Clinical trial NCT01632527: Study of Human Central Nervous System Stem Cells (HuCNS-SC) in Age-Related Macular Degeneration (AMD)
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Age Related Macular Degeneration
Macular Degeneration
AMD
Keywords: 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
Dry AMD
Human central nervous system stemcells
dry age-related macular degeneration
Lead sponsor: StemCells, Inc.
Sponsor class: INDUSTRY
First posted: 2012-07-03
Enrollment: 15
Sex: ALL
Minimum age (years): 50
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Eligibility 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)

Primary outcomes:
measure: Number of subjects with adverse events
timeFrame: One year following transplant surgery
Secondary outcomes:
measure: Assessment of visual function changes from baseline
timeFrame: At frequent intervals for one year following transplantation
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Retina-Vitreaous Associates Medical Group
state: California
city: Palo Alto
country: United States
facility: Byers Eye Institute at Stanford, Stanford Hospital and Clinics
state: California
city: New York
country: United States
facility: New York Eye and Ear Infirmary
state: New York
city: Abilene
country: United States
facility: Retina Research Institute of Texas
state: Texas
city: Dallas
country: United States
facility: Retina Foundation of the Southwest
state: Texas
Study officials:
affiliation: StemCells, Inc.
name: Stephen Huhn, M.D.
role: STUDY_DIRECTOR
References: