Clinical trial NCT03944239: Safety and Efficacy of Subretinal Transplantation of Clinical Human Embryonic Stem Cell Derived Retinal Pigment Epitheliums in Treatment of Retinitis Pigmentosa
Official title: Safety and Efficacy of Subretinal Transplantation of Clinical Human Embryonic Stem Cell Derived Retinal Pigment Epitheliums in Treatment of Retinitis Pigmentosa
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Retinitis Pigmentosa
Lead sponsor: Chinese Academy of Sciences
Sponsor class: OTHER_GOV
Collaborators: name: Beijing Tongren Hospital
First posted: 2019-05-09
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.).

Primary outcomes:
measure: Adverse events and serious adverse events occurred within 1 year after transplantation
timeFrame: one year
Secondary outcomes:
measure: Improvement of visual function
timeFrame: one year
Other outcomes:
Locations:
city: Beijing
country: China
facility: Beijing Tongren Hospitol,Capital Medical University
state: Beijing
Study officials:
affiliation: Institute of Zoology, Chinese Academy of Sciences
name: Zhou Qi, Doctor
role: PRINCIPAL_INVESTIGATOR
References: