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

Macular Degeneration Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01632527
• Other study ID #: StemCells CL-N01-AMD
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: June 21, 2012
• Last updated: September 10, 2015
• Start date: June 2012
• Est. completion date: June 2015

Study information

• Verified date: September 2015
• Source: StemCells, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: June 2015
• Est. primary completion date: June 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 50 Years and older

Eligibility

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 Design

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

Related Conditions & MeSH terms

• Age Related Macular Degeneration
• AMD
• Geographic Atrophy
• Macular Degeneration

Intervention

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

Locations

Country	Name	City	State
United States	Retina Research Institute of Texas	Abilene	Texas
United States	Retina Foundation of the Southwest	Dallas	Texas
United States	Retina-Vitreaous Associates Medical Group	Los Angeles	California
United States	New York Eye and Ear Infirmary	New York	New York
United States	Byers Eye Institute at Stanford, Stanford Hospital and Clinics	Palo Alto	California

Sponsors (1)

Lead Sponsor	Collaborator
StemCells, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of subjects with adverse events	Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.	One year following transplant surgery	Yes
Secondary	Assessment of visual function changes from baseline	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	No