Study of Human Central Nervous System Stem Cells (HuCNS-SC) in Age-Related Macular Degeneration (AMD)
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|ClinicalTrials.gov Identifier: NCT01632527|
Recruitment Status : Completed
First Posted : July 3, 2012
Last Update Posted : September 14, 2015
|Condition or disease||Intervention/treatment||Phase|
|Age Related Macular Degeneration Macular Degeneration AMD||Drug: HuCNS-SC cells||Phase 1 Phase 2|
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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||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 Start Date :||June 2012|
|Actual Primary Completion Date :||June 2015|
|Actual Study Completion Date :||June 2015|
Drug: HuCNS-SC cells
HuCNS-SC cells transplantation directly into the subretinal space of one eye (Study eye) in a single transplant procedure.
Other Name: Human Central Nervous System Stem Cells
- 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.
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): NCT01632527
|United States, California|
|Retina-Vitreaous Associates Medical Group|
|Los Angeles, California, United States, 90211|
|Byers Eye Institute at Stanford, Stanford Hospital and Clinics|
|Palo Alto, California, United States, 94303|
|United States, New York|
|New York Eye and Ear Infirmary|
|New York, New York, United States, 10003|
|United States, Texas|
|Retina Research Institute of Texas|
|Abilene, Texas, United States, 79606|
|Retina Foundation of the Southwest|
|Dallas, Texas, United States, 75231|
|Study Director:||Stephen Huhn, M.D.||StemCells, Inc.|