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

NCT05445063 | Recruiting Phase 1 DMC

• 1 other identifier: 2021-214
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Macular Degeneration

Keywords

Retinal disease; Macular degeneration; Stem Cells; RPE; Safety; Efficacy

Outcome Measures

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.

  12 months

Secondary Outcomes (7)

• Best Corrected Visual Acuity (BCVA)

  12 months

• Optical coherence tomography (OCT) imaging

  12 months

• Color and autofluorescence imaging

  12 months

• Fluorescein angiography

  12 months

• Fundus autofluorescence

  12 months

Study Arms (1)

Participants receiving intervention

EXPERIMENTAL

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

Biological: Autologous iPSC-derived RPE

Interventions

Autologous iPSC-derived RPE BIOLOGICAL

Autologous transplantation of iPSC-derived RPE

Participants receiving intervention

Eligibility Criteria

• Age: 50 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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;

Sponsors & Collaborators

• Beijing Tongren Hospital: lead

Study Sites (1)

Beijing Tongren Hospitol,Capital Medical University

Beijing, Beijing Municipality, 100730, China

RECRUITING

MeSH Terms

Conditions

Macular Degeneration; Retinal Diseases

Condition Hierarchy (Ancestors)

Retinal Degeneration; Eye Diseases

Central Study Contacts

Xiaohui Zhang

CONTACT

+0086-(010)58265915; zhangxh711@126.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 5, 2022
• First Posted: July 6, 2022
• Study Start: August 1, 2022
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: July 6, 2022

Record last verified: 2022-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)