Treatment of RP and LCA by Primary RPE Transplantation

Eyecure Therapeutics

Status and phase: Unknown; Early Phase 1

Conditions: Leber Congenital Amaurosis, Retinitis Pigmentosa

Treatments: Biological: Human primary Retinal Pigment Epithelial (HuRPE) cells

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT03566147

TR-RPE-RP/LCA

Details and patient eligibility

About

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

Enrollment: 30 estimated patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No 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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 3 patient groups

low dose group

Experimental group

Description:

300,000 HuRPE cells

Treatment:

Biological: Human primary Retinal Pigment Epithelial (HuRPE) cells

middle dose group

Experimental group

Description:

500,000 HuRPE cells

Treatment:

Biological: Human primary Retinal Pigment Epithelial (HuRPE) cells

high dose group

Experimental group

Description:

1,000,000 HuRPE cells

Treatment:

Biological: Human primary Retinal Pigment Epithelial (HuRPE) cells