Diabetic Macular Edema (DME)
Conditions
Keywords
AsclepiX, macular edema, DME, eye diseases, retinal diseases, AXT107, diabetes, duration of action, dose escalation, diabetic retinopathy, retinal degeneration
Brief summary
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).
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
DRUGAXT107 0.25 mg
Single intravitreal injection of AXT107 0.25 mg/eye
DRUGAXT107 0.5 mg
Single intravitreal injection of AXT107 0.5 mg/eye
Sponsors
AsclepiX Therapeutics, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety as Assessed by Incidence of Adverse Events (AEs) | Screening to Week 48 | Incidence of ocular (study eye) and systemic AEs |
Countries
United States
Participant flow
Pre-assignment details
No participants were assigned to the High Dose Arm/Group due to adverse events observed in the Low Dose and Mid Dose groups.
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 | 3 |
| Mid Dose AXT107 0.25 mg/eye
AXT107 0.25 mg: Single intravitreal injection of AXT107 0.25 mg/eye | 3 |
| Total | 6 |
Baseline characteristics
| Characteristic | Low Dose | Mid Dose | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 3 Participants | 3 Participants |
| Age, Categorical Between 18 and 65 years | 3 Participants | 0 Participants | 3 Participants |
| Age, Continuous | 58.3 years | 73.3 years | 65.8 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 3 Participants | 2 Participants | 5 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 1 Participants | 1 Participants |
| Region of Enrollment United States | 3 participants | 3 participants | 6 participants |
| Sex: Female, Male Female | 1 Participants | 0 Participants | 1 Participants |
| Sex: Female, Male Male | 2 Participants | 3 Participants | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 3 | 0 / 3 |
| other Total, other adverse events | 2 / 3 | 1 / 3 |
| serious Total, serious adverse events | 3 / 3 | 1 / 3 |
Outcome results
Primary
Safety as Assessed by Incidence of Adverse Events (AEs)
Incidence of ocular (study eye) and systemic AEs
Time frame: Screening to Week 48
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Low Dose | Safety as Assessed by Incidence of Adverse Events (AEs) | Serious Non-Ocular Adverse Event Not Related to treatment | 1 Participants |
| Low Dose | Safety as Assessed by Incidence of Adverse Events (AEs) | Serious Ocular Adverse Event Treatment Related | 2 Participants |
| Mid Dose | Safety as Assessed by Incidence of Adverse Events (AEs) | Serious Ocular Adverse Event Treatment Related | 1 Participants |
| Mid Dose | Safety as Assessed by Incidence of Adverse Events (AEs) | Serious Non-Ocular Adverse Event Not Related to treatment | 0 Participants |