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VEGFA-targeting Gene Therapy to Treat Retinal and Choroidal Neovascularization Diseases

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05099094
Recruitment Status : Recruiting
First Posted : October 29, 2021
Last Update Posted : August 23, 2022
Sponsor:
Collaborator:
Eye & ENT Hospital of Fudan University
Information provided by (Responsible Party):
Shanghai BDgene Co., Ltd.

Study Description
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Brief Summary:
Patients who respond to anti-VEGF therapy but with refractory retinal and choroidal neovascularization diseases including neovascular age-related macular degeneration (nAMD), diabetic macular edema (DME), and retinal vein occlusion-Macular edema (RVO-ME).

Condition or disease Intervention/treatment Phase
Neovascular Age-related Macular Degeneration Diabetic Macular Edema Retinal Vein Occlusion Genetic: BD311 Early Phase 1

Detailed Description:
Choroidal and retinal angiogenesis diseases are a group of diseases characterized by choroidal or retinal angiogenesis. These diseases are often correlated with the macular area, which may lead to significant visual loss. In this study, The IDLV vector is engineered to carry the VEGFA antibody gene. The gene is delivered to the RPE cells to express the VEGFA antibody which neutralizes the VEGFA activity in the posterior segment of the eye of individuals who have progressed to various forms of neovascular macular degeneration.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Safety and Efficacy Study of VEGFA-targeting Gene Therapy to Treat Refractory Retinal and Choroidal Neovascularization Diseases
Actual Study Start Date : November 25, 2021
Estimated Primary Completion Date : September 2023
Estimated Study Completion Date : September 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: BD311 Adults single group
Administered by suprachoroidal injection. Dosage form: injection solution. Dose: 500uL. Frequency of administration: one time injection.
 Genetic: BD311
Integration-deficient lentiviral vector (IDLV) expressing VEGFA antibody


Outcome Measures
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Primary Outcome Measures :
  1. Treatment-related adverse events [ Time Frame: At multiple timepoints after infusion up to 12 months. ]
    Observe and record incidences of AE and SAE related to VEGFA-targeting gene therapy drug BD311 (IDLV expressing VEGFA antibody) administration.


Secondary Outcome Measures :
  1. Changes in macular intraretinal fluid (IRF) [ Time Frame: At multiple timepoints after infusion up to 12 months. ]
    The presence of macular intraretinal fluid (IRF) will be determined by optical coherence tomography (OCT).

  2. Changes in subretinal fluid (SRF) [ Time Frame: At multiple timepoints after infusion up to 12 months. ]
    The presence of subretinal fluid (SRF) will be determined by optical coherence tomography (OCT).

  3. Change in central retinal thickness (CRT) [ Time Frame: At multiple timepoints after infusion up to 12 months. ]
    Central retinal thickness will be measured by Optical Coherence Tomography (OCT).

  4. Changes in the area of choroidal neovascularization [ Time Frame: At multiple timepoints after infusion up to 12 months. ]
    Using Fluorescein angiography (FFA) and indocyanine green angiography (ICGA) to assess the areas of choroidal neovascularization. Only for patients with nAMD.

  5. Changes in the area of fluorescein leakage [ Time Frame: At multiple timepoints after infusion up to 12 months. ]
    Using Fluorescein angiography (FFA) and indocyanine green angiography (ICGA) to assess the areas of fluorescein leakage. Only for patients with nAMD.

  6. The number of rescue treatments [ Time Frame: At multiple timepoints after infusion up to 12 months. ]
    Rescue treatments that require vitreous anti-VEGF injections due to illness.

  7. Evaluate the visual improvement [ Time Frame: At multiple timepoints after infusion up to 12 months. ]
    Subjects will be examined for best corrected visual acuity (BCVA).


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients with nAMD at the age ≥50; Or patients with diabetic macular edema (DME) at the age ≥18; Or patients with macular edema following retinal vein occlusion (RVO-ME) at the age ≥18.
  2. Early Treatment Diabetic Retinopathy Study (ETDRS) best corrected visual acuity ≤63 and letter score ≥19 Corresponding Snellen vision ≤20/63 and ≥20/400).
  3. OCT confirms the presence of intraretinal fluid or subretinal fluid in the fovea.
  4. Have received anti-VEGF therapy in the past and have responded to anti-VEGF therapy.
  5. With refractory conditions: repeated anti-VEGF treatments are required due to the disease condition. When the treatment is interrupted, the disease condition recurs (OCT examination indicates increased subretinal/inner effusion in the macula)
  6. For patients with both eyes suffered, enroll the one with more severe condition.
  7. Routine blood test, liver and kidney function, coagulation index of patients is normal：AST/ALT < 2.5 × ULN; TB < 1.5 × ULN; PT < 1.5 × ULN; Hb > 10 g/dL (male) and > 9 g/dL (female); PLT > 100 × 10^3/µL; eGFR > 30 mL/min/1.73 m^2.

Subjects voluntarily join the study, sign an informed consent form, have good compliance, and cooperate with follow-up.

Exclusion Criteria:

  1. Choroidal neovascularization or macular edema induced by other diseases.
  2. Any other factors that affect vision improvement in the study eye, such as fibrosis, atrophy, or RPE tear in the fovea of the macula.
  3. The study eye already has severe proliferative retinopathy, such as retinal neovascularization, traction retinal detachment, etc. (only for DME and RVO-ME patients) .
  4. Retinal detachment or advanced glaucoma in the study eye.
  5. Implants in the study eye (except intraocular lenses).
  6. Received internal eye surgery within 3 months prior to enrollment.
  7. Vitrectomy surgery on the study eye.
  8. Received intravitreal glucocorticoid or other clinical research drugs (except anti-VEGF therapy) within 6 months prior to enrollment.
  9. Myocardial infarction, cerebrovascular accident or transient ischemic attack occurred within 6 months prior to enrollment.
  10. Poorly controlled hypertension under maximum medication (systolic blood pressure>180 mmHg, diastolic blood pressure>100 mmHg).
  11. Poor blood glucose control under medication (fasting blood glucose is greater than or equal to 10.0 umol/L).
  12. Women who are willing to give birth; pregnant/breastfeeding women Have received gene therapy in the past.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05099094


Contacts
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Contact: Yujia Cai, PhD 17721291876 yujia.cai@bdgene.cn
Contact: Ting Xu 17721291876 ting.xu@bdgene.cn

Locations
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China, Shanghai
Eye & ENT Hospital of Fudan University Recruiting
Shanghai, Shanghai, China, 200032
Contact: Gezhi Xu    86-21-54237900    medcenter@fudan.edu.cn   
Contact: Jiaxu Hong    86-21-54237900    medcenter@fudan.edu.cn   
Sponsors and Collaborators
Shanghai BDgene Co., Ltd.
Eye & ENT Hospital of Fudan University
Investigators
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Study Director: Gezhi Xu, Dr Eye & ENT Hospital of Fudan University
More Information
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Responsible Party: Shanghai BDgene Co., Ltd.
ClinicalTrials.gov Identifier: NCT05099094    
Other Study ID Numbers: BD311
First Posted: October 29, 2021    Key Record Dates
Last Update Posted: August 23, 2022
Last Verified: August 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Shanghai BDgene Co., Ltd.:
Retinal diseases
Choroidal diseases
Neovascularization
nAMD
neovascular age-related macular degeneration
 DME
Diabetic macular edema
RVO-ME
Retinal vein occlusion-Macular edema
Additional relevant MeSH terms:
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Macular Degeneration
Macular Edema
Wet Macular Degeneration
Retinal Vein Occlusion
Choroidal Neovascularization
Neovascularization, Pathologic
Retinal Degeneration
Retinal Diseases
Eye Diseases
 Metaplasia
Pathologic Processes
Venous Thrombosis
Thrombosis
Embolism and Thrombosis
Vascular Diseases
Cardiovascular Diseases
Choroid Diseases
Uveal Diseases