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

September 10, 2015 updated by: StemCells, Inc.

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

50 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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 Names:
  • Human Central Nervous System Stem Cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
One year following transplant surgery

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
At frequent intervals for one year following transplantation

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

StemCells, Inc.

Investigators

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2012

Primary Completion (Actual)

June 1, 2015

Study Completion (Actual)

June 1, 2015

Study Registration Dates

First Submitted

June 21, 2012

First Submitted That Met QC Criteria

July 2, 2012

First Posted (Estimate)

July 3, 2012

Study Record Updates

Last Update Posted (Estimate)

September 14, 2015

Last Update Submitted That Met QC Criteria

September 10, 2015

Last Verified

September 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • StemCells CL-N01-AMD

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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