Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD)

Stargardt's Macular Dystrophy Clinical Trial

Official title:

A Phase I, Open-Label, 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: NCT01625559
• Other study ID #: CHA_CTP_0903
• Status: Completed
• Phase: Phase 1
• Start date: September 2012
• Est. completion date: April 2015

Study information

• Verified date: February 2024
• Source: CHABiotech CO., Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD

Description:

• to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD

• to evaluate the preliminary efficacy of MA09-hPRE in patients with SMD

Recruitment information / eligibility

• Status: Completed
• Enrollment: 3
• Est. completion date: April 2015
• Est. primary completion date: April 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 20 Years and older

Eligibility

Inclusion Criteria:

• Adult male or female over 20 years of age.

• Clinical diagnosis of advanced SMD.

• The visual acuity of the eye to receive the transplant will be no better than hand movement.

• The visual acuity of the eye that is not to receive the transplant will be no better than 24 (20/320) Early Treatment of Diabetic Retinopathy Study (ETDRS) letters.

Exclusion Criteria:

• History of malignancy.

• History of myocardial infarction in previous 12 months.

• History of diabetes mellitus.

• Any immunodeficiency.

• Any current immunosuppressive therapy other than intermittent or low dose cortico steroids.

• Serologic evidence of infection with Hepatitis B, Hepatitis C, or HIV.

• Current participation in any other clinical trial.

• Participation within previous 6 months in any clinical trial of a drug by ocular or systemic administration.

• Any other sight-threatening ocular disease.

• Any chronic ocular medications. Any history of retinal vascular disease (compromised blood-retinal barrier). Glaucoma. Uveitis or other intraocular inflammatory disease. Significant lens opacities or other media opacity. Ocular lens removal within previous 3 months

Related Conditions & MeSH terms

• Macular Degeneration
• Stargardt Disease
• Stargardt's Macular Dystrophy

Intervention

Biological:
• MA09-hRPE
MA09-hRPE cells

Locations

Country	Name	City	State
Korea, Republic of	CHA Bundang Medical Center	Seongnam-si	Gyeonggi-do

Sponsors (1)

Lead Sponsor	Collaborator
CHABiotech CO., Ltd

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	safety and tolerance of transplantation	The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe and tolerated in the absence of: Any grade 2 (NCI grading system) or greater adverse event related to the cell product; Any evidence that the cells are contaminated with an infectious agent; Any evidence that the cells show tumorigenic potential	18 months
Secondary	Evidence of successful engraftment	Evidence of successful engraftmentEvidence of successful engraftment will consist of: Structural evidence (OCT imaging, fluorescein angiography, autofluorescense photography, slit-lamp examination with fundus photography) that cells have been implanted in the correct location Electroretinographic evidence (mfERG) showing enhanced activity in the implant location	18 months