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

NCT02445612 | Completed DMC

• 2 other identifiers: 7316-CL-0004ACT MA09-hRPE 001(SMD)LTFU
• Study Type: observational
• Target: 13
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

Stargardt's Macular Dystrophy

Keywords

fundus flavimaculatus; ASP7316; SMD; long term follow-up; juvenile macular dystrophy

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Incidence of graft failure or rejection

  4 years

• Number of patients with changes in ocular examinations or images

  4 years

Study Arms (1)

Experimental: MA09-hRPE

Sub-retinal transplantation of MA09-hRPE cells

Biological: MA09-hRPE

Interventions

MA09-hRPE BIOLOGICAL

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

Also known as: ASP7316

Experimental: MA09-hRPE

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population is by invitation only for previous participants in the core protocol.

You may qualify if:

• 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.

Sponsors & Collaborators

• Astellas Institute for Regenerative Medicine: lead

Study Sites (3)

Jules Stein Eye Institute, UCLA School of Medicine

Los Angeles, California, 90095, United States

Location

Bascom Palmer Eye Institute

Miami, Florida, 33136, United States

Location

Wills Eye Institute-Mid Atlantic Retina

Philadelphia, Pennsylvania, 19107, United States

Location

Related 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/S0140-6736(12)60028-2. Epub 2012 Jan 24.

  PMID: 22281388 RESULT

• 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 pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies. Lancet. 2015 Feb 7;385(9967):509-16. doi: 10.1016/S0140-6736(14)61376-3. Epub 2014 Oct 15.

  PMID: 25458728 RESULT

Related Links

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

MeSH Terms

Conditions

Stargardt Disease

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Macular Degeneration; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Medical Director

  Astellas Institute for Regenerative Medicine

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 13, 2015
• First Posted: May 15, 2015
• Study Start: July 11, 2012
• Primary Completion: June 21, 2019
• Study Completion: June 21, 2019
• Last Updated: October 31, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)