ASP3021 eye injections for vision loss from age-related macular degeneration in Japan

A Phase 3/4, Multicenter, Single-arm Clinical Study to Assess the Safety of Intravitreal Administration of ASP3021 in Participants in Japan With Geographic Atrophy Secondary to Age-related Macular Degeneration

We will try monthly ASP3021 eye injections for 12 months in people in Japan aged 40 and older with geographic atrophy from AMD to see if the treatment is safe and well tolerated.

Quick facts

• Phase: Phase 4
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 40 Years and up
• Sex: All
• Sponsor: Astellas Pharma Inc (Industry-sponsored)
• Drugs / interventions: radiation
• Locations: 16 sites (Nagoya, Aichi-ken and 15 other locations)
• Trial ID: NCT06970665 on ClinicalTrials.gov

What this trial studies

This post-marketing, open-label Phase 4 study gives monthly intravitreal injections of ASP3021 (avacincaptad pegol/IZERVAY) for 12 months to people with geographic atrophy (GA) secondary to age-related macular degeneration. Participants must meet imaging and visual acuity criteria and will undergo regular clinic visits for safety checks, eye exams, and retinal imaging. The primary focus is safety and tolerability, with investigators collecting adverse event data, intraocular pressure measurements, and imaging to document GA. The study is being conducted at multiple university hospital sites in Japan.

Who should consider this trial

Good fit: Ideal candidates are adults in Japan aged 40 or older with GA secondary to AMD who have GA area between 2.0 and 17.5 mm², non-foveal central involvement, and best-corrected visual acuity between 20/25 and 20/320 and can undergo required imaging.

Not a fit: People with GA outside the specified lesion size or location, inadequate imaging, uncontrolled eye pressure, advanced central foveal destruction, or who are pregnant are unlikely to qualify or benefit from this protocol.

Why it matters

Potential benefit: If successful, this could confirm that monthly ASP3021 injections are a safe, tolerable option that may help preserve vision in people with GA from AMD.

How similar studies have performed: IZERVAY (avacincaptad pegol) has received marketing approval in Japan and prior clinical work with this agent and similar complement-targeting approaches has shown effects on GA progression, so this is a follow-up post-marketing safety effort.

Eligibility criteria

Inclusion Criteria:

* Participant has non-foveal central point involvement geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
* Participant has total GA area ≥ 2.0 and ≤ 17.5 mm\^2 (1 and 7 disc areas \[DA\] respectively), determined by fundus autofluorescence (FAF) screening images.
* If participant has multifocal GA, at least one focal lesion should measure ≥ 1.25 mm\^2 (0.5 DA).
* Participant has GA in part within 1500 microns from the fovea center point.
* Participant has GA that must be able to be photographed in its entirety.
* Participant has best corrected visual acuity (BCVA) between 20/25 and 20/320, inclusive.
* Participant has clear ocular media and adequate pupillary dilatation in both eyes to allow for all imaging procedures, including good quality stereoscopic fundus photography and FAF.
* Participant has intraocular pressure (IOP) of ≤ 21 mmHg or less.
* Female participant is not pregnant and at least 1 of the following conditions apply:

  * Not a women of childbearing potential (WOCBP).
  * WOCBP who has a negative serum pregnancy test at screening with a medical interview and agrees to follow the contraceptive guidance from the time of informed consent through at least 40 days after final study intervention administration.
* Female participant must not be breastfeeding or lactating starting at screening and throughout the investigational period and for 40 days after final study intervention administration.
* Female participant must not donate ova starting at first administration of study intervention and throughout the investigational period and for 40 days after final study intervention administration.
* Male participant must agree to use contraception with female partner(s) of childbearing potential (including breastfeeding partner) throughout the treatment period and for 40 days after final study intervention administration.
* Male participant must agree to remain abstinent or use a condom with pregnant partner(s) for the duration of the pregnancy throughout the investigational period and for 40 days after final study intervention administration.
* Male participant must not donate sperm during the treatment period and for 40 days after final study intervention administration.
* Participant has ability to return for all study visits for the 12-month duration of the study.
* Participant agrees not to participate in another interventional study while participating in the present study.

Exclusion Criteria:

* Participant has evidence of choroidal neovascularization (CNV) in either eye.
* Participant has GA secondary to any condition other than AMD in the study eye (e.g., drug-induced).
* Participant has any ocular condition in the study eye that would progress during the course of the study and that could affect central vision or otherwise be a confounding factor.
* Participant has the presence of intraocular inflammation (≥ trace cell or flare), macular hole, pathologic myopia, epiretinal membrane, evidence of significant vitreomacular traction, vitreous hemorrhage or aphakia (pseudophakia with or without an intact capsule is not an exclusion criteria) in the study eye.
* Participant has the presence or history of idiopathic or autoimmune-associated uveitis in either eye.
* Participant has significant media opacities, including cataract, which might interfere with visual acuity (VA), assessment of toxicity or retinal imaging (fundus photography, FAF, spectral domain optical coherence tomography \[SD-OCT\], enhanced depth imaging optical coherence tomography \[EDI OCT\], fluorescein angiography \[FA\] or indocyanine green angiography \[ICGA\]) in the course of the study in either eye. Participant should not be entered if there is likelihood that they will require cataract surgery in either eye during the study.
* Participant has the presence of other causes of CNV, including pathologic myopia, high myopia (spherical equivalent of -8 diopters or more, or axial length of 25 mm or more), ocular histoplasmosis syndrome, angioid streaks, choroidal rupture, and multifocal choroiditis in either eye.
* Participant has any ocular or periocular infection (including blepharitis), or ocular surface inflammation in past 12 weeks in the study eye.
* Participant has any sign of diabetic retinopathy in either eye.
* Participant has BCVA of 20/320 or worse in the fellow eye.
* Participant has any prior treatment for AMD (dry or wet) or any prior intravitreal (IVT) treatment for any indication in either eye, except oral supplements of vitamins and minerals.
* Participant has concomitant treatment with any ocular or systemic medication that is known to be toxic to the lens, retina, or optic nerve in either eye (including, but not limited to amiodarone, deferoxamine, chloroquine/hydroxychloroquine sulfate, tamoxifen, phenothiazines, ethambutol or fingolimod).
* Participant has had any intraocular surgery or thermal laser treatment in the study eye within 3 months prior to screening, or any prior thermal laser treatment in the macular region, regardless of indication in the study eye.
* Participant has a history of any of the following procedures in the study eye: posterior vitrectomy, filtering surgery (e.g. trabeculectomy), glaucoma drainage device, corneal transplant, or retinal detachment.
* Participant has any previous therapeutic radiation in the region of the study eye.
* Participant has a present or previous history of participation in a study of ASP3021.
* Participant has a history of systemic treatment with any anti-complement agent in past or the likelihood of treatment with any systemic anti-complement agent during the study.
* Participant has received any investigational therapy within 60 days or 5 half-lives, whichever is longer, prior to screening.
* Participant has a history or evidence of severe cardiac disease (e.g., New York Heart Association \[NYHA\] Functional Class III or IV, or history or clinical evidence of unstable angina, acute coronary syndrome, myocardial infarction or revascularization within last 6 months, or ventricular tachyarrhythmias requiring ongoing treatment.
* Participant has a history or evidence of clinically significant peripheral vascular disease, such as intermittent claudication or prior amputation.
* Participant has clinically significant impaired renal (serum creatinine \> 2.5 mg/dL or status post-renal transplant or receiving dialysis) or hepatic function. Participants with these conditions may be enrolled after consultation with the medical monitor.
* Participant has a history of stroke within 12 months prior to screening.
* Participant has had any major surgical procedure within 1 month prior to screening.
* Participant has any condition that makes the participant unsuitable for study participation.
* Participant has a known or suspected serious allergy to the fluorescein dye and/or the indocyanine green dye used in angiography, povidone-iodine or hypersensitivity to ASP3021 or any components of the formulation used.

Where this trial is running

Nagoya, Aichi-ken and 15 other locations

• Nagoya City University Hospital — Nagoya, Aichi-ken, Japan (Recruiting)
• Fukushima Medical University Hospital — Fukushima, Fukushima, Japan (Recruiting)
• Kagawa University Hospital — Kita-gun, Kagawa-ken, Japan (Recruiting)
• Yokohama City University Medical Center — Yokohama, Kanagawa, Japan (Recruiting)
• Kyoto University Hospital — Kyoto, Kyoto, Japan (Recruiting)
• Mie University Hospital — Tsu, Mie-ken, Japan (Recruiting)
• Nagasaki University Hospital — Nagasaki, Nagasaki, Japan (Recruiting)
• University of the Ryukyus hospital — Ginowan-shi, Okinawa, Japan (Recruiting)
• Kansai Medical University Hospital — Hirakata-shi, Osaka, Japan (Recruiting)
• The University of Osaka Hospital — Suita-shi, Osaka, Japan (Recruiting)
• Seirei Hamamatsu General Hospital — Hamamatsu, Shizuoka, Japan (Recruiting)
• Jichi Medical University Hospital — Shimotsuke-shi, Tochigi, Japan (Recruiting)
• Nihon University Hospital — Chiyoda-ku, Tokyo, Japan (Recruiting)
• Tokyo Women's Medical University Hospital — Shinjuku-ku, Tokyo, Japan (Recruiting)
• Toyama University Hospital — Toyama, Toyama, Japan (Recruiting)
• University Of Yamanashi Hospital — Chuo-shi, Yamanashi, Japan (Recruiting)

Study contacts

• Study coordinator: Astellas Pharma Inc.

Conditions Geographic Atrophy; Age-Related Macular Degeneration; avacincaptad pegol; IZERVAY; ASP3021