Study of Human Central Nervous System Stem Cells (HuCNS-SC) in Age-Related Macular Degeneration (AMD)
Study Details
Study Description
Brief Summary
The purpose of this Phase I/II study is to investigate the safety and preliminary efficacy of unilateral subretinal transplantation of HuCNS-SC cells in subjects with geographic atrophy secondary to age-related macular degeneration.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1/Phase 2 |
Detailed Description
This study is an open-label dose-escalation investigation of the safety and preliminary efficacy of unilateral subretinal transplantation of HuCNS-SC cells in subjects with Geographic Atrophy secondary to Age-Related Macular Degeneration (AMD). Subjects will be enrolled based on specific inclusion/exclusion criteria and evaluated at regular post transplant intervals. The investigation will be divided into two sequential cohorts. Subjects will be enrolled into each cohort based on best-corrected visual acuity (BCVA) visual acuity as determined by the Electronic Early Treatment Diabetic Retinopathy Study (E-ETDRS) acuity test. BCVA of less than or equal to 20/400 in the Study Eye will be enrolled in Cohort I. Subjects with BCVA of 20/320 to 20/100 in the Study Eye will be enrolled in Cohort II. Cohort I will consist of four subjects who will undergo transplant with 200,000 cells followed by four subjects who will undergo transplant with 1 million cells. Cohort II will consist of 7 subjects who will undergo transplant with 1 million cells. An independent Data Monitoring Committee (DMC) will review accruing safety data for all subjects.
HuCNS-SC cells will be transplanted by a board-certified vitreoretinal surgeon. The transplantation will be conducted in the eye with the inferior best-corrected visual acuity (BCVA) (i.e., the Study Eye). Only the Study Eye will undergo transplantation. The HuCNS-SC cells will be administered into the subretinal space through a standard surgical approach.
Immunosuppressive agents will be administered orally to all subjects for a period of three months after surgery.
Subjects will be monitored frequently for a total of one year after HuCNS-SC cell transplantation. An additional four years of monitoring will be conducted in a separate follow-up study that will commence with the termination visit of the Phase I/II investigation. The follow-up study will be conducted as a separate investigation.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: HuCNS-SC HuCNS-SC cells |
Drug: HuCNS-SC cells
HuCNS-SC cells transplantation directly into the subretinal space of one eye (Study eye) in a single transplant procedure.
Other Names:
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Outcome Measures
Primary Outcome Measures
- Number of subjects with adverse events [One year following transplant surgery]
Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.
Secondary Outcome Measures
- Assessment of visual function changes from baseline [At frequent intervals for one year following transplantation]
Assessments will include BCVA by the E-ETDRS acuity test, fluorescein angiography and fundus photography, spectral domain ocular coherence tomography (OCT), microperimetry, multifocal electroretinography, contrast sensitivity, and a standardized questionnaire of visual function.
Eligibility Criteria
Criteria
Inclusion Criteria:
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Diagnosis of age-related macular degeneration with geographic atrophy (GA)
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Only patients with a specific degree and extent of GA will be eligible
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Subjects with best-corrected visual acuity (BCVA) of less than or equal to 20/400 in the Study Eye will be enrolled in Cohort I. Subjects with BCVA of 20/320 to 20/100 in the Study Eye will be enrolled in Cohort II
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No prior or current choroidal neovascularization in either eye
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Must have adequate care-giver support and access to medical care in the local community
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Able to provide written informed consent prior to any study related procedures
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Agree to comply in good faith with all conditions of the study and to attend all required study visits
Exclusion Criteria:
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Prior vitreal or retinal surgery
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Glaucoma
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Atrophic macular disease of any other cause
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Diabetic retinopathy or diabetic macular edema in either eye
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Previous organ, tissue or bone marrow transplantation
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Previous participation in a gene transfer or a cell transplant trial
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Autoimmune disease
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Allergy to tacrolimus, mycophenolate mofetil (MMF), scopolamine, Cyclogyl, Moxifloxacin, or Gatifloxacin
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Current or prior malignancy (or is on chemotherapy)
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Retina-Vitreaous Associates Medical Group | Los Angeles | California | United States | 90211 |
| 2 | Byers Eye Institute at Stanford, Stanford Hospital and Clinics | Palo Alto | California | United States | 94303 |
| 3 | New York Eye and Ear Infirmary | New York | New York | United States | 10003 |
| 4 | Retina Research Institute of Texas | Abilene | Texas | United States | 79606 |
| 5 | Retina Foundation of the Southwest | Dallas | Texas | United States | 75231 |
Sponsors and Collaborators
- StemCells, Inc.
Investigators
- Study Director: Stephen Huhn, M.D., StemCells, Inc.
Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
None provided.- StemCells CL-N01-AMD