Treatment of RP and LCA by Primary RPE Transplantation

Treatment of Retinitis Pigmentosa and Leber Congenital Amaurosis by Primary Retinal Pigment Epithelial Cells Transplantation

Status

UNKNOWN

Phases

Early Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03566147

Enrollment

Registered

2018-06-21

Start date

2018-08-01

Completion date

2020-03-31

Last updated

2018-08-09

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Leber Congenital Amaurosis, Retinitis Pigmentosa

Brief summary

Early Phase I Study of the Safety and Preliminary Efficacy of Human primary Retinal Pigment Epithelial (HuRPE) Cells Subretinal Transplantation in Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) Patients

Interventions

BIOLOGICALHuman primary Retinal Pigment Epithelial (HuRPE) cells

HuRPE will be transplanted by a board-certified vitreoretinal surgeon, which will be administered into the subretinal space of experimental eye through a standard surgical approach.

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 70 Years

Healthy volunteers

Inclusion criteria

1. Diagnosis of RP or LCA; 2. Experimental eye with best-corrected visual acuity (BCVA) of no more than 35 letters or visual field of less than 10 degree; 3. Patient understand and sign the consent form.

Exclusion criteria

1. Blood routine examination is abnormal (hemoglobin\<10gm/dL; blood platelet count\<100k/mm³; neutrophil count\<1000/mm³) 2. Blood biochemistry is abnormal (ALT／AST\>1.5; creatinine\>1.3mg/dL) 3. Experimental eye has optic nerve atrophy caused by glaucoma 4. Experimental eye has retinal detachment, or has received retinal detachment surgery. 5. Patients with uveitis and other endophthalmitis 6. Patients with other ocular disease affecting vision. 7. Patients have participated in clinical study of ocular or systemic drug use in recent 6 months. 8. Patients with medical history of malignant cancer (except resected basal cell carcinoma and squamous-cell carcinoma). 9. Patients with medical history of myocardial infarction 10. Patient with diabetes 11. Patient with Parkinson disease or Alzheimer's disease 12. Patients are under the treatment of immunosuppressive agent (except intermittent, low-dose corticosteroid treatment). 13. Patients with other medical conditions that restricts the compliance and safety of patients, or affects experimental results.

Design outcomes

Primary

Measure	Time frame
best-corrected visual acuity (BCVA)	12 months

Countries

China

Contacts

Primary ContactJingxue Zhang, Dr

jingxuezh@163.com86-18610772626

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026