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

This study is currently recruiting participants.
Verified January 2014 by StemCells, Inc.
Information provided by (Responsible Party):
StemCells, Inc. Identifier:
First received: June 21, 2012
Last updated: January 17, 2014
Last verified: January 2014

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.

Condition Intervention Phase
Age Related Macular Degeneration
Macular Degeneration
Drug: HuCNS-SC cells
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:

Further study details as provided by StemCells, Inc.:

Primary Outcome Measures:
  • 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.

Secondary Outcome Measures:
  • 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.

Estimated Enrollment: 16
Study Start Date: June 2012
Estimated Study Completion Date: June 2014
Estimated Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: HuCNS-SC
HuCNS-SC cells
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

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 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.


Ages Eligible for Study:   50 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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)
  Contacts and Locations
Please refer to this study by its identifier: NCT01632527

Contact: Jocelyn Rojas, RN 510-456-4000

United States, California
Byers Eye Institute at Stanford, Stanford Hospital and Clinics Recruiting
Palo Alto, California, United States, 94303
Contact: Lorella Cabael    650-498-4486   
Contact: Ted Leng, M.D.    650-498-4486      
Principal Investigator: Theodore Leng, M.D.         
United States, New York
New York Eye and Ear Infirmary Recruiting
New York, New York, United States, 10003
Contact: Katy Tai, CCRC    212-979-4251   
Principal Investigator: Richard Rosen, M.D.         
United States, Texas
Retina Foundation of the Southwest Recruiting
Dallas, Texas, United States, 75231
Contact: Kirsten Locke, RN    214-363-3911   
Principal Investigator: David Birch, PhD         
Sponsors and Collaborators
StemCells, Inc.
Study Director: Stephen Huhn, M.D. StemCells, Inc.
  More Information

Additional Information:
No publications provided

Responsible Party: StemCells, Inc. Identifier: NCT01632527     History of Changes
Other Study ID Numbers: StemCells CL-N01-AMD
Study First Received: June 21, 2012
Last Updated: January 17, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by StemCells, Inc.:
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
Human central nervous system stemcells
dry age-related macular degeneration

Additional relevant MeSH terms:
Macular Degeneration
Geographic Atrophy
Retinal Degeneration
Retinal Diseases
Eye Diseases processed this record on April 17, 2014