Clinical trial NCT03558061: Evaluate the Effects and Safety of ALK4290 in Patients With Newly Diagnosed Wet Age-Related Macular Degeneration
Official title: A Single Arm Open-Label Study to Evaluate the Therapeutic Effects and Safety of a 6-Week Treatment Regimen of ALK4290 in Patients With Newly Diagnosed Wet Age-Related Macular Degeneration (wAMD)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Wet Age-related Macular Degeneration
Lead sponsor: Alkahest, Inc.
Sponsor class: INDUSTRY
First posted: 2018-06-15
Enrollment: 30
Sex: ALL
Minimum age (years): 50
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Best Corrected Visual Acuity (BCVA)
timeFrame: Baseline to 6 weeks
Secondary outcomes:
measure: Incidence of Treatment-emergent Adverse Events (Safety)
timeFrame: Baseline to 10 weeks
Other outcomes:
Locations:
city: Budapest
country: Hungary
facility: Jahn Ferenc South-Pest Hospital and Clinic
city: Miskolc
country: Hungary
facility: Borsod-Abauj-Zemplen County Hospital and Teaching Hospital
city: Nyíregyháza
country: Hungary
facility: Szabolcs-Szatmar-Bereg County Hospital and University Hospital
city: Szeged
country: Hungary
facility: University of Szeged Faculty of Medicine
city: Szombathely
country: Hungary
facility: Markusovszky University Teaching Hospital
Study officials:
affiliation: Alkahest, Inc.
name: Alkahest Medical Monitor
role: STUDY_DIRECTOR
References: