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A Study to Assess the Efficacy and Safety of AKST4290 With Aflibercept in Patients With Newly Diagnosed nAMD

Phase 2
Completed
Conditions
Neovascular Age-related Macular Degeneration
Interventions
Drug: AKST4290
Drug: Placebo
Drug: Aflibercept
Registration Number
NCT04331730
Lead Sponsor
Alkahest, Inc.
Brief Summary

This study will evaluate the efficacy and safety of AKST4290 in combination with aflibercept injections in subjects with newly diagnosed neovascular age-related macular degeneration (nAMD).

Detailed Description

This is a randomized, double-masked, placebo-controlled, dose-ranging, multicenter study to assess the efficacy and safety of AKST4290 administered orally at 400 mg b.i.d. or 800 mg b.i.d. in combination with intravitreal aflibercept injections (IAI), in subjects with newly diagnosed neovascular age-related macular degeneration (nAMD) who are naïve to treatment with anti-vascular endothelial growth factor (anti-VEGF) medications in the study eye. Subjects will be treated with AKST4290 800 mg daily, 1600 mg daily, or placebo for a total of 36 weeks.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
107
Inclusion Criteria
  • Men and women with newly diagnosed active Choroidal Neovascularization (CNV) secondary to Age-Related Macular Degeneration (AMD), diagnosed by a retinal specialist with all the following characteristics and ophthalmic inclusion criteria applied to the study eye, as assessed by a central reader:

    • Has been examined by a retinal specialist and found to be eligible to receive Intravitreal Aflibercept Injection (IAI) in the study eye.
    • No prior treatment for Neovascular Age-Related Macular Degeneration (nAMD) in the study eye.
    • Study eye has not undergone pars plana vitrectomy or glaucoma filtering surgery.
    • Participation in studies of investigational drugs must have been discontinued within 30 days or 5 half-lives of the drug (whichever was longer) prior to screening.
    • Central subfield thickness (CST) thickness ≥ 250 microns on SD-OCT (spectral domain OCT) (exclusive of subretinal pigment epithelial fluid, inclusive of SRF).
    • Presence of SRF (subretinal fluid) and/or IRF (intraretinal fluid) on SD-OCT.
    • Total lesion size not greater than 12 disc areas (30.48 mm2) (1 disc area = 2.54 mm2) on FA (fluorescein angiography).
    • If present, subretinal hemorrhage must comprise < 50% of the total lesion area on FA, SD-OCT, or FP/FAF (fundus photography/fundus autofluorescence).
    • No subfoveal fibrosis or atrophy on FA, SD-OCT, or FP/FAF.
    • Active CNV (choroidal neovascularization) membranes with subfoveal leakage or juxtafoveal leakage too close for laser photocoagulation.
  • BCVA (Best Corrected Visual Acuity) in the study eye between 70 and 24 letters inclusive.

  • Body mass index (BMI) between (and inclusive of) 18 and 40 at screening.

Key

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Exclusion Criteria
  • Participation in studies of investigational drugs within 30 days or 5 half-lives of the drug (whichever was longer) prior to screening.
  • Known hypersensitivity to the active substance or any of the excipients of AKST4290 or aflibercept.
  • Active or suspected ocular or periocular infection and/or active, severe intraocular inflammation.
  • Any form of macular degeneration that is not age-related (e.g., Best's disease, Stargardt's disease, Sorsby's disease).
  • Additional disease in the study eye that could compromise BCVA (i.e., uncontrolled glaucoma (IOP >24) with visual field loss, clinically significant diabetic macular edema, history of ischemic optic neuropathy or retinal vascular occlusion, vitreomacular traction, high myopia > 6 diopters, or genetic disorders such as retinitis pigmentosa).
  • Presence of RPE (Retinal Pigment Epithelium) tears or rips in the study eye.
  • Anterior segment and vitreous abnormalities in the study eye that would preclude adequate visualization with FP/FAF, 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 in an eye with a posterior chamber intraocular lens if performed a minimum of 1 month prior to enrollment) in the study eye.
  • Known allergy to fluorescein sodium.
  • Significant alcohol or drug abuse within past 2 years.
  • Based on ECG (electrocardiogram) reading, subjects with a risk of QT prolongation.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
AKST4290 (800 mg) + AfliberceptAKST4290Subjects will receive 400 mg AKST4290 twice daily for 36 weeks, in combination with intravitreal aflibercept injection treatment
AKST4290 (1600 mg) + AfliberceptAKST4290Subjects will receive 800 mg AKST4290 twice daily for 36 weeks, in combination with intravitreal aflibercept injection treatment
Placebo + AfliberceptPlaceboSubjects will receive placebo for 36 weeks, in combination with intravitreal aflibercept injection treatment
Placebo + AfliberceptAfliberceptSubjects will receive placebo for 36 weeks, in combination with intravitreal aflibercept injection treatment
AKST4290 (800 mg) + AfliberceptAfliberceptSubjects will receive 400 mg AKST4290 twice daily for 36 weeks, in combination with intravitreal aflibercept injection treatment
AKST4290 (1600 mg) + AfliberceptAfliberceptSubjects will receive 800 mg AKST4290 twice daily for 36 weeks, in combination with intravitreal aflibercept injection treatment
Primary Outcome Measures
NameTimeMethod
Mean Change From Baseline in Best Corrected Visual Acuity (BCVA) Per the Early Treatment Diabetic Retinopathy Study (ETDRS) Testing MethodBaseline to Week 36

Mean change from baseline in Best Corrected Visual Acuity (BCVA) per the Early Treatment Diabetic Retinopathy Study (ETDRS) testing method. BCVA will be assessed using ETDRS charts at 4 meters initial testing distance and assessed in both eyes. Score range is 0 to 93. A higher score indicates better vision.

Secondary Outcome Measures
NameTimeMethod
Time to PRN Injection (Arms 1 and 2 Only)Baseline to Week 36

Time to first use of intravitreal aflibercept injection, as needed (AKST4290 Arms only). UNITS: weeks.

Median Number of Aflibercept Injections Received Beginning at Week 12Week 12 to Week 36

Median number of injections received beginning at Week 12 as a rate. UNITS: number of injections per week from Week 12

Percentage of Subjects With Best Corrected Visual Acuity (BCVA) Change of ≥ 15 LettersBaseline to Week 36

Percentage of subjects with Best Corrected Visual Acuity (BCVA) change of ≥ 15 letters at Week 36.

Mean Change in Central Subfield Thickness (CST) Compared With Control Through Week 12Baseline to Week 12

Mean change in Central Subfield Thickness (CST) compared with control through Week 12. UNITS: micrometre

Number of Participants With Adverse Events Assessed by IntensityScreening to Week 40

Number of Participants with Adverse Events categorized by intensity

Time to the First Visit Where PRN Injection Criteria Are MetWeek 12 to the first visit meeting PRN injection criteria through week 36

Time to the first visit where PRN injection criteria are met starting at Week 12 will be calculated in weeks as the first date where PRN injection criteria are first met minus the date of first dose of study drug plus one, divided by seven. Subjects who do not experience the event of interest (meet the criteria for PRN IAI) while on the study will be censored at their last visit completed through Week 36. Units: weeks

Mean Change in Best Corrected Visual Acuity (BCVA) Per the Early Treatment Diabetic Retinopathy Study (ETDRS) Testing Method as Compared With ControlWeek 12 to Week 36

Mean change in Best Corrected Visual Acuity (BCVA) letter score per the Early Treatment Diabetic Retinopathy Study (ETDRS) testing method from Week 12 as compared to control at Week 36. BCVA will be assessed using ETDRS charts at 4 meters initial testing distance and assessed in both eyes. Score range is 0 to 93. A higher score indicates better vision.

Trial Locations

Locations (21)

nordBLICK Augenklinik Bellevue

🇩🇪

Kiel, Germany

Sierra Eye Associates

🇺🇸

Reno, Nevada, United States

Internationale Innovative Ophthalmochirurgie GbR

🇩🇪

Düsseldorf, Germany

PROVISUS Sp. z o.o.

🇵🇱

Częstochowa, Poland

Centrum Medyczne Dietla 19 Sp zoo

🇵🇱

Kraków, Poland

Szpital św. Wojciecha

🇵🇱

Poznań, Poland

Central Clinical Hospital of the MSWiA

🇵🇱

Warsaw, Poland

Augentagesklinik Rheine

🇩🇪

Rheine, Germany

Tęczówka (IRIS)

🇵🇱

Bialystok, Poland

Specjalistyczny Ośrodek Okulistyczny Oculomedica (Specialized Eye Center Oculomedica)

🇵🇱

Bydgoszcz, Poland

GANGLION Orvosi Központ

🇭🇺

Pécs, Hungary

Klinika Chirurgii Siatkówki i Ciała Szklistego Medical University in Lublin

🇵🇱

Lublin, Poland

ArtOptica Salon Okulistyczno

🇵🇱

Suwałki, Poland

Borsod-Abaúj-Zemplén Megyei Kórház és Egyetemi Oktató Kórház (Borsod-Abaúj-Zemplén County Hospital and University Teaching Hospital)

🇭🇺

Miskolc, Hungary

Optimum Profesorskie Centrum Okulistyki

🇵🇱

Gdańsk, Poland

Centrum Medyczne UNO-MED

🇵🇱

Tarnów, Poland

Retina-Vitreous Associates Medical Group

🇺🇸

Beverly Hills, California, United States

Retina Vitreous Associates of FL

🇺🇸

Saint Petersburg, Florida, United States

Jahn Ferenc Dél-pesti Kórház (Jahn Ferenc South-Pest Hospital)

🇭🇺

Budapest, Hungary

Magyar Honvédség Egészségügyi Központ, Szemészeti Osztály (Medical Centre, Hungarian Defence Forces, Ophthalmology Department)

🇭🇺

Budapest, Hungary

Szegedi Tudományegyetem Általános Orvostudományi Kar, Szent-Györgyi Albert Klinikai Központ, Szemészeti Klinika, (University of Szeged Faculty of Medicine, Albert-Szent Gyorgyi Health Care, Department of Ophthalmology)

🇭🇺

Szekszárd, Hungary

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