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.
Full Title of Study: “Phase I/II Study of the Safety and Preliminary Efficacy of Human Central Nervous System Stem Cells (HuCNS-SC) Subretinal Transplantation in Subjects With Geographic Atrophy of Age-Related Macular Degeneration”
- Study Type: Interventional
- Study Design
- Allocation: N/A
- Intervention Model: Single Group Assignment
- Primary Purpose: Treatment
- Masking: None (Open Label)
- Study Primary Completion Date: June 2015
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.
- Drug: HuCNS-SC cells
- HuCNS-SC cells transplantation directly into the subretinal space of one eye (Study eye) in a single transplant procedure.
Arms, Groups and Cohorts
- Experimental: HuCNS-SC
- HuCNS-SC cells
Clinical Trial Outcome Measures
- Number of subjects with adverse events
- Time Frame: One year following transplant surgery
- Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.
- Assessment of visual function changes from baseline
- Time Frame: 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.
Participating in This Clinical Trial
- Diagnosis of age-related macular degeneration with geographic atrophy (GA)
- Only patients with a specific degree and extent of GA will be eligible
- 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
- No prior or current choroidal neovascularization in either eye
- Must have adequate care-giver support and access to medical care in the local community
- Able to provide written informed consent prior to any study related procedures
- Agree to comply in good faith with all conditions of the study and to attend all required study visits
- Prior vitreal or retinal surgery
- Atrophic macular disease of any other cause
- Diabetic retinopathy or diabetic macular edema in either eye
- Previous organ, tissue or bone marrow transplantation
- Previous participation in a gene transfer or a cell transplant trial
- Autoimmune disease
- Allergy to tacrolimus, mycophenolate mofetil (MMF), scopolamine, Cyclogyl, Moxifloxacin, or Gatifloxacin
- Current or prior malignancy (or is on chemotherapy)
Gender Eligibility: All
Minimum Age: 50 Years
Maximum Age: N/A
Are Healthy Volunteers Accepted: No
- Lead Sponsor
- StemCells, Inc.
- Provider of Information About this Clinical Study
- Overall Official(s)
- Stephen Huhn, M.D., Study Director, StemCells, Inc.
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