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

Primary Purpose

Age-Related Macular Degeneration

Status

Withdrawn

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Sub-retinal transplantation of MA09-hRPE cells

tacrolimus and mycophenolate mofetil

Placebo tacrolimus and mycophenolate mofetil

Sham Surgery

About this trial

This is an interventional treatment trial for Age-Related Macular Degeneration

Eligibility Criteria

50 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Wills Eye Institute

Arms of the Study

Arm 1

Arm 2

Arm Type

Sham Comparator

Active Comparator

Arm Label

Control group

Active Group

Arm Description

Sham surgery and placebo immunosuppressive therapy (IMT)

Sub-retinal transplantation of MA09-hRPE cells and IMT

Outcomes

Primary Outcome Measures

Number of subjects with evidence of graft failure or rejection.

Secondary Outcome Measures

Change in area of geographic atrophy by Optical coherence tomography

Change from baseline to Week 78

Change in area of geographic atrophy by Autofluorescence

Change from baseline to Week 78

Change in average Best Corrected Visual Acuity (BCVA)

Change from baseline to Week 78

Full Information

NCT ID

NCT02563782

First Posted

September 21, 2015

Last Updated

July 25, 2017

Sponsor

Astellas Institute for Regenerative Medicine

1. Study Identification

Unique Protocol Identification Number

NCT02563782

Brief Title

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

Acronym

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

Study Type

Interventional

2. Study Status

Record Verification Date

July 2017

Overall Recruitment Status

Withdrawn

Why Stopped

Changes to the study design and the cell line

Study Start Date

August 24, 2015 (Actual)

Primary Completion Date

May 5, 2017 (Actual)

Study Completion Date

May 5, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Astellas Institute for Regenerative Medicine

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Age-Related Macular Degeneration

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Control group

Arm Type

Sham Comparator

Arm Description

Sham surgery and placebo immunosuppressive therapy (IMT)

Arm Title

Active Group

Arm Type

Active Comparator

Arm Description

Sub-retinal transplantation of MA09-hRPE cells and IMT

Intervention Type

Biological

Intervention Name(s)

Sub-retinal transplantation of MA09-hRPE cells

Intervention Description

transplantation

Intervention Type

Drug

Intervention Name(s)

tacrolimus and mycophenolate mofetil

Intervention Description

Immunosuppressive Agents

Intervention Type

Drug

Intervention Name(s)

Placebo tacrolimus and mycophenolate mofetil

Intervention Description

placebo

Intervention Type

Procedure

Intervention Name(s)

Sham Surgery

Intervention Description

Sham surgery

Primary Outcome Measure Information:

Title

Number of subjects with evidence of graft failure or rejection.

Time Frame

18 months

Secondary Outcome Measure Information:

Title

Change in area of geographic atrophy by Optical coherence tomography

Description

Change from baseline to Week 78

Time Frame

18 months

Title

Change in area of geographic atrophy by Autofluorescence

Description

Change from baseline to Week 78

Time Frame

18 months

Title

Change in average Best Corrected Visual Acuity (BCVA)

Description

Change from baseline to Week 78

Time Frame

18 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

50 Years

Maximum Age & Unit of Time

85 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Medical Director

Organizational Affiliation

Astellas Institute for Regenerative Medicine

Official's Role

Study Director

Facility Information:

Facility Name

Wills Eye Institute

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19107

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Undecided

Learn more about this trial

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

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