Study of Human Central Nervous System Stem Cells (HuCNS-SC) in Age-Related Macular Degeneration (AMD)
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| First Received Date ICMJE | June 21, 2012 | ||||||||
| Last Updated Date | May 14, 2013 | ||||||||
| Start Date ICMJE | June 2012 | ||||||||
| Estimated Primary Completion Date | December 2013 (final data collection date for primary outcome measure) | ||||||||
| Current Primary Outcome Measures ICMJE |
Number of subjects with adverse events [ Time Frame: One year following transplant surgery ] [ Designated as safety issue: Yes ] Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period. |
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| Original Primary Outcome Measures ICMJE | Same as current | ||||||||
| Change History | Complete list of historical versions of study NCT01632527 on ClinicalTrials.gov Archive Site | ||||||||
| Current Secondary Outcome Measures ICMJE |
Assessment of visual function changes from baseline [ Time Frame: At frequent intervals for one year following transplantation ] [ Designated as safety issue: No ] 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. |
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| Original Secondary Outcome Measures ICMJE | Same as current | ||||||||
| Current Other Outcome Measures ICMJE | Not Provided | ||||||||
| Original Other Outcome Measures ICMJE | Not Provided | ||||||||
| Descriptive Information | |||||||||
| Brief Title ICMJE | Study of Human Central Nervous System Stem Cells (HuCNS-SC) in Age-Related Macular Degeneration (AMD) | ||||||||
| Official Title ICMJE | 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 | ||||||||
| 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. |
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| 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 20/200 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 8 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. |
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| Study Type ICMJE | Interventional | ||||||||
| Study Phase | Phase 1 Phase 2 |
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| Study Design ICMJE | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
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| Condition ICMJE |
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| Intervention ICMJE | 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 |
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| Study Arm (s) | Experimental: HuCNS-SC
HuCNS-SC cells
Intervention: Drug: HuCNS-SC cells |
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| Publications * | Not Provided | ||||||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||||||
| Recruitment Status ICMJE | Recruiting | ||||||||
| Estimated Enrollment ICMJE | 16 | ||||||||
| Estimated Completion Date | March 2014 | ||||||||
| Estimated Primary Completion Date | December 2013 (final data collection date for primary outcome measure) | ||||||||
| Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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| Gender | Both | ||||||||
| Ages | 50 Years and older | ||||||||
| Accepts Healthy Volunteers | No | ||||||||
| Contacts ICMJE |
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| Location Countries ICMJE | United States | ||||||||
| Administrative Information | |||||||||
| NCT Number ICMJE | NCT01632527 | ||||||||
| Other Study ID Numbers ICMJE | StemCells CL-N01-AMD | ||||||||
| Has Data Monitoring Committee | Yes | ||||||||
| Responsible Party | StemCells, Inc. | ||||||||
| Study Sponsor ICMJE | StemCells, Inc. | ||||||||
| Collaborators ICMJE | Not Provided | ||||||||
| Investigators ICMJE |
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| Information Provided By | StemCells, Inc. | ||||||||
| Verification Date | May 2013 | ||||||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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