A Phase I/IIa, Open-Label, Single-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial(MA09-hRPE) Cells in Patients With Advanced Dry Age-related Macular Degeneration(AMD)
The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified August 2012 by CHABiotech CO., Ltd.
Recruitment status was: Recruiting
Information provided by (Responsible Party):
CHABiotech CO., Ltd
First received: August 22, 2012
Last updated: October 22, 2012
Last verified: August 2012
To evaluate the safety and tolerability of MA09-hRPE cellular therapy in patients with advanced dry AMD To evaluate the safety of the surgical procedures when used to implant MA09-hRPE cells To assess the number of hRPE cells to be transplanted in future studies To evaluate on an exploratory basis potential efficacy endpoints to be used in future studies of MA09-hRPE cellular therapy.
Dry Age Related Macular Degeneration
||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
||A Phase I/IIa, Open-Label, Single-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial(MA09-hRPE) Cells in Patients With Advanced Dry Age-related Macular Degeneration(AMD)
Primary Outcome Measures:
- Safety of hESC derived RPE cells [ Time Frame: 12 months ]
The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe 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
Secondary Outcome Measures:
- exploratory evaluations for potential efficacy endpoints [ Time Frame: 12months ]
Secondary endpoints will be evaluated as exploratory evaluations for potential efficacy endpoints.
- Change in the mean of BCVA
- Autofluorescense photography
- Reading speed
Evidence of successful engraftment will consist of:
- Structural evidence (OCT imaging, fluorescein angiography, slitlamp examination with fundus photography) that cells have been implanted in the correct location
- Electroretinographic evidence (mfERG) showing enhanced activity in the implant location
| Estimated Enrollment:
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||February 2016 (Final data collection date for primary outcome measure)
Experimental: Biological: MA09-hRPE Cellular therapy
Biological: MA09-hRPE Cellular therapy
Cohort 1 50,000 cells
Cohort 2 100,000 cells
Cohort 3 150,000 cells
Cohort 4 200,000 cells
- Cohort 1- 50,000 MA09-hRPE cells transplanted
- Cohort 2- 100,000 MA09-hRPE cells transplanted
- Cohort 3- 150,000 MA09-hRPE cells transplanted
- Cohort 4- 200,000 MA09-hRPE cells transplanted
|Ages Eligible for Study:
||55 Years and older (Adult, Senior)
|Sexes Eligible for Study:
|Accepts Healthy Volunteers:
- 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 (BCVA) of the eye to receive the transplant will be no better than 20/400.
- The visual acuity (BCVA) of the eye that is NOT to receive the transplant will be no worse than 20/400.
- 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.
Normal serum chemistry complete blood count [CBC], prothrombin time [PT], and activated partial thromboplastin time [aPTT] Negative urine screen for drugs of abuse. Negative human immunodeficiency virus (HIV), hepatitis B (HBV), hepatitis C (HCV) serologies.
No history of malignancy
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.
If male, willing to use barrier and spermicidal 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
- Presence of active or inactive CNV in the eye to be treated.
- Presence or history of retinal dystrophy, retinitis pigmentosa, chorioretinitis, central serious choroidopathy, diabetic retinopathy or other retinal vascular or degenerative disease other than ARMD.
- History of optic neuropathy.
- Macular atrophy due to causes other than AMD.
- Presence of glaucomatous optic neuropathy in the study eye, uncontrolled IOP, or use of two or more agents to control IOP (acetazolamide, beta blocker, alpha-1-agonist, prostaglandins, anhydrous carbonic inhibitors)
- Cataract of sufficient severity likely to necessitate surgical extraction within 1 year.
- History of retinal detachment repair in the study eye.
- Axial myopia of greater than -8 diopters
- Axial length greater than 28 mm.
- History of myocardial infarction in previous 12 months.
- History of diabetes mellitus.
- History of cognitive impairments or dementia which may impact the patients ability participate in the informed consent process and to appropriately complete evaluations.
- Any immunodeficiency.
- Any current immunosuppressive therapy other than intermittent or low dose corticosteroids.
- Alanine transaminase/aspartate aminotransferase (ALT/AST) >1.5 times the upper limit of normal or any known liver disease.
- Renal insufficiency, as defined by creatine level >1.3 mg/dL.
- A hemoglobin concentration of less than 10 gm/dL, a platelet count of less than 100k/mm3 or an absolute neutrophil count of less than 1000/mm3 at study entry.
- 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 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.
- Ocular surgery in the study eye in the previous 3 months
- If female, pregnancy or lactation.
- Any other medical condition, which, in the Investigator's judgment, will interfere with the patient's ability to comply with the protocol, compromises patient safety, or interferes with the interpretation of the study results.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01674829
|CHA Bundang Medical Center
|Seongnam-si, Gyeonggi-do, Korea, Republic of, 463-712, |
|Contact: Wonkyung Song, MD. PhD. 82-31-780-5479 |
|Principal Investigator: Wonkyung Song, MD. PhD |
CHABiotech CO., Ltd
||Wonkyung Song, MD. PhD.
||CHA Bundang Medical Center
||CHABiotech CO., Ltd
History of Changes
|Other Study ID Numbers:
CHA CTP 1101
|Study First Received:
||August 22, 2012
||October 22, 2012
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on August 17, 2017