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

Phase 1

Terminated

• Conditions: Neovascular Age-Related Macular Degeneration (nAMD)
• Interventions: Drug: AXT107 0.1 mg; Drug: AXT107 0.5 mg; Drug: AXT107 0.25 mg

• Registration Number: NCT04746963
• Lead Sponsor: AsclepiX Therapeutics, Inc.

Brief Summary

This is an open-label, dose-escalating, 48-week study assessing the safety, tolerability, bioactivity and duration of action of a single intravitreal injection of 0.1 mg, 0.25 mg, or 0.5 mg AXT107 in approximately 18 subjects (up to 6 subjects per dose) with nAMD.

Detailed Description

Upon providing informed consent, subjects will be sequentially enrolled into the study. Decision regarding dose escalation will be based on the recommendation from the Data Monitoring Committee (DMC). The first 3 eligible subjects will receive the low dose of AXT107 injection. After the 3 low dose subjects complete a 7-day follow-up, the DMC will review their safety data. If an acceptable safety profile is determined by the DMC, 3 additional subjects will be enrolled to receive the mid dose of AXT107 injection. Upon completion of a 7-day follow-up, review of the safety data, and determination of an acceptable safety profile by the DMC for the mid dose subjects, 3 additional subjects will be enrolled to receive the high dose of AXT107 injection.

Study Timeline

• Study Start: 2021-02-01
• Study First Submitted: 2021-02-05
• Study First Submitted QC: 2021-02-09
• Study First Posted: 2021-02-10
• Primary Completion: 2022-10-01
• Study Completion: 2022-10-01
• Results First Submitted: 2024-01-29
• Status Verified: 2024-03
• Results First Submitted QC: 2024-03-20
• Last Update Submitted: 2024-03-20
• Results First Posted: 2024-03-22
• Last Update Posted: 2024-03-22

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 3

Inclusion Criteria

• Patients 50 years of age or older diagnosed in the study eye with subfoveal choroidal neovascularization (CNV) or juxtafoveal CNV secondary to AMD
• Best corrected visual acuity (BCVA) Early Treatment Diabetic Retinopathy Study (ETDRS) letter score of 65 to 10 in the study eye
• Willing and able to comply with clinic visits and study-related procedures
• Provide signed inform consent

Exclusion Criteria

• Macular edema or CMV secondary to any causes other than AMD in the study eye
• Previously-treated patients who are not responders to anti-VEGF
• Any condition that may preclude improvement in visual acuity in the study eye
• Previous vitreoretinal surgery, filtration surgery, and cataract surgery within 3 months in the study eye

Note: Other inclusion/exclusion criteria apply

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Low Dose	AXT107 0.1 mg	AXT107 0.1 mg/eye
High Dose	AXT107 0.5 mg	AXT107 0.5 mg/eye
Mid Dose	AXT107 0.25 mg	AXT107 0.25 mg/eye

Primary Outcome Measures

Name	Time	Method
Number of Participants Assessed by Incidence of Adverse Events (AEs)	Screening to Week 48	Incidence of ocular (study eye) and systemic adverse events (AEs)

Trial Locations

Locations (1)

AsclepiX Investigative Site; 🇺🇸 The Woodlands, Texas, United States