Treatment of Age-related Macular Degeneration by Fetal Retinal Pigment Epithelial Cells Transplantation

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

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 Overview

• Status: Unknown
• Conditions: Macular Degeneration; Age Related Macular Degeneration; AMD
• Intervention / Treatment: Drug: fRPE cells

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.

• Study Type: Interventional
• Enrollment (Actual): 6
• Phase: Early Phase 1

Contacts and Locations

Study Locations

• China
  • Jiangsu
    • Nanjing, Jiangsu, China, 210029
      • The First Affiliated Hospital with Nanjing Medical University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 50 years to 90 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: fRPE cells; Subretinal transplantation of fRPE cells in experimental eye	Drug: fRPE cells; fRPE cells transplantation directly into the subretinal space of one eye (experimental eye) in a single transplant procedure; Other Names: human fetal retinal pigment epithelial cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of subjects with adverse events	Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.	Two years following transplantation

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Two years following transplantation
Assessment of visual function changes	Assessments will include fluorescein angiography, fundus photography, spectral domain ocular coherence tomography (SD-OCT), microperimetry and multifocal electroretinography.	Two years following transplantation
Assessment of systemic condition	Items will include vital signs, electrocardiogram, blood and urine routine examination, tumor marker.	Two years following transplantation

Collaborators and Investigators

• Sponsor: The First Affiliated Hospital with Nanjing Medical University
• Investigators: Principal Investigator: Qinghuai Liu, M.D., The First Affiliated Hospital with Nanjing Medical University

Study record dates

Study Major Dates

• Study Start (Actual): February 13, 2016
• Primary Completion (Anticipated): August 1, 2018
• Study Completion (Anticipated): December 1, 2018

Study Registration Dates

• First Submitted: August 8, 2016
• First Submitted That Met QC Criteria: August 14, 2016
• First Posted (Estimate): August 16, 2016

Study Record Updates

• Last Update Posted (Actual): June 16, 2017
• Last Update Submitted That Met QC Criteria: June 14, 2017
• Last Verified: May 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Macular Degeneration
• Other Study ID Numbers: JSPH-fRPE-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes