Safety and Efficacy of Subretinal Transplantation of Clinical Human Embryonic Stem Cell Derived Retinal Pigment Epitheliums in Treatment of Retinitis Pigmentosa

NCT03944239 | Unknown Phase 1

• 1 other identifier: ChineseASZQ-002
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

This project intends to transplant a clinical level human embryonic stem cells derived retinal pigment epithelium into subretinal space to treat eetinitis pigmentosa(RP) diseases. 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 0f RPE transplants to treat RP disease.

Conditions

Retinitis Pigmentosa

Outcome Measures

Primary Outcomes (1)

• Adverse events and serious adverse events occurred within 1 year after transplantation

  The occurrence of adverse events (AE), severe adverse events (SAE), treatment-related adverse events (TEAE), and clinically significant vital signs during the study period.

  one year

Secondary Outcomes (1)

• Improvement of visual function

  one year

Study Arms (1)

retinal pigment epitheliums transplantation

EXPERIMENTAL

Transplant clinical-grade hESC derived retinal pigment epitheliums into subretinal of patients with retinitis pigmentosa.The dosage is 150000.

Biological: Retinal pigment epitheliums transplantation

Interventions

Retinal pigment epitheliums transplantation BIOLOGICAL

Subretinal transplantation of clinical human embryonic stem cell derived retinal pigment epitheliums.

retinal pigment epitheliums transplantation

Eligibility Criteria

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

You may qualify if:

• Age above 18 and under 80 years of age, both gender, in good health.
• Clinical diagnosis of retinitis pigmentosa, the results of eye electrophysiological examination accord with retinitis pigmentosa.
• The BCVA of target eye will not be better than 20/400.
• The BCVA of contralateral eye is not worse than 20/400.
• Diopter is smaller than 8.00 D, axial 28 mm or less.
• Voluntary as test subjects, signed informed consent, regular follow-up will be effected according to the specified time.

You may not qualify if:

• Subject with other additional retinal diseases, like diabetic or hypertensive retinopathy vascular diseases.
• Target eye ever have had a eye surgery;
• Subject with active serious diseases of the digestive system, liver kidney impairment (ALT/AST) \> 1.5 or any known liver disease, creatine \> 1.3 mg/dL) and/serious temic diseases, such as cardiovascular tem, respiratory tem, nervous tem, endocrine tem urogenital tem diseases, etc.), the history of malignancy;
• Have been ready for pregnancy during test, are lactating women;
• Ready to birth of men during the test;
• Subject with any immunodeficiency;
• Subject in the immunosuppressive therapy in the current;
• Subject with the tacrolimus or other large ring lactone class drug allergies;
• Participate in any clinical subjects in nearly six months;
• Has a history of alcohol or illicit drug abuse;
• Poor adherence to complete studies;
• Researchers believe that there may be an increased risk subjects or interfere with the clinical trials of any situation (such as when a patient is prone to mental tension, depression, mental illness, cognitive dysfunction, etc.).

Sponsors & Collaborators

• Qi Zhou: lead
• Beijing Tongren Hospital: collaborator

Study Sites (1)

Beijing Tongren Hospitol,Capital Medical University

Beijing, Beijing Municipality, 100730, China

RECRUITING

MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

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
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: 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: December 27, 2018
• First Posted: May 9, 2019
• Study Start: May 1, 2020
• Primary Completion: October 1, 2021
• Study Completion: December 1, 2021
• Last Updated: March 26, 2020

Record last verified: 2020-03

Locations

CN (1)