Treatment of Dry Age Related Macular Degeneration Disease With Retinal Pigment Epithelium Derived From Human Embryonic Stem Cells

NCT03046407 | Unknown Phase 1 DMC

• 1 other identifier: ChineseASZQ-004
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

This project intends to transplant human embryonic stem cells derived retinal pigment epitheliums into subretinal space of patients to treat dry age-related macular degeneration(dry-AMD).And we will assess the safety and efficacy of RPE transplants to treat dry AMD.

Conditions

Dry Age-related Macular Degeneration

Outcome Measures

Primary Outcomes (1)

• safety and tolerance of transplantation

  The safety and tolerance of transplantation of hESC-derived RPE will be considered safe: no above moderate adverse events or severe adverse events which related to transplantation of retinal pigment epithelial cells ; Cells without infectious; No tumorigenicity. Through the clinical signs of subjects and laboratory examination to judge the tolerance, integrity, repellency of RPE cells, and monitoring the presence of local or systemic infection, and presence of metastatic tumor cells.

  12 months

Secondary Outcomes (5)

• Efficacy:Early treatment of diabetic retinopathy eye chart (ETDRs)

  12 months

• Efficacy：Best corrected visual acuity（BCVA）

  12 months

• Efficacy：Optical coherent tomography (OCT)

  12 months

• Efficacy：fundus autofluorescence

  12 months

• Efficacy：vision inspection

  12 months

Study Arms (1)

retinal pigment epithelium transplantation

EXPERIMENTAL

transplant retinal pigment epithelium derived from human embryonic stem cells into subretinal space of patients with dry age-related macular degeneration(dry AMD).

Biological: retinal pigment epithelium transplantation

Interventions

retinal pigment epithelium transplantation BIOLOGICAL

Transplant retinal pigment epithelium derived from human embryonic stem cells into subretinal space of patients with dry age-related macular degeneration(dry AMD).

retinal pigment epithelium transplantation

Eligibility Criteria

• Age: 55 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Aged 55-80 years;
• Clinical diagnosis is consistent with the definition of late dry AMD in the age-related eye disease study (AREDS) (with one or more \>250 micron geographic atrophy in the fovea;
• No CNV;
• The BCVA of target eye will not be better than 20/200;
• D\<diopter\<+8.00D，21mm\<axis oculi≤28mm;
• voluntary as test subjects, signed informed consent, regular follow-up on time.

You may not qualify if:

• The macular atrophy caused by other diseases in addition to AMD;
• Suffer from retinitis pigmentosa, choroidal retinitis, central serous chorositis, diabetic retinopathy or other retinal vascular and degenerative diseases besides AMD;
• Lens opacities (affecting the central vision), glaucoma, uveitis, retinal detachment, optic neuropathy and other ocular history;
• Other intraocular surgery history besides cataract surgery;
• In the last 6 months, there were severe heart failure (New York Heart Association grade III and IV) or the left ventricular ejection fraction \<35% in any examinations
• One of the following circumstances: (1) dialysis or eGFR\<20ml/min/1.73m2; (2) urinary protein / urinary creatinine is ≥1g/g; (3) creatinine or albumin / urinary creatinine is ≥600mg/g;
• Chronic liver disease, ALT increased \>3 times normal value of the upper limit;
• Combined with other serious systemic diseases, such as cor pulmonale, severe COPD (FEV1%\<50%) and so on;
• Combined with severe infectious diseases (such as HIV, syphilis antibody positive, etc;
• The quantitative detection of HCV-RNA was positive, the quantitative detection of HBV-DNA was greater than 103 IU/ml, and tuberculosis was in the contagious period, etc;
• Patients who are using anticoagulant, or the platelet function is still not restored to normal after stopping antiplatelet drugs for 10 days（The results of VerifyNow test show that AUC is greater than 470 and PRU is more than 208）;
• Abnormal blood coagulation function or other obvious abnormal laboratory test results;
• Malignant tumor and history of malignant tumor;
• Women who are pregnant，prepare to be pregnant during the trial, be lactating；men who prepare to have baby during the trial;
• Any immune deficiency;

Sponsors & Collaborators

• Chinese Academy of Sciences: lead
• The First Affiliated Hospital of Zhengzhou University: collaborator

Study Sites (1)

The first affiliated hospital of Zhengzhou university

Zhengzhou, Henan, 450052, China

RECRUITING

Study Officials

• Zhou Qi, Doctor

  Institute of Zoology, Chinese Academy of Sciences

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Wang Liu, Doctor

CONTACT

+86-01064807858; wangliu@ioz.ac.cn

Hao Jie, Doctor

CONTACT

+86-01062558737; haojie@ioz.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Masking Details: Open Label
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER GOV
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Deputy director of Institute of zoology, chinese academy of sciences, and vice president of medical school， University of chinese academy of sciences

Study Record Dates

• First Submitted: February 2, 2017
• First Posted: February 8, 2017
• Study Start: September 6, 2017
• Primary Completion: January 1, 2019
• Study Completion: December 1, 2020
• Last Updated: January 31, 2018

Record last verified: 2017-12

Locations

CN (1)