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RecruitingNCT05445063Updated Jul 6, 2022

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

A Phase 1 interventional study of Autologous iPSC-derived RPE in Macular Degeneration, sponsored by Beijing Tongren Hospital. Recruiting at 1 site in China. Open to participants aged 50 Years to 75 Years. Per ClinicalTrials.gov, last updated 2022-07-06.

Sponsored by Beijing Tongren Hospital · Phase 1, Interventional, and Treatment

From the registry’s dates

  • Primary completion was expected by Jul 2026, 3 months ago, but the record still lists the study as recruiting.
Phase
Phase 1
Study type
Interventional
Enrollment
10
Allocation
Not applicable
Ages
50 Years to 75 Years
Sex
All
01

Study summary

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.

Read the detailed description

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.

02

Conditions studied

  • Macular Degeneration

Keywords

  • Retinal disease
  • Macular degeneration
  • Stem Cells
  • RPE
  • Safety
  • Efficacy
03

In context

Macular Degeneration

1,474 studies on the registry are indexed under Macular Degeneration; 206 are open to participants now.

This study's planned enrollment of 10 is below the median of 51 across 985 interventional studies indexed under Macular Degeneration.

Browse Macular Degeneration studies →

Lead sponsor

Beijing Tongren Hospital is the lead sponsor of 154 studies on the registry; 49 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
50 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
10 participants (estimated)

Study arms

  • Experimental
    Participants receiving intervention

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

    Biological: Autologous iPSC-derived RPE

Interventions

  • BiologicalAutologous iPSC-derived RPE

    Autologous transplantation of iPSC-derived RPE

06

What researchers measure

Primary outcomes

  1. Safety measure

    Safety will be assessed by Adverse Events (AEs) of special interest in regards to the investigational product. This will include obtaining information about Serious Adverse Events (SAEs) that are neurologic, infectious, hematologic or fatal, any AE that causes the subject to withdraw from the study, any new diagnosis of an ocular or immune-mediated disorder, cancer (irrespective of prior history), ectopic or proliferative cell growth (RPE or non-RPE) with adverse clinical consequence, unexpected, clinically significant AE possibly related to the cell transplant procedure or the investigational product (autologous iPSC-RPE), pregnancy in a female subject or the partner of a male subject and pregnancy outcome.

    Time frame: 12 months

Secondary outcomes

  1. Best Corrected Visual Acuity (BCVA)

    Change in visual acuity will be measured by Early Treatment Diabetic Retinopathy Study (ETDRS) chart.

    Time frame: 12 months

  2. Optical coherence tomography (OCT) imaging

    The number of patients with serious retinal detachment, retinal hemorrhage, and cystoid macular edema, and the change in target treatment areas.

    Time frame: 12 months

  3. Color and autofluorescence imaging

    Change in target treatment areas.

    Time frame: 12 months

  4. Fluorescein angiography

    Change in target treatment areas.

    Time frame: 12 months

  5. Fundus autofluorescence

    Change in target treatment areas.

    Time frame: 12 months

  6. Microperimetry

    Exploratory evaluations for the change of retinal sensitivity from baseline in the region of interest.

    Time frame: 12 months

  7. Electroretinography (ERG)

    Exploratory evaluations for the change of retinal electrophysiology responses from baseline.

    Time frame: 12 months

07

Study locations

1 of 1 sites recruiting
  • Beijing Tongren Hospitol,Capital Medical University
    Beijing, Beijing 100730, China
    Recruiting
08

References and documents

Individual participant data

Plan to share: No — Detailed plan will be made based on institute regulations and funder policies. The investigators will adjust the plan case-by-case accordingly.

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 6, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT05445063
Lead sponsor
Beijing Tongren Hospital
Responsible party
Sponsor
First posted
Jul 6, 2022
Start date
Aug 2022 (estimated)
Primary completion
Jul 2026 (estimated)
Completion
Dec 2026 (estimated)
Last update
Jul 6, 2022

Study contacts

Xiaohui Zhang
Contact
zhangxh711@126.com
+0086-(010)58265915

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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