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Safety and Bioactivity of AXT107 in Subjects With Neovascular Age-Related Macular Degeneration (nAMD) (DISCOVER)

Primary Purpose

Neovascular Age-related Macular Degeneration

Status
Not yet recruiting
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
AXT107 Low Dose
AXT107 Mid Dose
AXT107 High Dose
Sponsored by
AsclepiX Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Neovascular Age-related Macular Degeneration

Eligibility Criteria

50 Years - 99 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: 50 years of age or older Presence of active subfoveal choroidal neovascularization (CNV) (any subtype) or juxtafoveal CNV with leakage affecting the fovea secondary to AMD and the area of the CNV lesion must beat least 50% of the total lesion size confirmed by the Investigator Evidence of subretinal or intraretinal fluid or retinal cystic changes evidenced by Spectral Domain Optical Coherence Tomography (SD-OCT) at Screening accompanied by finding on SD- OCT suggestive of CNV secondary to AMD confirmed by the Investigator BCVA in the study eye between 65 and 25 ETDRS letters (20/50 and 20/320 Snellen equivalent) at Baseline (Day 0) BCVA of 34 ETDRS letters or better (20/200 or better Snellen equivalent) in the non-study eye at Baseline (Day 0) Able and willing to give signed informed consent and follow study instructions Has been/is a prior partial responder to an anti-VEGF agent Exclusion Criteria: Previously treated patients who are non-responders to anti-VEGF as determined by the Investigator Any prior use of Brolucizumab; use of Aflibercept within 8 weeks or use of Ranibizumab or Bevacizumab within 6 weeks from Baseline (Day 0) in the study eye Any concurrent disease that would require medical or surgical intervention during the study in the study eye (e.g., retinal detachment, significant cataract) Any condition that may preclude improvement in visual acuity or affect the evaluation of the study eye after resolution of AMD (e.g., extensive macular hemorrhage ≥ 50% lesion size, media clarity insufficient to obtain quality images, presence of diabetic retinopathy) Other causes of CNV (e.g., pathologic myopia, ocular histoplasmosis syndrome, angioid streaks, and multifocal choroiditis) in the study eye confirmed by the Investigator Chronic uveitis or ongoing clinically significant infection or inflammation (e.g., keratitis, scleritis) in either eye

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm 3

    Arm Type

    Experimental

    Experimental

    Experimental

    Arm Label

    Low Dose

    Mid Dose

    High Dose

    Arm Description

    AXT107 0.125 mg/eye

    AXT107 0.250 mg/eye

    AXT107 0.500 mg/eye

    Outcomes

    Primary Outcome Measures

    Incidence of Treatment-Emergent Adverse Events
    Primary Outcome Measure: 1.Safety as Assessed by change from baseline in Incidence of Adverse Events (AEs) Incidence of ocular (study eye) and systemic AEs [Time Frame: Screening to Week 40]

    Secondary Outcome Measures

    Mean Change in Central Subfield Thickness (CST)
    Secondary Outcome Measures: Efficacy as Assessed by Central Subfield Thickness (CST, measured in µm) Mean change in CST assessed by spectral domain optical coherence tomography [Time Frame: Day 0 to Week 40]
    Mean Change in Visual Function
    Secondary Outcome Measures: Visual Function as Assessed by Mean change in Best Corrected Visual Acuity (BCVA) (number of letters in Early Treatment Diabetic Retinopathy Study (EDTRS) chart)
    Visual Function Subgroup Analysis
    Secondary Outcome Measures: Visual Function as Assessed by Early Treatment Diabetic Retinopathy Study (EDTRS) Chart Percentage of subjects improving ≥5, ≥10, and ≥15 letters in Best Corrected Visual Acuity (BCVA)

    Full Information

    First Posted
    March 24, 2023
    Last Updated
    May 5, 2023
    Sponsor
    AsclepiX Therapeutics, Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT05859776
    Brief Title
    Safety and Bioactivity of AXT107 in Subjects With Neovascular Age-Related Macular Degeneration (nAMD)
    Acronym
    DISCOVER
    Official Title
    Phase 1/2a Study of the Safety and Bioactivity of AXT107 Injected Suprachoroidally in Subjects With Neovascular Age-Related Macular Degeneration (nAMD)
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    May 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    December 2023 (Anticipated)
    Primary Completion Date
    February 2025 (Anticipated)
    Study Completion Date
    April 2025 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    AsclepiX Therapeutics, Inc.

    4. Oversight

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

    5. Study Description

    Brief Summary
    The goal of this clinical trial is to understand the safety of AXT107 injected suprachoroidally in participants with nAMD. The main question[s] it aims to answer are: Safety of the maximum tolerable dose of AXT107 Bioactivity and duration of action of AXT107 injected suprachoroidally Participants will be injected with AXT107 and will be followed on a regular monitoring visits through 9 months post single injection.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Neovascular Age-related Macular Degeneration

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 1, Phase 2
    Interventional Study Model
    Sequential Assignment
    Masking
    None (Open Label)
    Allocation
    Non-Randomized
    Enrollment
    15 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    Low Dose
    Arm Type
    Experimental
    Arm Description
    AXT107 0.125 mg/eye
    Arm Title
    Mid Dose
    Arm Type
    Experimental
    Arm Description
    AXT107 0.250 mg/eye
    Arm Title
    High Dose
    Arm Type
    Experimental
    Arm Description
    AXT107 0.500 mg/eye
    Intervention Type
    Drug
    Intervention Name(s)
    AXT107 Low Dose
    Other Intervention Name(s)
    Gersizangatide
    Intervention Description
    Single suprachoroidal injection of AXT107 (0.125 mg/eye)
    Intervention Type
    Drug
    Intervention Name(s)
    AXT107 Mid Dose
    Other Intervention Name(s)
    Gersizangatide
    Intervention Description
    Single suprachoroidal injection of AXT107 (0.250 mg/eye)
    Intervention Type
    Drug
    Intervention Name(s)
    AXT107 High Dose
    Other Intervention Name(s)
    Gersizangatide
    Intervention Description
    Single suprachoroidal injection of AXT107 (0.500 mg/eye)
    Primary Outcome Measure Information:
    Title
    Incidence of Treatment-Emergent Adverse Events
    Description
    Primary Outcome Measure: 1.Safety as Assessed by change from baseline in Incidence of Adverse Events (AEs) Incidence of ocular (study eye) and systemic AEs [Time Frame: Screening to Week 40]
    Time Frame
    40 Weeks
    Secondary Outcome Measure Information:
    Title
    Mean Change in Central Subfield Thickness (CST)
    Description
    Secondary Outcome Measures: Efficacy as Assessed by Central Subfield Thickness (CST, measured in µm) Mean change in CST assessed by spectral domain optical coherence tomography [Time Frame: Day 0 to Week 40]
    Time Frame
    40 Weeks
    Title
    Mean Change in Visual Function
    Description
    Secondary Outcome Measures: Visual Function as Assessed by Mean change in Best Corrected Visual Acuity (BCVA) (number of letters in Early Treatment Diabetic Retinopathy Study (EDTRS) chart)
    Time Frame
    Day 0 to Week 40
    Title
    Visual Function Subgroup Analysis
    Description
    Secondary Outcome Measures: Visual Function as Assessed by Early Treatment Diabetic Retinopathy Study (EDTRS) Chart Percentage of subjects improving ≥5, ≥10, and ≥15 letters in Best Corrected Visual Acuity (BCVA)
    Time Frame
    Day 0 to Week 40

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    50 Years
    Maximum Age & Unit of Time
    99 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: 50 years of age or older Presence of active subfoveal choroidal neovascularization (CNV) (any subtype) or juxtafoveal CNV with leakage affecting the fovea secondary to AMD and the area of the CNV lesion must beat least 50% of the total lesion size confirmed by the Investigator Evidence of subretinal or intraretinal fluid or retinal cystic changes evidenced by Spectral Domain Optical Coherence Tomography (SD-OCT) at Screening accompanied by finding on SD- OCT suggestive of CNV secondary to AMD confirmed by the Investigator BCVA in the study eye between 65 and 25 ETDRS letters (20/50 and 20/320 Snellen equivalent) at Baseline (Day 0) BCVA of 34 ETDRS letters or better (20/200 or better Snellen equivalent) in the non-study eye at Baseline (Day 0) Able and willing to give signed informed consent and follow study instructions Has been/is a prior partial responder to an anti-VEGF agent Exclusion Criteria: Previously treated patients who are non-responders to anti-VEGF as determined by the Investigator Any prior use of Brolucizumab; use of Aflibercept within 8 weeks or use of Ranibizumab or Bevacizumab within 6 weeks from Baseline (Day 0) in the study eye Any concurrent disease that would require medical or surgical intervention during the study in the study eye (e.g., retinal detachment, significant cataract) Any condition that may preclude improvement in visual acuity or affect the evaluation of the study eye after resolution of AMD (e.g., extensive macular hemorrhage ≥ 50% lesion size, media clarity insufficient to obtain quality images, presence of diabetic retinopathy) Other causes of CNV (e.g., pathologic myopia, ocular histoplasmosis syndrome, angioid streaks, and multifocal choroiditis) in the study eye confirmed by the Investigator Chronic uveitis or ongoing clinically significant infection or inflammation (e.g., keratitis, scleritis) in either eye

    12. IPD Sharing Statement

    Plan to Share IPD
    Undecided

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    Safety and Bioactivity of AXT107 in Subjects With Neovascular Age-Related Macular Degeneration (nAMD)

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