA    415-972-4607    kmiani@westcoastretina.com   
Contact: C.         
Principal Investigator: Richard McDonald, MD         
Israel
Hadassah Ein Kerem University Hospital Recruiting
Jerusalem, Israel, 91120
Contact: Devora Marks Ohana    972-2-6776324    dryamdstudy@gmail.com   
Principal Investigator: Tareq Jaouni, MD         
Rabin Medical Center Recruiting
Petah Tikva, Israel
Contact: Vivi Dagan    972-3-9377199    eyeclinic@clalit.org.il   
Principal Investigator: Rita Ehrlich, MD         
Kaplan Medical Center Recruiting
Rehovot, Israel
Contact: Michal Scwartzberg    972-8-9441691    kaplaneye1@gmail.com   
Principal Investigator: Haia Morori-Katz, MD         
Tel Aviv Souraski Medical Center Recruiting
Tel Aviv, Israel, 91121
Contact: Sagit Bechor    03-6974361    sagitba@tlvmc.gov.il   
Principal Investigator: Adiel Barak, MD, Prof.         
Sponsors and Collaborators
Cell Cure Neurosciences Ltd.
Investigators
Study Director: Maria Gurevich Cell Cure Neurosciences Ltd.
Principal Investigator: Tareq Jaouni, MD Hadassah Ein Kerem University Hospital, Israel
Principal Investigator: Rita Ehrlich, MD Rabin Medical Center, Israel
Principal Investigator: Haia Morori-Katz, MD Kaplan 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