NCT01344993

Brief Summary

This is a safety and tolerability trial to evaluate the effect of subretinal injection of human embryonic stem cell derived retinal pigment epithelium cells in patients with dry Age Related Macular Degeneration (AMD) and to perform exploratory evaluation of potential efficacy endpoints to be used in future studies retinal pigment epithelium (RPE) cellular therapy.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
13

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jun 2011

Longer than P75 for phase_1

Geographic Reach
1 country

4 active sites

Status
completed

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

First Submitted

Initial submission to the registry

April 28, 2011

Completed
1 day until next milestone

First Posted

Study publicly available on registry

April 29, 2011

Completed
1 month until next milestone

Study Start

First participant enrolled

June 9, 2011

Completed
4.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 19, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 19, 2015

Completed
Last Updated

October 31, 2024

Status Verified

October 1, 2024

Enrollment Period

4.2 years

First QC Date

April 28, 2011

Last Update Submit

October 29, 2024

Conditions

Dry Age Related Macular Degeneration

Keywords

Dry AMDGeographic atrophy

Outcome Measures

Primary Outcomes (1)

  • Safety of hESC derived RPE cells

    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

    12 Months

Secondary Outcomes (1)

  • exploratory evaluations for potential efficacy endpoints.

    12 months

Study Arms (1)

MA09-hRPE

EXPERIMENTAL

Experimental: Subretinal injection of MA09-hRPE * Cohort 1 50,000 cells * Cohort 2 100,000 cells * Cohort 2a Better Vision 100,000 cells * Cohort 3 150,000 cells * Cohort 4 200,000 cells

Biological: MA09-hRPE

Interventions

MA09-hRPEBIOLOGICAL

Cohort 1 50,000 cells Cohort 2 100,000 cells Cohort 2a Better Vision 100,000 cells Cohort 3 150,000 cells Cohort 4 200,000 cells

MA09-hRPE

Eligibility Criteria

Age55 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Adult male or female over 55 years of age.
  • Patient should be in sufficiently good health to reasonably expect survival for at least four years after treatment
  • Clinical findings consistent with advanced dry AMD with evidence of one or more areas of \>250microns of geographic atrophy (as defined in the Age-Related eye Disease Study \[AREDS\] study) involving the central fovea.
  • GA defined as attenuation or loss of RPE as observed by biomicroscopy, OCT, and FA.
  • No evidence of current or prior choroidal neovascularization in the treated eye
  • The visual acuity of the eye to receive the transplant will be no better than 20/400. The visual acuity of the eye in the better vision cohort to receive the transplant will be no better than 20/100.
  • The visual acuity of the eye that is not to receive the transplant will be no better than 20/400 for the worse vision patients and no worse than 20/100 for the better vision patients.
  • Electrophysiological findings consistent with advanced dry AMD.
  • Medically suitable to undergo vitrectomy and subretinal injection.
  • Medically suitable for general anesthesia or waking sedation, if needed.
  • Medically suitable for transplantation of an embryonic stem cell line:
  • Any laboratory value which falls slightly outside of the normal range will be reviewed by the Medical Monitor and Investigators to determine its clinical significance. If it is determined not to be clinically significant, the patient may be enrolled into the study.

You may not qualify if:

  • Negative urine screen for drugs of abuse.
  • Negative human immunodeficiency virus (HIV), hepatitis B (HBV), hepatitis C (HCV) serologies.
  • No history of malignancy (with the exception of successfully treated (excised) basal cell carcinoma\[skin cancer\] or successfully treated squamous cell carcinoma of the skin).
  • Negative cancer screening within previous 6 months:
  • complete history \& physical examination;
  • dermatological screening exam for malignant lesions;
  • negative fecal occult blood test \& negative colonoscopy within previous 7 years;
  • negative chest roentgenogram (CXR);
  • normal CBC \& manual differential;
  • negative urinalysis (U/A);
  • normal thyroid exam;
  • if male, normal testicular examination; digital rectal examination (DRE) and prostate specific antigen (PSA);
  • if female, normal pelvic examination with Papanicolaou smear; and
  • If female, normal clinical breast exam and, negative mammogram.
  • If female and of childbearing potential, willing to use two effective forms of birth control during the study.
  • +33 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Jules Stein Eye Institute, UCLA School of Medicine

Los Angeles, California, 90095, United States

Location

Bascom Palmer Eye Institute

Miami, Florida, United States

Location

Mass Eye and Ear

Boston, Massachusetts, United States

Location

Wills Eye Institute-Mid Atlantic Retina

Philadelphia, Pennsylvania, 19107, United States

Location

Related Publications (2)

  • Schwartz SD, Regillo CD, Lam BL, Eliott D, Rosenfeld PJ, Gregori NZ, Hubschman JP, Davis JL, Heilwell G, Spirn M, Maguire J, Gay R, Bateman J, Ostrick RM, Morris D, Vincent M, Anglade E, Del Priore LV, Lanza R. Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies. Lancet. 2015 Feb 7;385(9967):509-16. doi: 10.1016/S0140-6736(14)61376-3. Epub 2014 Oct 15.

    PMID: 25458728DERIVED

  • Schwartz SD, Hubschman JP, Heilwell G, Franco-Cardenas V, Pan CK, Ostrick RM, Mickunas E, Gay R, Klimanskaya I, Lanza R. Embryonic stem cell trials for macular degeneration: a preliminary report. Lancet. 2012 Feb 25;379(9817):713-20. doi: 10.1016/S0140-6736(12)60028-2. Epub 2012 Jan 24.

    PMID: 22281388DERIVED

Related Links

MeSH Terms

Conditions

Geographic Atrophy

Condition Hierarchy (Ancestors)

Macular DegenerationRetinal DegenerationRetinal DiseasesEye Diseases

Study Officials

  • Medical Director

    Astellas Institute for Regenerative Medicine

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
OTHER
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 28, 2011

First Posted

April 29, 2011

Study Start

June 9, 2011

Primary Completion

August 19, 2015

Study Completion

August 19, 2015

Last Updated

October 31, 2024

Record last verified: 2024-10

Data Sharing

IPD Sharing
Will not share

Access to anonymized individual participant level data will not be provided for this trial. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.

Locations

US(4)