Study to Evaluate Sub-retinal Transplantation of Retinal Pigmented Epithelial Cells in Patients With Dry AMD

NCT ID: NCT02563782

Last Updated: 2017-07-26

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE2
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-08-24
• Study Completion Date: 2017-05-05

Brief Summary

To evaluate the safety of 3 regimens of short-term, low-dose systemic IMT as rejection prophylaxis prior to and/or following transplant of MA09-hRPE cells.

Detailed Description

This study will be a Phase 2, double-masked, randomized, parallel group, sham surgery/placebo control, multi-center trial. Subjects will be randomized in a 3:1 ratio to either the treatment or control group respectively. Subjects randomized to the treatment group will receive transplantation with 200,000 MA09-hRPE (human embryonic stem cell derived retinal pigmented epithelial)cells in one eye. Subjects randomized to the control group will have a sham surgery without transplantation of MA09-hRPE cells. The study eye must meet all eligibility criteria. If both eyes meet all eligibility criteria, then the study eye will be the eye with the worst Best Corrected Visual Acuity (BCVA) score at screening. If both eyes have identical BCVA scores, then the study eye will be chosen by the Investigator and the subject. There will be 3 cohorts, each with a different regimen of low-dose IMT [tacrolimus and mycophenolate mofetil (MMF)]. Subjects will be randomized to treatment or control within cohorts, defined by severity of BCVA in the study eye at Screening. Enrollment in Cohort 1 and 2 will be concurrent. Enrollment into Cohort 3 will begin once Cohort 2 is fully enrolled.

Conditions

Age-Related Macular Degeneration

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Control group

Sham surgery and placebo immunosuppressive therapy (IMT)

Group Type: SHAM_COMPARATOR

Placebo tacrolimus and mycophenolate mofetil

Intervention Type: DRUG

placebo

Sham Surgery

Intervention Type: PROCEDURE

Sham surgery

Active Group

Sub-retinal transplantation of MA09-hRPE cells and IMT

Group Type: ACTIVE_COMPARATOR

Sub-retinal transplantation of MA09-hRPE cells

Intervention Type: BIOLOGICAL

transplantation

tacrolimus and mycophenolate mofetil

Intervention Type: DRUG

Immunosuppressive Agents

Interventions

Sub-retinal transplantation of MA09-hRPE cells

transplantation

Intervention Type: BIOLOGICAL

tacrolimus and mycophenolate mofetil

Immunosuppressive Agents

Intervention Type: DRUG

Placebo tacrolimus and mycophenolate mofetil

placebo

Intervention Type: DRUG

Sham Surgery

Sham surgery

Intervention Type: PROCEDURE

Eligibility Criteria

Inclusion Criteria

• Geographic atrophy (GA) secondary to AMD with no evidence of prior to active choroidal neovascularization (CNV) in the study eye.
• BCVA in the study eye must be between 4 and 58 Early Treatment of Diabetic Retinopathy Study (EDTRS) letters (20/800 to 20/80; 0.025-0.25)
• Subjects must be willing to take IMT and willing to discontinue any medication that has a known strong interaction with tacrolimus or mycophenolate mofetil (MMF)

Exclusion Criteria

• Macular atrophy due to causes other than AMD
• Other sight-threatening ocular disease
• Current or prior history of optic neuropathy, retinal dystrophy, retinitis pigmentosa, chorioretinitis, vasoocclusive disease, retinal vascular disease or retinal degenerative disease OTHER than AMD
• History of uveitis
• History of allergic reaction to sulfa drugs
• Solid organ or bone marrow transplant recipient
• History of malignancy within the previous 5 years (except for BCC (basal cell carcinoma), SCC (squamous cell carcinoma) or in-situ cervical)
• History of myocardial infarction with past 12 months
• History of clinically significant cardiac dysrhythmia
• History of diabetes mellitus, bowel disease, tuberculosis
• Prior treatment for non-exudative AMD
• Intraocular, refractive or cataract surgery in the last 12 weeks
• Prior retinal surgery, vitrectomy, macular laser photocoagulation, external beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery (except cataract surgery)
• Receipt of gene transfer of cell transplant therapy in a prior clinical trial
• Participation in any other interventional clinical trial within the last 12 weeks

• Minimum Eligible Age: 50 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Astellas Institute for Regenerative Medicine

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director

Role: STUDY_DIRECTOR

Astellas Institute for Regenerative Medicine

Locations

Wills Eye Institute

Philadelphia, Pennsylvania, United States

Countries

United States

Other Identifiers

MA09-hRPE AMD 02 PORTRAY

Identifier Type: OTHER

Identifier Source: secondary_id

7317-CL-0002

Identifier Type: -

Identifier Source: org_study_id