A Clinical Trial to compare Efficacy, Safety, Tolerability of RBS-001 to Eylea® in Subjects with Neovascular Age-Related Macular Degeneration

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Rophibio Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11]

Trial duration

completed 18 Apr 2025

Decision date (initial)

2024-05-13

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

Rophibio Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Bioequivalence, Efficacy

To demonstrate the equivalence of RBS-001 and Eylea® for BCVA change from baseline measured by
ETDRS letter score at 8 weeks after treatment.

Secondary objectives 3

1. To evaluate other efficacy of RBS-001 in comparison with Eylea® at each visit after treatment versus baseline
2. To evaluate the safety and tolerability of RBS-001 in comparison with Eylea®
3. To evaluate the immunogenicity of RBS-001 in comparison with Eylea®

Conditions and MedDRA coding

Neovascular age-related macular degeneration (nAMD)

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Age ≥ 50 years at screening
2. Individuals with active CNV lesion secondary to AMD in the study eye, proven by fluorescein angiography (FA) and confirmed by the central reading center during the screening period
3. Individuals with CNV area in the study eye accounting for ≥ 50% of the total lesion, including macular hemorrhage, scar, atrophy, fibrosis and neovascularization, proven by FA and confirmed by the central reading center during the screening period
4. Individuals with intraretinal or subretinal fluid in the study eye due to active CNV, proven by optical coherence tomography (OCT) and confirmed by the central reading center during the screening period
5. Individuals with BCVA of 34 to 73 letters measured by ETDRS letter score at the screening and baseline visits in the study eye
6. Individuals who voluntarily decide to participate in the clinical study after being fully informed of the details of the clinical study and who provide written consent to comply with the study instructions during the clinical study

Exclusion criteria 11

1. 1.Individuals whose study eye lesion meets any of the following criteria (to be confirmed by the central reading center during the screening period) Item Criterion Total lesion* size > 23 mm2 [9 disc areas (DAs)] (Must be proven by FA) Subretinal hemorrhage (or subfoveal hemorrhage) ≥ 50% of the total lesion* [≥ 1 DA (In the case of subfoveal hemorrhage, fovea must be surrounded 270 degrees by visible CNV.)] Scar or fibrosis ≥ 50% of the total lesion*or Scar, fibrosis, or atrophy involving the center of the fovea Retinal pigment epithelial tears or rips Macular involvement Macular hole At any stage, if present in the study eye Other causes of CNV Ocular histoplasmosis syndrome, trauma, multifocal choroiditis, angioid streaks, history of choroidal rupture, pathologic myopia (spherical equivalent of negative 8 diopters or more or axial length of 25 mm or more), etc. *Including macular hemorrhage, scar, atrophy, fibrosis, and neovascularization
2. 10.Individuals considered by the investigator to be ineligible for study participation for any reasons other than the inclusion and exclusion criteria
3. 11.Employees of investigational sites, individuals directly involved with the conduct of the study, prisoners, and persons who are legally institutionalized
4. 7.Individuals who have only one functional eye (monocular vision)
5. 2.Individuals with any of the following concurrent diseases at screening or for a specified period of time: (1) Concurrent ocular disease Eye Criterion Study eye Significant ocular media opacity such as a cataract which, in the judgment of the investigator, interferes with retinal visualization or retinal imaging tests Aphakia or the absence of the posterior capsule [except for eyes with intraocular lens implant having undergone yttrium aluminum garnet (YAG) laser posterior capsulotomy] Vitreous hemorrhage within the past 4 weeks Uncontrolled ocular hypertension (defined as IOP ≥ 25 mmHg despite adequate treatment) Fellow eye Any diagnosis or signs of nAMD requiring treatment with an IVT anti-VEGF agent during the screening period or at study treatment initiation Either eye Scleromalacia Active or suspected periocular/ocular infection or ocular inflammation (e.g., keratitis, scleritis, etc.) within 4 weeks prior to the first dose of the IP (2) Systolic blood pressure ≥ 160 mmHg or diastolic blood pressure ≥ 100 mmHg (despite adequate treatment) (3) Uncontrolled diabetes mellitus, at the investigator’s discretion (4) Congestive heart failure with New York Heart Association (NYHA) functional classification 3 or 4 or any clinically significant heart disease including ventricular arrhythmia and atrial fibrillation, at the investigator’s discretion (5) Active systemic infection undergoing treatment or recurrent clinically significant infections within 4 weeks prior to the first dose of the IP
6. 3.Individuals with any medical history of the following at screening: (1) Other ophthalmic disease in the study eye that may affect safety/efficacy assessments in the subject or may require medical/surgical interventions during the clinical study at the investigator’s discretion (e.g., vitreomacular traction, glaucoma undergoing treatment, retinal detachment, corneal dystrophy, etc.) (2) Diabetic retinopathy (DR)*, diabetic macular edema (DME), retinal vein occlusion (RVO), uveitis, or other vascular disease affecting the retina (other than nAMD) in either eye *Mild non-proliferative DR will be permitted. (3) Stroke, transient ischemic attack, pulmonary embolism, deep venous thrombosis, or myocardial infarction within the past 24 weeks (4) Hypersensitivity reactions to aflibercept or other drugs to be used in the clinical study (fluorescein, mydriatic drops, etc.) (5) Malignancy within the last 5 years (however, individuals with basal cell, cutaneous squamous cell carcinoma, in situ cervical cancer, or in situ breast ductal carcinoma who are in stable long-term follow-up without therapeutic medication, procedures, or surgery can participate in this clinical trial) (6) Organ or bone marrow transplantation
7. 4.Individuals with a history of any of the following medication or non-pharmacological treatment for the study eye: (1) Glaucoma filtering surgery, vitrectomy or corneal transplantation (2) Simple intraocular or periocular surgery (e.g., cataract surgery, simple neodymium yttrium aluminum garnet (Nd:YAG) laser capsulotomy on a pseudophakic eye due to posterior capsular opacification, etc.) within 12 weeks or eyelid surgery within 4 weeks prior to the screening visit [Laser iridotomy will be permitted.] (3) Macular photodynamic therapy (PDT) with verteporfin, transpupillary thermotherapy, radiotherapy, or retinal laser treatment (e.g., focal laser photocoagulation, pan-retinal photocoagulation, etc.) (4) Periocular radiotherapy (5) Any anti-vascular endothelial growth factor (anti-VEGF) treatment for nAMD (including participation in other clinical studies (6) Treatment for retinal detachment (medication or non-pharmacological treatment) (7) IVT corticosteroid injection, sub-tenon or periocular corticosteroid injection within 24 weeks or IVT corticosteroid implantation within 36 months prior to the screening visiti (8)Topical ocular corticosteroids administered for ≥ 30 consecutive days or for ≥ 60 non-consecutive days within 90 days prior to randomization
8. 5.Individuals with any of the following medication or non-pharmacological treatment history: (1) Systemic anti-VEGF therapy within 12 weeks prior to the first dose of the IP (3) Any anti-VEGF treatment of nAMD in the fellow eye within 8 weeks prior to the first dose of the IP (including anti-VEGF) (Dietary supplements and vitamins will be permitted.) (4) Current use at screening of medications known to be toxic to the lens, retina, or optic nerve, including deferoxamine, chloroquine/hydroxychloroquine, tamoxifen, phenothiazines, vigabatrin, ethambutol. (5) Systemic corticosteroids administered within 12 weeks prior to the first dose of the IP (prednisolone ≤ 10 mg/day and equivalent dose administered for 14 days or less or inhaled/intranasal/dermal agents will be permitted.) (6) Other IP within 12 weeks or 5 times the half-life (whichever is longer) prior to the first dose of the IP
9. 6.Individuals with BCVA of fewer than 34 letters measured by ETDRS letter score at the screening and baseline visits in the fellow eye
10. 8.Pregnant [human chorionic gonadotropin (hCG) positive] or breastfeeding women of childbearing potential at the screening and baseline visits
11. 9.Men or women of childbearing potential who are unwilling to use adequate methods of contraception* from the time of written informed consent to 12 weeks after the last dose of the IP * Hormonal contraceptives (oral contraceptive pill, contraceptive patch, etc.), intrauterine device or intrauterine system implantation, sterilization procedure or surgery (vasectomy, bilateral tubal ligation, etc.), complete abstinence

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline (Day 1) in BCVA measured by ETDRS letter score at 8 weeks after the IP treatment

Secondary endpoints 8

1. Change from baseline (Day 1) in BCVA measured by ETDRS letter score at all visits after the IP treatment will be analyzed using MMRM that includes the data from Weeks 4, 8, 12, 16, 20 and 24 after IP treatment
2. Change from baseline (Day 1) in BCVA measured by ETDRS letter score at each time point (within 52 weeks) after the IP treatment
3. Proportion of subjects whose BCVA measured by ETDRS letter score decreases by ≥ 5, ≥ 10 and ≥ 15 letters from baseline (Day 1) at all visits after the IP treatment
4. Proportion of subjects whose BCVA measured by ETDRS letter score increases by ≥5, ≥10 and ≥15 letters from baseline (Day 1) at all visits after the IP treatment
5. Change from baseline (Day 1) in CST measured by OCT at each time point (within 52 weeks) after the IP treatment
6. Proportion of subjects with intraretinal or subretinal fluid measured by OCT at each measurement time point after the IP treatment compared to baseline (Day 1)
7. Change from screening in CNV area assessed by FA at 24 and 52 weeks after the IP treatment
8. Proportion of subjects having leakage assessed by FA at 24 and 52 weeks after the IP treatment compared to screening

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Comparator 1

Auxiliary 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Rophibio Inc.

Sponsor organisation

Rophibio Inc.

Address

Room 406 194-25 Osongsaengmyeong 1-Ro

City

Cheongju-Si

Postcode

28160

Country

Korea, Republic of

Scientific contact point

Organisation

Rophibio Inc.

Contact name

Clinical Trial Info Desk

Public contact point

Organisation

Rophibio Inc.

Contact name

Clinical Trial Info Desk

Third parties 8

Locations

4 EU/EEA countries · 31 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 34 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications