Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy Patients

Stargardt's Macular Dystrophy Clinical Trial

Official title:

Long Term Follow Up to 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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02445612
• Other study ID #: 7316-CL-0004
• Secondary ID: ACT MA09-hRPE 00
• Status: Completed
• Start date: July 11, 2012
• Est. completion date: June 21, 2019

Study information

• Verified date: November 2020
• Source: Astellas Pharma Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The purpose of this study is to evaluate the long term safety and tolerability of MA09-hRPE cellular therapy in patients with advanced Stargardt's Macular Dystrophy (SMD) from one to five years following the surgical procedure to implant the MA09-hRPE cells.

Description:

This study is a long term, follow up to the ACT MA09-hRPE 001 phase I/II trial, the phase I/II trial (referred to as the core protocol) was an open-label, non-randomized, dose escalation, multi-center trial. Thirteen SMD patients were treated in this trial. Ten patients with profound vision loss (visual acuity <=20/400) received a single subretinal injection of MA09-hRPE cells, starting at a dose of 50,000 MA09-hRPE cells (three patients), 100,000 MA09-hRPE cells transplanted (three patients), 150,000 MA09-hRPE cells transplanted (three patients) and increasing to a maximum dose of 200,000 MA09-hRPE cells transplanted (one patient). Three patients with severe to moderate vision loss (visual acuity <= 20/100) received a dose of 100,000 MA09-hRPE cells. All patients who participated in the core protocol are eligible for participation in this follow-up protocol.

The first visit of this long term follow-up protocol will correspond to the last visit of the core protocol, and will take place at 12 months post cell implantation. Informed consent will be obtained at this visit.

Patients will be evaluated at 18, 24, 36, 48 and 60 months post-transplant (or more frequently as clinically indicated). Follow-up will include obtaining information about ophthalmological findings and events of special interest as defined in the Primary Outcomes. At the last visit of this follow-up study, whether at 60 months post-transplant or at early discontinuation, patients will be invited to participate in a Safety Surveillance Study for an additional 10 years, under a separate protocol, which will continue to monitor the long term risks of MA09-hRPE cell transplantation.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 13
• Est. completion date: June 21, 2019
• Est. primary completion date: June 21, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Must have been treated with hESC-RPE cell transplant in the core protocol.

• Able to understand and willing to sign the informed consent to participate in the follow-up study.

Exclusion Criteria:

• There are no exclusion criteria

Related Conditions & MeSH terms

• Macular Degeneration
• Stargardt's Macular Dystrophy

Intervention

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

Locations

Country	Name	City	State
United States	Jules Stein Eye Institute, UCLA School of Medicine	Los Angeles	California
United States	Bascom Palmer Eye Institute	Miami	Florida
United States	Wills Eye Institute-Mid Atlantic Retina	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Astellas Institute for Regenerative Medicine

Country where clinical trial is conducted

United States, 

References & Publications (2)

Schwartz SD, Hubschman JP, Heilwell G, Franco-Cardenas V, Pan CK, Ostrick RM, Mickunas E, Gay R, Klimanskaya I, Lanza R. Embryonic stem cell trials for macular degeneration: a preliminary report. Lancet. 2012 Feb 25;379(9817):713-20. doi: 10.1016/S0140-67 — View Citation

Schwartz SD, Regillo CD, Lam BL, Eliott D, Rosenfeld PJ, Gregori NZ, Hubschman JP, Davis JL, Heilwell G, Spirn M, Maguire J, Gay R, Bateman J, Ostrick RM, Morris D, Vincent M, Anglade E, Del Priore LV, Lanza R. Human embryonic stem cell-derived retinal pi — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety assessed by Adverse Events (AEs) of special interest in regards to the investigational product	This will include obtaining information about ophthalmological findings and Serious Adverse Events (SAEs) that are neurologic, infectious, hematologic or fatal, pregnancy in a female subject or the partner of a male subject and pregnancy outcome, any adverse event (AE) that causes a subject to withdraw from the study, any new diagnosis of an ocular or immune-mediated disorder, cancer, ectopic or proliferative cell growth (Retinal pigment epithelium (RPE) or non-RPE) with adverse clinical consequence, and unexpected, clinically significant AEs possibly related to the cell transplant procedure or the investigational product (MA09-hRPE cells).	4 years
Secondary	Incidence of graft failure or rejection	Evidence of graft failure or rejection will consist of: Presence of retinal edema, cystoid macular edema, retinal white dots, retinal hemorrhage, serous retinal detachment, subretinal exudates, subretinal fibrosis, or vascular and/or optic disc leakage, elevated intraocular pressure or hypotony. Evidence of unanticipated and persistent or increasing non-infectious ocular inflammation (e.g., vasculitis, retinitis, choroiditis, vitritis, pars planitis, anterior segment inflammation/uveitis).	4 years
Secondary	Number of patients with changes in ocular examinations or images	The number of patients with clinically significant absolute values or changes from baseline in Intra-ocular pressure (IOP) and Best Corrected Visual Acuity (BCVA) will be summarized.	4 years

External Links

•   Link to results on the Astellas Clinical Study Results website.