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

Age-Related Macular Degeneration Clinical Trial

— PORTRAY  

Official title:

Ph 2, Double-Masked, Randomized, Parallel, Sham Surgery/Placebo Control, Multi-Center Study to Evaluate Systemic IMT Regimens as Graft Rejection Prophylaxis Following Transplantation of hESC Derived RPE Cells in Patients With AMD

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT02563782
• Other study ID #: 7317-CL-0002
• Secondary ID: MA09-hRPE AMD 02
• Status: Withdrawn
• Phase: Phase 2
• First received: September 21, 2015
• Last updated: July 25, 2017
• Start date: August 24, 2015
• Est. completion date: May 5, 2017

Study information

• Verified date: July 2017
• Source: Astellas Pharma Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: May 5, 2017
• Est. primary completion date: May 5, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 50 Years to 85 Years

Eligibility

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

Related Conditions & MeSH terms

• Age-Related Macular Degeneration
• Macular Degeneration

Intervention

Biological:
• Sub-retinal transplantation of MA09-hRPE cells
transplantation

Drug:
• tacrolimus and mycophenolate mofetil
Immunosuppressive Agents
• Placebo tacrolimus and mycophenolate mofetil
placebo

Procedure:
• Sham Surgery
Sham surgery

Locations

Country	Name	City	State
United States	Wills Eye Institute	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Astellas Institute for Regenerative Medicine

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of subjects with evidence of graft failure or rejection.		18 months
Secondary	Change in area of geographic atrophy by Optical coherence tomography	Change from baseline to Week 78	18 months
Secondary	Change in area of geographic atrophy by Autofluorescence	Change from baseline to Week 78	18 months
Secondary	Change in average Best Corrected Visual Acuity (BCVA)	Change from baseline to Week 78	18 months