Back to resultsTreatment of Age-related Macular Degeneration by Fetal Retinal Pigment Epithelial Cells Transplantation
Primary Purpose
Age Related Macular Degeneration, Macular Degeneration, AMD
Status
Unknown status
Phase
Early Phase 1
Locations
China
Study Type
Interventional
Intervention
fRPE cells
Sponsored by
About this trial
This is an interventional treatment trial for Age Related Macular Degeneration
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of dry AMD or non-exudative AMD (geographical atrophy>250μm, involving central fovea of macular; or AMD with fibrous disciform scar and maintained stationary for at least 3 months)
- Experimental eye with best-corrected visual acuity (BCVA) of no more than 20/400 and control eye with BCVA of more than or equal to 20/400
- Patients are in good state
- Patient understand and sign the consent form.
Exclusion Criteria:
- Blood routine examination is abnormal (hemoglobin<10gm/dL; blood platelet count<100k/mm³; neutrophil count<1000/mm³)
- Blood biochemistry is abnormal (ALT／AST>1.5; creatinine>1.3mg/dL)
- Experimental eye has optic nerve atrophy caused by glaucoma
- Patients need cataract surgery within a year
- Patients have received cataract surgery and other ocular surgery in recent 3 months
- Experimental eye has retinal detachment, or has received retinal detachment surgery.
- Patients with uveitis and other endophthalmitis
- Patients with other ocular disease affecting vision.
- Patients have participated in clinical study of ocular or systemic drug use in recent 6 months.
- Patients with medical history of malignant cancer (except resected basal cell carcinoma and squamous-cell carcinoma).
- Patients with medical history of myocardial infarction
- Patient with diabetes
- Patient with Parkinson disease or Alzheimer's disease
- Patients are under the treatment of immunosuppressive agent (except intermittent, low-dose corticosteroid treatment).
- Patients with other medical conditions that restricts the compliance and safety of patients, or affects experimental results.
Sites / Locations
- The First Affiliated Hospital with Nanjing Medical University
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
fRPE cells
Arm Description
Subretinal transplantation of fRPE cells in experimental eye
Outcomes
Primary Outcome Measures
Number of subjects with adverse events
Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.
Secondary Outcome Measures
Assessment of standardized ETDRS acuity testing
ETDRS stands for Early Treatment Diabetic Retinopathy Study which is designed to accurately measure visual acuity via ETDRS Scoring.
Assessment of visual function changes
Assessments will include fluorescein angiography, fundus photography, spectral domain ocular coherence tomography (SD-OCT), microperimetry and multifocal electroretinography.
Assessment of systemic condition
Items will include vital signs, electrocardiogram, blood and urine routine examination, tumor marker.
Full Information
NCT ID
NCT02868424
First Posted
August 8, 2016
Last Updated
June 14, 2017
Sponsor
The First Affiliated Hospital with Nanjing Medical University
1. Study Identification
Unique Protocol Identification Number
NCT02868424
Brief Title
Treatment of Age-related Macular Degeneration by Fetal Retinal Pigment Epithelial Cells Transplantation
Official Title
Early Phase I Study of the Safety and Preliminary Efficacy of Human Fetal Retinal Pigment Epithelial(fRPE) Cells Subretinal Transplantation in Age-Related Macular Degeneration(AMD) Patients
Study Type
Interventional
2. Study Status
Record Verification Date
May 2017
Overall Recruitment Status
Unknown status
Study Start Date
February 13, 2016 (Actual)
Primary Completion Date
August 2018 (Anticipated)
Study Completion Date
December 2018 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
The First Affiliated Hospital with Nanjing Medical University
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Early Phase I Study of the Safety and Preliminary Efficacy of Human Fetal Retinal Pigment Epithelial(fRPE) Cells Subretinal Transplantation in Age-Related Macular Degeneration(AMD) Patients
Detailed Description
This study is a perspective, single-arm and open-labeled investigation of the safety and preliminary efficacy of unilateral subretinal transplantation of fRPE cells in subjects with dry Age-Related Macular Degeneration and non-exudative AMD. The investigators will recruit and enroll 6 patients based on specific inclusive/exclusive criteria. Experimental and self-controlled eye will be determined based on best-corrected visual acuity (BCVA). The eye with BCVA of no more than 20/400 will be determined as experimental eye, which will be divided into 3 groups and undergo subretinal injection of 3 different dosages of fRPE cells (100.000, 200,000 or 500,000) respectively, while the other one (BCVA of more than or equal to 20/400) as control eye, will not receive the surgery.
fRPE cells will be obtained from the fetuses aborted in the Department of Obstetrics and Gynecology in Jiangsu Province Hospital from 2015 to 2018. The obtained fRPE cells will meet specific inclusive/exclusive criteria and conform to Good manufacturing practices (GMP). fRPE will be transplanted by a board-certified vitreoretinal surgeon, which will be administered into the subretinal space of experimental eye through a standard surgical approach.
Immunosuppressive agents will be administered orally to all subjects after transplantation. Dosage and time duration of immunosuppressive agents will be regulated strictly relying on the condition of immune rejection. Subjects will be monitored with ophthalmologic and systemic examinations frequently at regular post-transplant intervals after fRPE cell transplantation.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Age Related Macular Degeneration, Macular Degeneration, AMD
7. Study Design
Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)
8. Arms, Groups, and Interventions
Arm Title
fRPE cells
Arm Type
Experimental
Arm Description
Subretinal transplantation of fRPE cells in experimental eye
Intervention Type
Drug
Intervention Name(s)
fRPE cells
Other Intervention Name(s)
human fetal retinal pigment epithelial cells
Intervention Description
fRPE cells transplantation directly into the subretinal space of one eye (experimental eye) in a single transplant procedure
Primary Outcome Measure Information:
Title
Number of subjects with adverse events
Description
Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.
Time Frame
Two years following transplantation
Secondary Outcome Measure Information:
Title
Assessment of standardized ETDRS acuity testing
Description
ETDRS stands for Early Treatment Diabetic Retinopathy Study which is designed to accurately measure visual acuity via ETDRS Scoring.
Time Frame
Two years following transplantation
Title
Assessment of visual function changes
Description
Assessments will include fluorescein angiography, fundus photography, spectral domain ocular coherence tomography (SD-OCT), microperimetry and multifocal electroretinography.
Time Frame
Two years following transplantation
Title
Assessment of systemic condition
Description
Items will include vital signs, electrocardiogram, blood and urine routine examination, tumor marker.
Time Frame
Two years following transplantation
10. Eligibility
Sex
All
Minimum Age & Unit of Time
50 Years
Maximum Age & Unit of Time
90 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Inclusion Criteria:
Diagnosis of dry AMD or non-exudative AMD (geographical atrophy>250μm, involving central fovea of macular; or AMD with fibrous disciform scar and maintained stationary for at least 3 months)
Experimental eye with best-corrected visual acuity (BCVA) of no more than 20/400 and control eye with BCVA of more than or equal to 20/400
Patients are in good state
Patient understand and sign the consent form.
Exclusion Criteria:
Blood routine examination is abnormal (hemoglobin<10gm/dL; blood platelet count<100k/mm³; neutrophil count<1000/mm³)
Blood biochemistry is abnormal (ALT／AST>1.5; creatinine>1.3mg/dL)
Experimental eye has optic nerve atrophy caused by glaucoma
Patients need cataract surgery within a year
Patients have received cataract surgery and other ocular surgery in recent 3 months
Experimental eye has retinal detachment, or has received retinal detachment surgery.
Patients with uveitis and other endophthalmitis
Patients with other ocular disease affecting vision.
Patients have participated in clinical study of ocular or systemic drug use in recent 6 months.
Patients with medical history of malignant cancer (except resected basal cell carcinoma and squamous-cell carcinoma).
Patients with medical history of myocardial infarction
Patient with diabetes
Patient with Parkinson disease or Alzheimer's disease
Patients are under the treatment of immunosuppressive agent (except intermittent, low-dose corticosteroid treatment).
Patients with other medical conditions that restricts the compliance and safety of patients, or affects experimental results.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Qinghuai Liu, M.D.
Organizational Affiliation
The First Affiliated Hospital with Nanjing Medical University
Official's Role
Principal Investigator
Facility Information:
Facility Name
The First Affiliated Hospital with Nanjing Medical University
City
Nanjing
State/Province
Jiangsu
ZIP/Postal Code
210029
Country
China
12. IPD Sharing Statement
Plan to Share IPD
Yes
Learn more about this trial
Treatment of Age-related Macular Degeneration by Fetal Retinal Pigment Epithelial Cells Transplantation
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