Treatment of RP and LCA by Primary RPE Transplantation

Early Phase 1

• Conditions: Leber Congenital Amaurosis, Retinitis Pigmentosa
• Interventions: Biological: Human primary Retinal Pigment Epithelial (HuRPE) cells

• Registration Number: NCT03566147
• Lead Sponsor: Eyecure Therapeutics Inc.

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

Detailed Description

Not available

Study Timeline

• Status Verified: 2018-06
• Study First Submitted: 2018-06-12
• Study First Submitted QC: 2018-06-12
• Study First Posted: 2018-06-21
• Study Start: 2018-08-01
• Last Update Submitted: 2018-08-07
• Last Update Posted: 2018-08-09
• Primary Completion: 2019-12-31
• Study Completion: 2020-03-31

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 30

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
low dose group	Human primary Retinal Pigment Epithelial (HuRPE) cells	300,000 HuRPE cells
middle dose group	Human primary Retinal Pigment Epithelial (HuRPE) cells	500,000 HuRPE cells
high dose group	Human primary Retinal Pigment Epithelial (HuRPE) cells	1,000,000 HuRPE cells

Primary Outcome Measures

Name	Time	Method
best-corrected visual acuity (BCVA)	12 months

Trial Locations

Locations (1)

Beijing Tongren Hospital; 🇨🇳 Beijing, China