Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retin… (NCT01469832) | Clinical Trial Compass
CompletedPhase 1/2
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)
United Kingdom12 participantsStarted 2011-12-13
Plain-language 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 cellular therapy.
Who can participate
Age range18 Years
SexALL
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Inclusion Criteria:
* 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:
compl…
What they're measuring
1
safety and tolerance of transplantation
Timeframe: 12 months
Trial details
NCT IDNCT01469832
SponsorAstellas Institute for Regenerative Medicine