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Treatment of RP and LCA by Primary RPE Transplantation

Primary Purpose

Leber Congenital Amaurosis, Retinitis Pigmentosa

Status
Unknown status
Phase
Early Phase 1
Locations
China
Study Type
Interventional
Intervention
Human primary Retinal Pigment Epithelial (HuRPE) cells
Sponsored by
Eyecure Therapeutics Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Leber Congenital Amaurosis, Retinitis Pigmentosa

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept 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.

Sites / Locations

  • Beijing Tongren HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

low dose group

middle dose group

high dose group

Arm Description

300,000 HuRPE cells

500,000 HuRPE cells

1,000,000 HuRPE cells

Outcomes

Primary Outcome Measures

best-corrected visual acuity (BCVA)

Secondary Outcome Measures

Full Information

First Posted
June 12, 2018
Last Updated
August 7, 2018
Sponsor
Eyecure Therapeutics Inc.
Collaborators
Beijing Tongren Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT03566147
Brief Title
Treatment of RP and LCA by Primary RPE Transplantation
Official Title
Treatment of Retinitis Pigmentosa and Leber Congenital Amaurosis by Primary Retinal Pigment Epithelial Cells Transplantation
Study Type
Interventional

2. Study Status

Record Verification Date
June 2018
Overall Recruitment Status
Unknown status
Study Start Date
August 1, 2018 (Anticipated)
Primary Completion Date
December 31, 2019 (Anticipated)
Study Completion Date
March 31, 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Eyecure Therapeutics Inc.
Collaborators
Beijing Tongren Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Leber Congenital Amaurosis, Retinitis Pigmentosa

7. Study Design

Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
low dose group
Arm Type
Experimental
Arm Description
300,000 HuRPE cells
Arm Title
middle dose group
Arm Type
Experimental
Arm Description
500,000 HuRPE cells
Arm Title
high dose group
Arm Type
Experimental
Arm Description
1,000,000 HuRPE cells
Intervention Type
Biological
Intervention Name(s)
Human primary Retinal Pigment Epithelial (HuRPE) cells
Intervention Description
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.
Primary Outcome Measure Information:
Title
best-corrected visual acuity (BCVA)
Time Frame
12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Diagnosis of RP or LCA; Experimental eye with best-corrected visual acuity (BCVA) of no more than 35 letters or visual field of less than 10 degree; Patient understand and sign the consent form. Exclusion Criteria: Blood routine examination is abnormal (hemoglobin<10gm/dL; blood platelet count<100k/mm³; neutrophil count<1000/mm³) Blood biochemistry is abnormal (ALT／AST>1.5; creatinine>1.3mg/dL) Experimental eye has optic nerve atrophy caused by glaucoma Experimental eye has retinal detachment, or has received retinal detachment surgery. Patients with uveitis and other endophthalmitis Patients with other ocular disease affecting vision. Patients have participated in clinical study of ocular or systemic drug use in recent 6 months. Patients with medical history of malignant cancer (except resected basal cell carcinoma and squamous-cell carcinoma). Patients with medical history of myocardial infarction Patient with diabetes Patient with Parkinson disease or Alzheimer's disease Patients are under the treatment of immunosuppressive agent (except intermittent, low-dose corticosteroid treatment). Patients with other medical conditions that restricts the compliance and safety of patients, or affects experimental results.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Jingxue Zhang, Dr
Phone
86-18610772626
Email
jingxuezh@163.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ningli Wang
Organizational Affiliation
Beijing Tongren Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Beijing Tongren Hospital
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jingxue Zhang, Dr
Phone
18610772626
Email
jingxuezh@163.com

12. IPD Sharing Statement

Plan to Share IPD
No

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Treatment of RP and LCA by Primary RPE Transplantation

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