Safety and Tolerability of Sub-retinal Transplantation of hESC Derived RPE (MA09-hRPE) Cells in Patients With Advanced Dry Age Related Macular Degeneration (Dry AMD)
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ClinicalTrials.gov Identifier: NCT01344993 |
Recruitment Status :
Completed
First Posted : April 29, 2011
Last Update Posted : July 7, 2021
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Tracking Information | |||||
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First Submitted Date ICMJE | April 28, 2011 | ||||
First Posted Date ICMJE | April 29, 2011 | ||||
Last Update Posted Date | July 7, 2021 | ||||
Actual Study Start Date ICMJE | June 9, 2011 | ||||
Actual Primary Completion Date | August 19, 2015 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
Safety of hESC derived RPE cells [ Time Frame: 12 Months ] The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe and tolerated in the absence of:
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Original Primary Outcome Measures ICMJE | Same as current | ||||
Change History | |||||
Current Secondary Outcome Measures ICMJE |
exploratory evaluations for potential efficacy endpoints. [ Time Frame: 12 months ] Secondary endpoints will be evaluated as exploratory evaluations for potential efficacy endpoints.
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Original Secondary Outcome Measures ICMJE |
exploratory evaluations for potential efficacy endpoints. [ Time Frame: 12 months ] Secondary endpoints will be evaluated as exploratory evaluations for potential efficacy endpoints.
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Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Safety and Tolerability of Sub-retinal Transplantation of hESC Derived RPE (MA09-hRPE) Cells in Patients With Advanced Dry Age Related Macular Degeneration | ||||
Official Title ICMJE | A Phase I/II, Open-Label, Multi-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (MA09-hRPE) Cells in Patients With Advanced Dry AMD | ||||
Brief Summary | This is a safety and tolerability trial to evaluate the effect of subretinal injection of human embryonic stem cell derived retinal pigment epithelium cells in patients with dry Age Related Macular Degeneration (AMD) and to perform exploratory evaluation of potential efficacy endpoints to be used in future studies retinal pigment epithelium (RPE) cellular therapy. | ||||
Detailed Description | This study is a Phase I/II, open-label, non randomized, sequential, multi-center clinical trial. There will be 5 cohorts, the 4 low vision cohorts will contain 3 patients, the better vision cohort will contain 4 patients. The enrolled cohorts will be as follows: Three AMD patients- 50,000 MA09-hRPE cells transplanted Three AMD patients- 100,000 MA09-hRPE cells transplanted Four Better Vision AMD patients- 100,000 MA09-hRPE cells transplanted Three AMD patients- 150,000 MA09-hRPE cells transplanted Three AMD patients- 200,000 MA09-hRPE cells transplanted Patients will be enrolled sequentially, and within each cohort of 3 patients, each patient's clinical course over the first 6 weeks following cell transplantation will be reviewed by an independent (DSMB) before enrollment is opened for the next 2 patients. A full safety assessment of all 3 patients in each cohort will be made by the DSMB when the 3rd patient in each cohort completes 4 weeks of follow-up, and before the first patient in the next cohort receives a cell transplant. The exception is the better vision group where all patients may be enrolled once DSMB approval has been received. Each cohort will be enrolled sequentially in turn, with the exception of the better vision cohort which may be enrolled in parallel with the other cohorts. The day of the cell implantation will be Day 0, and patients will remain in the study until the last visit at 12 months. |
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Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 1 Phase 2 |
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Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Other |
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Condition ICMJE | Dry Age Related Macular Degeneration | ||||
Intervention ICMJE | Biological: MA09-hRPE
Cohort 1 50,000 cells Cohort 2 100,000 cells Cohort 2a Better Vision 100,000 cells Cohort 3 150,000 cells Cohort 4 200,000 cells
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Study Arms ICMJE | Experimental: MA09-hRPE
Experimental: Subretinal injection of MA09-hRPE
Intervention: Biological: MA09-hRPE
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Completed | ||||
Actual Enrollment ICMJE |
13 | ||||
Original Estimated Enrollment ICMJE |
12 | ||||
Actual Study Completion Date ICMJE | August 19, 2015 | ||||
Actual Primary Completion Date | August 19, 2015 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Any laboratory value which falls slightly outside of the normal range will be reviewed by the Medical Monitor and Investigators to determine its clinical significance. If it is determined not to be clinically significant, the patient may be enrolled into the study.
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 55 Years and older (Adult, Older Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT01344993 | ||||
Other Study ID Numbers ICMJE | 7316-CL-0002 ACT MA09-hRPE AMD-001 ( Other Identifier: Sponsor ) |
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Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product | Not Provided | ||||
IPD Sharing Statement ICMJE |
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Current Responsible Party | Astellas Pharma Inc ( Astellas Institute for Regenerative Medicine ) | ||||
Original Responsible Party | Edmund Mickunas, VP Regulatory Affairs, Advanced Cell Technology, Inc | ||||
Current Study Sponsor ICMJE | Astellas Institute for Regenerative Medicine | ||||
Original Study Sponsor ICMJE | Ocata Therapeutics | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE |
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PRS Account | Astellas Pharma Inc | ||||
Verification Date | June 2021 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |