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

Overview

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 Title of Study: “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

  • Study Type: Interventional
  • Study Design
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: June 2015

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.

Interventions

  • Drug: HuCNS-SC cells
    • HuCNS-SC cells transplantation directly into the subretinal space of one eye (Study eye) in a single transplant procedure.

Arms, Groups and Cohorts

  • Experimental: HuCNS-SC
    • HuCNS-SC cells

Clinical Trial Outcome Measures

Primary Measures

  • 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.

Secondary Measures

  • 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.

Participating in This Clinical Trial

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)

Gender Eligibility: All

Minimum Age: 50 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • StemCells, Inc.
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Stephen Huhn, M.D., Study Director, StemCells, Inc.

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