Safety and Bioactivity of AXT107 in Subjects With Diabetic Macular Edema (CONGO)

Phase 1/2a Study of the Safety and Bioactivity of AXT-107 in Subjects With Diabetic Macular Edema (DME)

This study 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 Diabetic Macular Edema (DME).

Study Overview

• Status: Terminated
• Conditions: Diabetic Macular Edema (DME)
• Intervention / Treatment: Drug: AXT107 0.1 mg; Drug: AXT107 0.25 mg; Drug: AXT107 0.5 mg

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 Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Gilbert, Arizona, United States, 85053
      • AsclepiX Investigative Site
  • California
    • Los Angeles, California, United States, 90211
      • AsclepiX Investigative Site
  • Florida
    • Saint Petersburg, Florida, United States, 33711
      • AsclepiX Investigative Site
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • AsclepiX Investigative Site
  • Nevada
    • Reno, Nevada, United States, 89502
      • AsclepiX Investigative Site
  • Oregon
    • Eugene, Oregon, United States, 97401
      • AsclepiX Investigative Site
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19107
      • AsclepiX Investigative Site
  • Texas
    • Abilene, Texas, United States, 79606
      • AsclepiX Investigative Site
    • McAllen, Texas, United States, 78550
      • AsclepiX Investigative Site
    • The Woodlands, Texas, United States, 77384
      • AsclepiX Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Patients 18 years of age or older with diabetic macular edema (DME) diagnosis secondary to diabetes mellitus Type 1 or 2
• Best corrected visual acuity (BCVA) Early Treatment Diabetic Retinopathy Study (ETDRS) letter score of 65 to 23 in the study eye
• Willing and able to comply with clinic visits and study-related procedures
• Provide signed inform consent

Exclusion Criteria:

• Any signs of high risk proliferative diabetic retinopathy in the study
• Previously-treated patients who are not responders to anti-VEGF
• Panretinal laser photocoagulation within 6 months and macular laser photocoagulation with 3 months of screening in the study eye

Note: Other inclusion/exclusion criteria apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Low Dose; AXT107 0.1 mg/eye	Drug: AXT107 0.1 mg; Single intravitreal injection of AXT107 0.1 mg/eye; Other Names: Gersizangatide
Experimental: Mid Dose; AXT107 0.25 mg/eye	Drug: AXT107 0.25 mg; Single intravitreal injection of AXT107 0.25 mg/eye; Other Names: Gersizangatide
Experimental: High Dose; AXT107 0.5 mg/eye	Drug: AXT107 0.5 mg; Single intravitreal injection of AXT107 0.5 mg/eye; Other Names: Gersizangatide

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety as Assessed by Incidence of Adverse Events (AEs)	Incidence of ocular (study eye) and systemic AEs	Screening to Week 48

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy as Assessed by Central Retinal Thickness (CST)	Mean change in CST assessed by spectral domain optical coherence tomography	Day 0 to Week 48
Efficacy as Assessed by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart	Mean change in BCVA	Day 0 to Week 48
Efficacy as Assessed by Early Treatment Diabetic Retinopathy Study (ETDRS) Chart	Percentage of subjects improving ≥5, ≥10, and ≥15 letters in BCVA	Day 0 to Week 48

Collaborators and Investigators

• Sponsor: AsclepiX Therapeutics, Inc.
• Investigators: Study Director: Amir Shojaei, PhD, Asclepix Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): December 10, 2020
• Primary Completion (Actual): October 1, 2022
• Study Completion (Actual): October 1, 2022

Study Registration Dates

• First Submitted: December 17, 2020
• First Submitted That Met QC Criteria: January 4, 2021
• First Posted (Actual): January 6, 2021

Study Record Updates

• Last Update Posted (Actual): March 22, 2024
• Last Update Submitted That Met QC Criteria: March 20, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: diabetes; retinal degeneration; macular edema; diabetic retinopathy; dose escalation; DME; AsclepiX; eye diseases; retinal diseases; AXT107; duration of action
• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Macular Degeneration; Macular Edema; Edema
• Other Study ID Numbers: AXT107-CS101

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No