Study of Human Central Nervous System Stem Cells (HuCNS-SC) in Age-Related Macular Degeneration (AMD)

StemCells, Inc

Status and phase

Completed

Phase 2

Phase 1

Conditions

Age Related Macular Degeneration

Macular Degeneration

AMD

Treatments

Drug: HuCNS-SC cells

Study type

Interventional

Funder types

Industry

Identifiers

NCT01632527

StemCells CL-N01-AMD

Details and patient eligibility

About

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 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.

Enrollment

15 patients

Sex

All

Ages

50+ years old

Volunteers

No Healthy Volunteers

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)