Subretinal Transplantation of Retinal Pigment Epitheliums in Treatment of Age-related Macular Degeneration Diseases

• ClinicalTrials.gov Identifier: NCT02755428
• Recruitment Status: Unknown
• First Posted: April 28, 2016
• Last Update Posted: February 1, 2018
• Sponsor: Chinese Academy of Sciences
• Collaborator: Beijing Tongren Hospital
• Information provided by (Responsible Party): Qi Zhou, Chinese Academy of Sciences

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Dry Age-related Macular Degeneration	Biological: retinal pigment epithelium transplantation	Phase 1; Phase 2

Detailed Description:

This project intends to transplant human embryonic stem cells derived retinal pigment epitheliums into subretinal space to treat age-related macular degeneration(AMD).

Through the statistical analysis EDTRS, BCVA, OCT, ERG, Fluorescein angiography, Ophthalmic AB ultrasound changes between before and after the treatment to assess the efficacy and safety of RPE transplants to treat AMD disease.

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 10 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Masking Description: Open Label
• Primary Purpose: Other
• Official Title: Safety and Efficacy of Subretinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigment Epitheliums in Treatment of Age-related Macular Degeneration Diseases
• Estimated Study Start Date: January 2018
• Estimated Primary Completion Date: January 2019
• Estimated Study Completion Date: December 2020

Arms and Interventions

Arm	Intervention/treatment
Experimental: retinal pigment epithelium transplantation; Subretinal transplantation of human embryonic stem cell derived retinal pigment epitheliums.	Biological: retinal pigment epithelium transplantation; Transplant retinal pigment epithelium derived from human embryonic stem cells into subretinal space of patients with dry age-related macular degeneration(dry AMD).

Outcome Measures

Primary Outcome Measures :

1. safety and tolerance of transplantation [ Time Frame: one year ]
   The safety and tolerance of transplantation of clinical grade 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.

Secondary Outcome Measures :

1. Efficacy:Early treatment of diabetic retinopathy eye chart (ETDRs) [ Time Frame: one year ]
   Visual function measure: change in visual acuity
2. Efficacy：Best corrected visual acuity（BCVA） [ Time Frame: one year ]
   Visual function measure: change in visual acuity
3. Efficacy：Optical coherent tomography (OCT) [ Time Frame: one year ]
   Transplant and host retina integrity and survival
4. Efficacy：fundus autofluorescence [ Time Frame: one year ]
   Transplant and host retina integrity and survival

Eligibility Criteria

• Ages Eligible for Study: 55 Years to 80 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• 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;
• -8.00D<diopter<+8.00D，21mm<axis oculi≤28mm;
• voluntary as test subjects, signed informed consent, regular follow-up on time.

Exclusion Criteria:

• 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;
• Glucocorticoids or immunosuppressive drugs have been used in the last 3 months;
• Antipsychotic drugs have been used in the last 3 months, such as antidepressants, antipsychotic drugs, and so on;
• With hypersensitivity to tacrolimus or other macrolides;
• The history of addiction to alcoholism or prohibited drugs;
• Being participating in any intervention clinical trials;
• Poor compliance, difficult to complete the study;
• The person who did not receive the informed consent;
• Some researchers believe that there may be situations that can increase risks of the subjects or interfere clinical trials (for example, patients are prone to mental stress, depression, mental disorders, cognitive dysfunction, etc.).

Contacts and Locations

Contacts

Contact: Wang Liu, Doctor; +86-01064807858; wangliu@ioz.ac.cn

Contact: Hao Jie, Doctor; +86-01062558737; haojie@ioz.ac.cn

Locations

China, Beijing

Beijing Tongren Hospitol,Capital Medical University; Recruiting

Beijing, Beijing, China, 100730

Contact: Wei Wen bin, Doctor +86-01058265736 weiwenbintr@163.com

Contact: Yan Yan ni, Doctor +86-01058269804 ynyan1988@sina.com

Sponsors and Collaborators

Chinese Academy of Sciences

Beijing Tongren Hospital

Investigators

• Principal Investigator: Zhou Qi, Doctor; Institute of zoology, Chinese Academy of Sciences

More Information

• Responsible Party: Qi Zhou, The vice President and deputy director of Institute of zoology, chinese academy of sciences, Chinese Academy of Sciences
• ClinicalTrials.gov Identifier: NCT02755428
• Other Study ID Numbers: ChineseASZQ-001
• First Posted: April 28, 2016
• Last Update Posted: February 1, 2018
• Last Verified: December 2017

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Undecided

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Macular Degeneration; Retinal Degeneration; Retinal Diseases; Eye Diseases