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

Study Description

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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.

Condition or disease	Intervention/treatment	Phase
Age Related Macular Degeneration; Macular Degeneration; AMD	Drug: HuCNS-SC cells	Phase 1; Phase 2

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.

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	15 participants
Intervention Model:	Single Group Assignment
Masking:	None (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
Study Start Date :	June 2012
Actual Primary Completion Date :	June 2015
Actual Study Completion Date :	June 2015

Arms and Interventions

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Arm	Intervention/treatment
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

Outcome Measures

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Primary Outcome Measures :

1. 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 Outcome Measures :

1. 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.

Eligibility Criteria

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Ages Eligible for Study:	50 Years and older (Adult, Senior)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

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)

Contacts and Locations

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Locations

United States, California
Retina-Vitreaous Associates Medical Group
Los Angeles, California, United States, 90211
Byers Eye Institute at Stanford, Stanford Hospital and Clinics
Palo Alto, California, United States, 94303
United States, New York
New York Eye and Ear Infirmary
New York, New York, United States, 10003
United States, Texas
Retina Research Institute of Texas
Abilene, Texas, United States, 79606
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231

Sponsors and Collaborators

StemCells, Inc.

Investigators

Study Director:	Stephen Huhn, M.D.	StemCells, Inc.

More Information

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	StemCells, Inc.
ClinicalTrials.gov Identifier:	NCT01632527 History of Changes
Other Study ID Numbers:	StemCells CL-N01-AMD
First Posted:	July 3, 2012
Last Update Posted:	September 14, 2015
Last Verified:	September 2015

Keywords provided by StemCells, Inc.:

Age-Related Macular Degeneration; Geographic Atrophy of Age-Related Macular Degeneration; Macular Degeneration; dry age-related macular degeneration; Dry AMD with GA; Stem Cell Transplantation	Stem Cell transplant; Stem cells for AMD; Human neural stem cells; multipotent stem cells; Dry AMD; Human central nervous system stemcells

Additional relevant MeSH terms:

Macular Degeneration; Geographic Atrophy; Retinal Degeneration; Retinal Diseases; Eye Diseases