Study of Human Central Nervous System Stem Cells (HuCNS-SC) in Age-Related Macular Degeneration (AMD)
Status: | Completed |
---|---|
Conditions: | Ocular |
Therapuetic Areas: | Ophthalmology |
Healthy: | No |
Age Range: | 50 - Any |
Updated: | 4/21/2016 |
Start Date: | June 2012 |
End Date: | June 2015 |
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
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.
of unilateral subretinal transplantation of HuCNS-SC cells in subjects with geographic
atrophy secondary to age-related macular degeneration.
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.
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.
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)
We found this trial at
5
sites
Click here to add this to my saved trials

Click here to add this to my saved trials

Click here to add this to my saved trials

Click here to add this to my saved trials

Click here to add this to my saved trials
