AMD - Age-Related Macular DegenerationPhase 1/2NCT07418177
Study in Human iPSCs-derived RPE Cells Transplantation for Advanced (Late-stage) AMD
A Phase 1/2 study testing hiPSC-RPEs in people with AMD - Age-Related Macular Degeneration, run by The First Affiliated Hospital with Nanjing Medical University at 1 site in China.
Trial summary
Phase I Study of the Safety and Preliminary Efficacy of Human induced pluripotent stem cells-derived Retinal Pigment Epithelial Cells (hiPSC-RPEs) Subretinal Transplantation in Adanced (Late-Stage) Age-Related Macular Degeneration(AMD) Patients. Read more about this study
- Who is it for?
- Ages 50 to 85 years
- Conditions the study is for
- AMD - Age-Related Macular Degeneration
- What is being tested?
- hiPSC-RPEs
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 14 Oct 2025to 31 Dec 2028
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- (1)Age ≥50 years and ≤85 years, regardless of gender.
- (2)Patients with atrophic AMD in the macular area (geographic atrophy >250μm, involving the foveal center); or inactive neovascular AMD patients who have undergone treatment for wet AMD or have naturally converted to non-exudative AMD (referring to patients with fibrovascular scar formation, who are unresponsive to anti-VEGF treatment, and have no macular edema on OCT and/or no fluorescein leakage on FFA), with a scar diameter not exceeding 6 PD.
- (3)Best-corrected visual acuity (BCVA) of the study eye ≥5 letters and ≤34 letters (using the ETDRS visual acuity chart).
- (4)If both eyes meet the criteria, the more severely affected eye will be selected; if the severity is the same in both eyes, the right eye will be designated as the study eye.
- (5)The patient or their legal representative has signed a written informed consent form.
Who cannot join
- Ocular Diseases:
- (1)Vitreous hemorrhage in the study eye within 2 months prior to screening.
- (2)Presence of a scar or fibrosis involving the fovea in the study eye, or a peripheral scar area greater than 6 PD, indicating severe irreversible visual loss.
- (3)Presence of pseudoexfoliation syndrome, central retinal vein occlusion, intraocular hemorrhage causing visual loss, rhegmatogenous retinal detachment, macular hole, or active choroidal neovascularization in the study eye.
- (4)Afferent pupillary defect (APD) in the study eye.
- (5)Significant media opacity in the study eye (excluding lens opacities) that may interfere with visual function assessment or fundus examination.
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- (6)Acute infectious inflammation in either eye during the screening period.
- (7)Patients with intraocular pressure higher than 25 mmHg despite medical treatment.
- (8)Monocular blind patients.
- Ocular Treatments:
- (9)The study eye has undergone focal/grid laser photocoagulation within 3 months prior to screening.
- (10)The study eye has undergone any intraocular surgery within 3 months prior to screening.
- (11) Any eye has received anti-angiogenic therapy (including any anti-VEGF agents, e.g., Aflibercept [Eylea®], Ranibizumab [Lucentis®], Bevacizumab [Avastin®]) within 1 month prior to screening; or has undergone cataract surgery within 1 month prior to screening with unstable postoperative ocular conditions.
- Systemic Diseases, Treatments, and Other Conditions:
- (12)Patients with an allergic reaction or history of allergy to sodium fluorescein and indocyanine green, a history of allergy to protein products used for treatment or diagnosis, and patients with allergies to two or more drugs and/or non-drug factors, or those currently suffering from allergic diseases.
- (13)Patients with uncontrolled diabetes (fasting blood glucose ≥7.0 mmol/L or 2-hour postprandial blood glucose ≥11.1 mmol/L).
- (14)Patients with a history of surgical procedures within 1 month prior to screening, or those with unhealed wounds, ulcers, fractures, etc.
- (15)Patients with uncontrolled hypertension (defined as a single measurement of systolic blood pressure >180 mmHg, two consecutive measurements of systolic blood pressure >160 mmHg, or diastolic blood pressure >100 mmHg despite optimal treatment).
- (16)Patients with a history of myocardial infarction within 6 months prior to enrollment.
- (17)Patients with active disseminated intravascular coagulation or a significant tendency to bleed; patients who have used anticoagulant or antiplatelet aggregation drugs within 14 days prior to screening, except for aspirin/nonsteroidal anti-inflammatory drugs.
- (18) Presence of any uncontrolled systemic clinical conditions, such as interstitial pneumonia, severe psychiatric, neurological, cardiovascular, or respiratory diseases, malignant tumors, Parkinson's disease, Alzheimer's disease, or other severe systemic disorders.
- (19)Women of childbearing age with a positive pregnancy test during the screening period. Women who wish to breastfeed during the study period, and all patients of childbearing age (male and female) who do not agree to take effective contraceptive measures (such as intrauterine devices, oral contraceptives, or condoms) throughout the study period and for 30 days after the end of the visit period.
- (20)Patients who have participated in or are currently participating in other clinical studies within 30 days prior to screening.
- (21)Hemoglobin <100 g/L, platelets ≤100×10^9/L, neutrophil count <1.0×10^9/L, ALT/AST >1.5×upper limit of normal (ULN), creatinine >1.3 mg/dL.
- (22)Subjects with any clinically significant finding in virological tests (HIV, HBV, HCV and TPPA).
- (23)Patients deemed unsuitable for inclusion by the investigator, or other medical conditions that limit the subject's compliance, safety, or affect the results of the trial results.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital with Nanjing Medical University
Nanjing, Jiangsu, 210029
Yue Wang, +86 13405818362
About this study
Official title: Study in Human iPSCs-derived Retinal Pigment Epithelium Cells Transplantation for Advanced (Late-stage) Age-Related Macular Degeneration
Phase I Study of the Safety and Preliminary Efficacy of Human induced pluripotent stem cells-derived Retinal Pigment Epithelial Cells (hiPSC-RPEs) Subretinal Transplantation in Adanced (Late-Stage) Age-Related Macular Degeneration(AMD) Patients.
- Conditions
- AMD - Age-Related Macular Degeneration
- Being tested
- hiPSC-RPEs
- Keywords
- Age-Related Macular Degeneration, AMD, hiPSC-RPEs, geographic atrophy, wet AMD
- Medical subject headings
- Macular Degeneration, Geographic Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hiPSC-RPEsBiological
Also called HiO-630
hiPSC-RPEs transplantation directly into the subretinal space of one eye (experimental eye) in a single transplant procedure
| Study group | Role | What they receive |
|---|---|---|
| HiO-630 Dose-Escalation Cohorts | Experimental | Participants will receive a single subretinal injection of HiO-630. Three ascending dose cohorts are included: 200,000 cells/eye (low dose), 400,000 cells/eye (mid dose), and 600,000 cells/eye (high dose). Enrollment into the next higher-dose cohort will commence upon completion of safety assessment for the preceding cohort. Receives: Biological: hiPSC-RPEs |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and Severity of Treatment-Related Adverse Events
Any adverse reactions/adverse events (AEs) related to hiPSC-RPEs treatment occurring within 2 years after the completion of hiPSC-RPEs administration in all subjects, and their severity (graded according to CTCAE criteria).
Measured: Within 2 years after administration
Secondary outcomes (2)
Assessment of visual acuity using the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart.
Best-corrected visual acuity (BCVA) of the study eye and fellow eye will be measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart. Subjects wearing soft contact lenses shall remove the lenses and wait for 30 minutes prior to the examination.
Under standard illumination conditions, subjects wear their best refractive correction, occlude the non-tested eye, and identify letters line-by-line at a distance of 4 meters. One point is awarded for each correctly identified letter. If ≥20 letters are correctly identified at 4 meters, the ETDRS score equals the number of letters correctly read at 4 m plus 30. If fewer than 20 letters are correctly identified, testing of the first 6 lines will be performed at 1 meter, and the final score is the sum of correctly identified letters obtained at 4 m and 1 m.
Measured: From enrollment to the end of study at 12 months
Change-from-baseline in National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ-25) total score
NEI-VFQ-25 questionnaire will be used to assess trial participants. Uniformly trained investigators will administer each item following standardized instructions. Participants will respond based on their actual visual function and daily life experience, and investigators will record responses truthfully. Domain scores and the composite score will be calculated according to the NEI-VFQ-25 scoring rules. All scores are transformed to a scale ranging from 0 to 100, with higher scores indicating better visual function and visual-related quality of life.
Measured: From enrollment to the end of study at 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Feb 2026
- Study start14 Oct 2025
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AMD - Age-Related Macular DegenerationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The First Affiliated Hospital with Nanjing Medical University
- Sponsor type
- University, hospital or other
- Collaborators
- HELP Therapeutics Co., Ltd.
- Responsible party
- Sponsor
- Investigators
- Songtao Yuan, M.D., Principal investigator, The First Affiliated Hospital with Nanjing Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07418177
- Sponsor's protocol ID
- JSPH-iPSCs-RPE-001
- Status last verified
- Oct 2025
- First submitted
- 9 Jan 2026
- Last updated
- 19 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No