Age Related Macular DegenerationPhase 1/2NCT01632527
Study of Human Central Nervous System Stem Cells (HuCNS-SC) in Age-Related Macular Degeneration (AMD)
A Phase 1/2 study testing HuCNS-SC cells in people with Age Related Macular Degeneration and Macular Degeneration, run by StemCells, Inc. at 5 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Age Related Macular Degeneration, Macular Degeneration, AMD
- What is being tested?
- HuCNS-SC cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 5 sitesUnited States
- Study dates
- Jun 2012to Jun 2015
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- Autoimmune disease
- Allergy to tacrolimus, mycophenolate mofetil (MMF), scopolamine, Cyclogyl, Moxifloxacin, or Gatifloxacin
- Current or prior malignancy (or is on chemotherapy)
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Retina Research Institute of Texas
Abilene, Texas, 79606
Retina Foundation of the Southwest
Dallas, Texas, 75231
Retina-Vitreaous Associates Medical Group
Los Angeles, California, 90211
New York Eye and Ear Infirmary
New York, New York, 10003
Byers Eye Institute at Stanford, Stanford Hospital and Clinics
Palo Alto, California, 94303
About this study
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
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.
Read the 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.
- Conditions
- Age Related Macular Degeneration, Macular Degeneration, AMD
- Being tested
- HuCNS-SC cells
- 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
- Medical subject headings
- Macular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HuCNS-SC cellsDrug
Also called Human Central Nervous System Stem Cells
HuCNS-SC cells transplantation directly into the subretinal space of one eye (Study eye) in a single transplant procedure.
| Study group | Role | What they receive |
|---|---|---|
| HuCNS-SC | Experimental | HuCNS-SC cells Receives: Drug: HuCNS-SC cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with adverse events
Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.
Measured: One year following transplant surgery
Secondary outcomes (1)
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.
Measured: At frequent intervals for one year following transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jul 2012 (estimated)
- Study startJun 2012
- Primary completionJun 2015
- Study completionJun 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Age Related Macular DegenerationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- StemCells, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Stephen Huhn, M.D., Study director, StemCells, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01632527
- Sponsor's protocol ID
- StemCells CL-N01-AMD
- Status last verified
- Sep 2015
- First submitted
- 21 Jun 2012
- Last updated
- 14 Sep 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01632527
- Acronym / sponsor ID
- StemCells CL-N01-AMD
- 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
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- StemCells, Inc.Industry (pharma, biotech, devices)
- Study start
- Jun 2012
- Primary completion
- Jun 2015 (actual)
- Study completion
- Jun 2015 (actual)
- First posted
- 3 Jul 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HuCNS-SC | Experimental | Drug: HuCNS-SC cellsHuCNS-SC cells |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HuCNS-SC cellsAlso: Human Central Nervous System Stem CellsHuCNS-SC cells transplantation directly into the subretinal space of one eye (Study eye) in a single transplant procedure. | Drug | HuCNS-SC |
Primary outcome
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.
1 secondary outcome
Assessment of visual function changes from baseline
Time frame: At frequent intervals for one year following transplantation
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Sep 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Stephen Huhn, M.D. study director
StemCells, Inc.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing HuCNS-SC cells in people with Age Related Macular Degeneration and Macular Degeneration, run by StemCells, Inc. at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Sep 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of subjects with adverse events (time frame: One year following transplant surgery). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Sep 2015.