NCT02563782

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.

Trial Health

30
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Timeline
Completed

Started Aug 2015

Geographic Reach
1 country

1 active site

Status
withdrawn

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 24, 2015

Completed
28 days until next milestone

First Submitted

Initial submission to the registry

September 21, 2015

Completed
9 days until next milestone

First Posted

Study publicly available on registry

September 30, 2015

Completed
1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 5, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 5, 2017

Completed
Last Updated

July 26, 2017

Status Verified

July 1, 2017

Enrollment Period

1.7 years

First QC Date

September 21, 2015

Last Update Submit

July 25, 2017

Conditions

Age-Related Macular Degeneration

Outcome Measures

Primary Outcomes (1)

  • Number of subjects with evidence of graft failure or rejection.

    18 months

Secondary Outcomes (3)

  • Change in area of geographic atrophy by Optical coherence tomography

    18 months

  • Change in area of geographic atrophy by Autofluorescence

    18 months

  • Change in average Best Corrected Visual Acuity (BCVA)

    18 months

Study Arms (2)

Control group

SHAM COMPARATOR

Sham surgery and placebo immunosuppressive therapy (IMT)

Drug: Placebo tacrolimus and mycophenolate mofetilProcedure: Sham Surgery

Active Group

ACTIVE COMPARATOR

Sub-retinal transplantation of MA09-hRPE cells and IMT

Biological: Sub-retinal transplantation of MA09-hRPE cellsDrug: tacrolimus and mycophenolate mofetil

Interventions

Sub-retinal transplantation of MA09-hRPE cellsBIOLOGICAL

transplantation

Active Group
tacrolimus and mycophenolate mofetilDRUG

Immunosuppressive Agents

Active Group
Placebo tacrolimus and mycophenolate mofetilDRUG

placebo

Control group
Sham SurgeryPROCEDURE

Sham surgery

Control group

Eligibility Criteria

Age50 Years - 85 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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)

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Wills Eye Institute

Philadelphia, Pennsylvania, 19107, United States

Location

MeSH Terms

Conditions

Macular Degeneration

Interventions

TacrolimusMycophenolic Acid

Condition Hierarchy (Ancestors)

Retinal DegenerationRetinal DiseasesEye Diseases

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic ChemicalsCaproatesAcids, AcyclicCarboxylic AcidsFatty AcidsLipids

Study Officials

  • Medical Director

    Astellas Institute for Regenerative Medicine

    STUDY DIRECTOR
0

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 21, 2015

First Posted

September 30, 2015

Study Start

August 24, 2015

Primary Completion

May 5, 2017

Study Completion

May 5, 2017

Last Updated

July 26, 2017

Record last verified: 2017-07

Locations

US(1)