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| Sponsor: | Advanced Cell Technology |
|---|---|
| Information provided by (Responsible Party): | Advanced Cell Technology |
| ClinicalTrials.gov Identifier: | NCT01345006 |
Purpose
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 Stargardt's Macular Dystrophy (SMD).
| Condition | Intervention | Phase |
|---|---|---|
|
Stargardt's Macular Dystrophy |
Biological: MA09-hRPE Cellular therapy |
Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label |
| Official Title: | 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 Stargardt's Macular Dystrophy (SMD) |
The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe and tolerated in the absence of:
Evidence of successful engraftment will consist of:
| Estimated Enrollment: | 12 |
| Study Start Date: | April 2011 |
| Estimated Study Completion Date: | September 2013 |
| Estimated Primary Completion Date: | July 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Treatment
Patients will undergo subretinal injection of MA09-hRPE
|
Biological: MA09-hRPE Cellular therapy
Cohort 1 50,000 cells Cohort 2 100,000 cells Cohort 3 150,000 cells Cohort 4 200,000 cells |
This study is a Phase I/II, open-label, non randomized, sequential, multi-center clinical trial. There will be 3 cohorts, each consisting of 3 patients. The enrolled cohorts will be as follows:
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.
Each cohort will be enrolled sequentially in turn.
The day of the cell implantation will be Day 0, and patients will remain in the study until the last visit at 12 months.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| United States, California | |
| Jules Stein Eye Institute, UCLA School of Medicine | Recruiting |
| Los Angeles, California, United States, 90095 | |
| Contact: Logan Hitchcock 310-825-3046 | |
| Principal Investigator: Steven Schwartz, MD | |
| United States, Pennsylvania | |
| Wills Eye Institute-Mid Atlantic Retina | Recruiting |
| Philadelphia, Pennsylvania, United States | |
| Contact: Shellie Markun research@midatlanticretina.com | |
| Principal Investigator: Carl D Regillo, MD | |
| Principal Investigator: | Steven Schwartz, MD | Jules Stein Eye Institute, UCLA School of Medicine, Los Angeles, CA |
| Principal Investigator: | Carl Regillo, MD | Wills Eye Institute, Philadelphia, PA |
More Information
| Responsible Party: | Advanced Cell Technology |
| ClinicalTrials.gov Identifier: | NCT01345006 History of Changes |
| Other Study ID Numbers: | ACT SMD 01 MA09-hRPE |
| Study First Received: | April 28, 2011 |
| Last Updated: | February 13, 2012 |
| Health Authority: | United States: Food and Drug Administration |
|
SMD fundus flavimaculatus juvenile macular dystrophy |
|
Macular Degeneration Retinal Degeneration Retinal Diseases Eye Diseases |