Neovascular Age-Related Macular Degeneration (nAMD)
Conditions
Keywords
AsclepiX, Neovascular Age-Related Macular Degeneration, nAMD, eye diseases, retinal diseases, AXT107, duration of action, dose escalation, macular degeneration
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.
Interventions
DRUGAXT107 0.1 mg
Single intravitreal injection of AXT107 0.1 mg/eye (low dose)
DRUGAXT107 0.25 mg
Single intravitreal injection of AXT107 0.25 mg/eye (mid dose)
DRUGAXT107 0.5 mg
Single intravitreal injection of AXT107 0.5 mg/eye (high dose)
Sponsors
AsclepiX Therapeutics, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
50 Years to No maximum
Healthy volunteers
No
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/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Assessed by Incidence of Adverse Events (AEs) | Screening to Week 48 | Incidence of ocular (study eye) and systemic adverse events (AEs) |
Countries
United States
Participant flow
Pre-assignment details
Participants in the mid Dose and high Dose arms were not enrolled due to adverse events observed in the Low Dose arm participants
Participants by arm
| Arm | Count |
|---|---|
| Low Dose AXT107 0.1 mg/eye
AXT107 0.1 mg: Single intravitreal injection of AXT107 0.1 mg/eye (low dose) | 3 |
| Total | 3 |
Baseline characteristics
| Characteristic | Low Dose |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 3 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Age, Continuous | 79 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 3 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Region of Enrollment United States | 3 participants |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 3 |
| other Total, other adverse events | 1 / 3 |
| serious Total, serious adverse events | 0 / 3 |
Outcome results
Primary
Number of Participants Assessed by Incidence of Adverse Events (AEs)
Incidence of ocular (study eye) and systemic adverse events (AEs)
Time frame: Screening to Week 48
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Low Dose | Number of Participants Assessed by Incidence of Adverse Events (AEs) | 1 Participants |