NCT01345006

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 Stargardt's Macular Dystrophy (SMD).

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

3 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
2 months until next milestone

Study Start

First participant enrolled

June 16, 2011

Completed
4.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 10, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 10, 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

Stargardt's Macular Dystrophy

Keywords

juvenile macular dystrophySMDfundus flavimaculatus

Outcome Measures

Primary Outcomes (2)

  • The safety and tolerance of transplantation of hESC-derived RPE cells MA09-hRPE

    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

  • Safety Assessments

    * Adverse Event and Serious Adverse Event assessment * Clinical monitoring * Serial vital signs * Clinical laboratory tests * Directed ophthalmological monitoring * Monitoring of RPE cells acceptance/integrity/rejection * Monitoring of local and systemic infection * Monitoring of tumorigenic cell transformation

    12 months

Secondary Outcomes (1)

  • Evidence of successful engraftment

    12 months

Study Arms (1)

MA09-hRPE

EXPERIMENTAL

Patients will undergo subretinal injection of MA09-hRPE

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

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Adult male or female over 18 years of age.
  • Clinical diagnosis of advanced SMD.
  • If known, the patient's genotype will be recorded in the medical history, if unknown, patient will allow for the submission of a sample for genotyping.Clinical findings consistent with SMD.
  • 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.
  • Peripheral visual field constriction documented on standard kinetic visual field testing.
  • Electrophysiological findings consistent with SMD.
  • 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:
  • Normal serum chemistry (sequential multi-channel analyzer 20 \[SMA- 20\]) and hematology (complete blood count \[CBC\], prothrombin time \[PT\], and activated partial thromboplastin time \[aPTT\]) screening tests.
  • 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 basal cell or squamous cell carcinoma of the skin.
  • Negative cancer screening within previous 6 months:
  • +13 more criteria

You may not qualify if:

  • History of malignancy,with the exception of successfully treated basal cell or squamous cell carcinoma of the skin.
  • History of myocardial infarction in previous 12 months.
  • History of diabetes mellitus.
  • Any immunodeficiency.
  • Any current immunosuppressive therapy other than intermittent or low dose corticosteroids.
  • Serologic evidence of infection with Hepatitis B, Hepatitis C, or HIV.
  • Current participation in any other clinical trial.
  • Participation within previous 6 months in any clinical trial of a drug by ocular or systemic administration.
  • Any other sight-threatening ocular disease.
  • Any chronic ocular medications.
  • Any history of retinal vascular disease (compromised blood-retinal barrier.
  • Glaucoma.
  • Uveitis or other intraocular inflammatory disease.
  • Significant lens opacities or other media opacity.
  • Ocular lens removal within previous 3 months.
  • +2 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Jules Stein Eye Institute, UCLA School of Medicine

Los Angeles, California, 90095, United States

Location

Bascom Palmer Eye institute

Miami, Florida, 33136, United States

Location

Wills Eye Institute-Mid Atlantic Retina

Philadelphia, Pennsylvania, 19107, United States

Location

Related Publications (3)

  • Davis JL. The Blunt End: Surgical Challenges of Gene Therapy for Inherited Retinal Diseases. Am J Ophthalmol. 2018 Dec;196:xxv-xxix. doi: 10.1016/j.ajo.2018.08.038. Epub 2018 Sep 5.

    PMID: 30194931DERIVED

  • 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

Stargardt Disease

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesMacular DegenerationRetinal DegenerationRetinal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

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 16, 2011

Primary Completion

August 10, 2015

Study Completion

August 10, 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(3)