Study of Human Central Nervous System Stem Cells (HuCNS-SC) in Age-Related Macular Degeneration (AMD)
Primary Purpose
Age Related Macular Degeneration, Macular Degeneration, AMD
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
HuCNS-SC cells
Sponsored by
About this trial
This is an interventional treatment trial for Age Related Macular Degeneration focused on measuring Age-Related Macular Degeneration, Dry AMD with GA, Geographic Atrophy of Age-Related Macular Degeneration, Stem Cell Transplantation, Stem Cell transplant, Stem cells for AMD, Human neural stem cells, multipotent stem cells, Macular Degeneration, Dry AMD, Human central nervous system stemcells, dry age-related macular degeneration
Eligibility Criteria
Inclusion Criteria:
- 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
Exclusion Criteria:
- Prior vitreal or retinal surgery
- Glaucoma
- 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)
Sites / Locations
- Retina-Vitreaous Associates Medical Group
- Byers Eye Institute at Stanford, Stanford Hospital and Clinics
- New York Eye and Ear Infirmary
- Retina Research Institute of Texas
- Retina Foundation of the Southwest
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
HuCNS-SC
Arm Description
HuCNS-SC cells
Outcomes
Primary Outcome Measures
Number of subjects with adverse events
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
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01632527
Brief Title
Study of Human Central Nervous System Stem Cells (HuCNS-SC) in Age-Related Macular Degeneration (AMD)
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 Type
Interventional
2. Study Status
Record Verification Date
September 2015
Overall Recruitment Status
Completed
Study Start Date
June 2012 (undefined)
Primary Completion Date
June 2015 (Actual)
Study Completion Date
June 2015 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
StemCells, Inc.
4. Oversight
Data Monitoring Committee
Yes
5. 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Age Related Macular Degeneration, Macular Degeneration, AMD
Keywords
Age-Related Macular Degeneration, Dry AMD with GA, Geographic Atrophy of Age-Related Macular Degeneration, Stem Cell Transplantation, Stem Cell transplant, Stem cells for AMD, Human neural stem cells, multipotent stem cells, Macular Degeneration, Dry AMD, Human central nervous system stemcells, dry age-related macular degeneration
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)
8. Arms, Groups, and Interventions
Arm Title
HuCNS-SC
Arm Type
Experimental
Arm Description
HuCNS-SC cells
Intervention Type
Drug
Intervention Name(s)
HuCNS-SC cells
Other Intervention Name(s)
Human Central Nervous System Stem Cells
Intervention Description
HuCNS-SC cells transplantation directly into the subretinal space of one eye (Study eye) in a single transplant procedure.
Primary Outcome Measure Information:
Title
Number of subjects with adverse events
Description
Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.
Time Frame
One year following transplant surgery
Secondary Outcome Measure Information:
Title
Assessment of visual function changes from baseline
Description
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.
Time Frame
At frequent intervals for one year following transplantation
10. Eligibility
Sex
All
Minimum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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
Exclusion Criteria:
Prior vitreal or retinal surgery
Glaucoma
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Stephen Huhn, M.D.
Organizational Affiliation
StemCells, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Retina-Vitreaous Associates Medical Group
City
Los Angeles
State/Province
California
ZIP/Postal Code
90211
Country
United States
Facility Name
Byers Eye Institute at Stanford, Stanford Hospital and Clinics
City
Palo Alto
State/Province
California
ZIP/Postal Code
94303
Country
United States
Facility Name
New York Eye and Ear Infirmary
City
New York
State/Province
New York
ZIP/Postal Code
10003
Country
United States
Facility Name
Retina Research Institute of Texas
City
Abilene
State/Province
Texas
ZIP/Postal Code
79606
Country
United States
Facility Name
Retina Foundation of the Southwest
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
12. IPD Sharing Statement
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Study of Human Central Nervous System Stem Cells (HuCNS-SC) in Age-Related Macular Degeneration (AMD)
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