Status:
COMPLETED
Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)
Lead Sponsor:
Astellas Institute for Regenerative Medicine
Conditions:
Stargardt's Macular Dystrophy
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
The purpose of this study is: To evaluate the safety and tolerability of RPE cellular therapy in patients with SMD . To evaluate potential efficacy endpoints to be used in future studies RPE cellula...
Detailed Description
This study is a Phase I/II, open-label, non randomized, sequential, multi-center clinical trial. There will be 5 cohorts, the 4 low vision cohorts will contain 3 patients, the better vision cohort wil...
Eligibility Criteria
Inclusion
- Adult male or female over 18 years of age. Clinical diagnosis of 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.
- Independently verified 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.
- Electrophysiological findings consistent with SMD . Medically suitable to undergo vitrectomy and subretinal injection. Medically suitable for general anaesthesia 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), and 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:
- complete history \& physical examination; dermatological screening exam for malignant lesions; negative fecal occult blood test negative chest roentgenogram (CXR); normal CBC \& manual differential; negative urinalysis (U/A);normal thyroid exam and thyroid panel; if male, normal testicular examination; if over age 40, 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 if 50 years of age or older, negative mammogram.
- If female and of childbearing potential, willing to use an effective form of birth control during the study.
- If male, willing to use barrier and spermicide contraception during the study. Willing to defer all future blood, blood component or tissue donation. Able to understand and willing to sign the informed consent.
Exclusion
- 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
Key Trial Info
Start Date :
December 13 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 30 2015
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT01469832
Start Date
December 13 2011
End Date
September 30 2015
Last Update
October 31 2024
Active Locations (3)
Enter a location and click search to find clinical trials sorted by distance.
1
Lothian Health Board Headquarters at Waverley Gate
Edinburgh, United Kingdom, EH1 3EG
2
Moorefields Eye Hospital NHS Foundation Trust
London, United Kingdom, EC1V2PD
3
Newcastle on Tyne NHS Foundation Trust
Newcastle upon Tyne, United Kingdom, NE7 7DN