Wet Age-related Macular Degeneration
Conditions
Brief summary
This study will evaluate the therapeutic effects and safety of 800 mg ALK4290 administered daily over a 6-week dosing period in newly diagnosed patients with wet age-related macular degeneration (wAMD).
Detailed description
This study is designed to evaluate the therapeutic effects and safety of oral ALK4290 administered at 800 mg daily over a 6-week dosing period in newly diagnosed (treatment naïve) patients with wAMD. The study agent will be orally self-administered. All subjects will receive the study agent, ALK4290. This study does not contain a placebo.
Interventions
DRUGALK4290
ALK4290 400 mg tablet twice a day
Sponsors
Alkahest, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
50 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Men and women with newly diagnosed active CNV secondary to AMD, diagnosed by a retinal specialist with all the following characteristics and ophthalmic inclusion criteria applied to the study eye: * No prior treatment for wAMD in the study eye and no current or planned concomitant intravitreal anti-VEGF treatment in the fellow eye * Central subfield retinal thickness ≥ 250 microns on SD-OCT (exclusive of subretinal pigment epithelial fluid, inclusive of SRF) * Presence of SRF and/or IRF on SD-OCT * Any active CNV with subfoveal leakage as determined by FA * Total lesion size not greater than 12 disc areas on FA * If present, subretinal hemorrhage must comprise \< 50% of the total lesion area on FA * No subfoveal fibrosis or atrophy on FA * BCVA letter score, as measured by ETDRS in the study eye, between 70 and 24 letters, inclusive, at screening * Patients 50 years of age or older at screening visit 1 * Body mass index (BMI) between18 and ≤ 40 at screening visit 1 * Female subjects must not be pregnant or breastfeeding. Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry. * Signed informed consent consistent with ICH-GCP guidelines and local legislation prior to participation in the trial, which includes medication washout and restrictions
Exclusion criteria
* Previous participation in any studies of investigational drugs within 1 month preceding screening visit * Any form of macular degeneration that is not age-related (e.g., Best's disease, Stargardt's disease, Sorsby's disease, etc.) * Additional eye disease in the study eye that could compromise BCVA (i.e., uncontrolled glaucoma (intraocular pressure \> 24) with visual field loss, clinically significant diabetic maculopathy, history of ischemic optic neuropathy or retinal vascular occlusion, vitreomacular traction, monocular vision, or genetic disorders such as retinitis pigmentosa; high myopia \> 8 diopters) * The presence of polypoidal choroidal vasculopathy (PCV) or retinal angiomatous proliferation (RAP) in the study eye * Anterior segment and vitreous abnormalities in the study eye that would preclude adequate observation with fundus photography/FA or SD-OCT * Intraocular surgery in the study eye within 3 months prior to screening * Aphakia or total absence of the posterior capsule (yttrium aluminum garnet (YAG) laser capsulotomy permitted, a minimum of 1 month prior to enrollment) in the study eye
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Best Corrected Visual Acuity (BCVA) | Baseline to 6 weeks | Mean change in BCVA letter score as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) method. The ETDRS chart assesses changes in vision based on the number of letters correctly read by the subject. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Treatment-emergent Adverse Events (Safety) | Baseline to 10 weeks | Treatment-emergent adverse events identified by the Common Terminology Criteria for Adverse Events (CTCAE v4.03) |
Countries
Hungary
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Active ALK4290 800 mg daily | 30 |
| Total | 30 |
Baseline characteristics
| Characteristic | Active |
|---|---|
| Age, Continuous | 73.1 years STANDARD_DEVIATION 8.78 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 30 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 30 Participants |
| Region of Enrollment Hungary | 8 participants |
| Region of Enrollment Poland | 22 participants |
| Sex: Female, Male Female | 18 Participants |
| Sex: Female, Male Male | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 30 |
| other Total, other adverse events | 14 / 30 |
| serious Total, serious adverse events | 0 / 30 |
Outcome results
Primary
Best Corrected Visual Acuity (BCVA)
Mean change in BCVA letter score as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) method. The ETDRS chart assesses changes in vision based on the number of letters correctly read by the subject.
Time frame: Baseline to 6 weeks
Population: Evaluable Set
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Active | Best Corrected Visual Acuity (BCVA) | 7.0 letters | Standard Deviation 12.51 |
Secondary
Incidence of Treatment-emergent Adverse Events (Safety)
Treatment-emergent adverse events identified by the Common Terminology Criteria for Adverse Events (CTCAE v4.03)
Time frame: Baseline to 10 weeks
Population: Safety Set
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Active | Incidence of Treatment-emergent Adverse Events (Safety) | 14 Participants |