Safety and Efficacy of Autologous Transplantation of iPSC-RPE in the Treatment of Macular Degeneration

NCT ID: NCT05445063

Last Updated: 2022-07-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-08-31

Study Completion Date

2026-12-31

Brief Summary

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This project intends to perform autologous transplantation of induced pluripotent stem cell-derived retinal pigment epithelium (iPSC-RPE). The clinical-grade RPE will be transplanted into subretinal space to treat refractory age-related macular degeneration. The efficacy and safety of RPE transplants to treat macular degeneration will be monitored and analyzed with results from EDTRS, BCVA, OCT, ERG, microperimetry, and fluorescein angiography, before and after the treatment.

Detailed Description

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The investigators will generate iPSC lines from recruited participants and differentiate the iPSC into RPE. Autologous iPSC-derived RPE will be transplanted into participants eyes by subretinal injections. The safety and efficacy will be closely monitored and analyzed.

Conditions

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Macular Degeneration

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Participants receiving intervention

Participants will receive autologous transplantation of induced pluripotent stem cell-derived retinal pigment epitheliums.

Group Type EXPERIMENTAL

Autologous iPSC-derived RPE

Intervention Type BIOLOGICAL

Autologous transplantation of iPSC-derived RPE

Interventions

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Autologous iPSC-derived RPE

Autologous transplantation of iPSC-derived RPE

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Aged 50-75 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 um geographic atrophy in the fovea;
* Clinical diagnosis is wet AMD, but no obvious efficacy after conventional treatment;
* The BCVA of the target eye will be 0.05 to 0.3;
* Voluntary as test subjects, informed consent, regular follow-up on time.

Exclusion Criteria

* One-eyed subjects;
* Macular atrophy caused by other diseases in addition to AMD;
* Suffer from retinitis pigmentosa, choroidal retinitis, central serous choroiditis, 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 histories;
* Other intraocular surgery histories besides cataract surgery;
* Combined with severe systemic diseases, such as heart failure, liver disease, renal insufficiency, cor pulmonale, COPD in the previous 12 months;
* Combined with severe infectious diseases, such as HIV, HBV, HCV, syphilis, tuberculosis, etc;
* Abnormal blood coagulation function or other laboratory tests;
* If female and of childbearing potential, pregnant, breastfeeding, or planning to become pregnant through the study;
* If male, refuse to use barrier and spermicide contraception during the study;
* Malignant tumor and history of malignancy;
* Any immune deficiency;
* Allergy to tacrolimus or other macrolides;
* Any immune deficiency;
* Use glucocorticoids, immunosuppressive drugs, or antipsychotic drugs in the previous 3 months;
* Use anticoagulant, or the platelet function is still not restored to normal after stopping antiplatelet drugs for 10 days;
* A history of addiction to alcoholism or prohibited drugs;
* Be participating in other intervention clinical trials or receiving other study medications;
* Poor compliance, difficulty to complete the study, or refusal to informed consent;
* Some other situations which might increase the risks of the subjects or interfere with clinical trials, such as mental disorders, cognitive dysfunction, etc.
Minimum Eligible Age

50 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Beijing Tongren Hospital

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Beijing Tongren Hospitol,Capital Medical University

Beijing, Beijing Municipality, China

Site Status RECRUITING

Countries

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China

Central Contacts

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Xiaohui Zhang

Role: CONTACT

+0086-(010)58265915

Facility Contacts

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Zi-Bing Jin, Doctor

Role: primary

+0086-(010)58265913

Other Identifiers

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2021-214

Identifier Type: -

Identifier Source: org_study_id

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