Safety of BBC1501 Intravitreal Injection in Patients With Neovascular Age-Related Macular Degeneration (nAMD)

Study Overview
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Brief Summary
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This open-label study is being conducted to evaluate the initial safety and tolerability of BBC1501 IVT in patients with nAMD. The primary objective of this study is to evaluate the safety and tolerability of 3 ascending doses of IVT BBC1501 in patients with nAMD. The secondary objective of this study is to exploratory of BBC1501 efficacy following 3 ascending dose of BBC1501 in nAMD patient.

Official Title
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A Phase 1, Open Label, Ascending Dose Study to Evaluate the Safety of BBC1501 Administered by Intravitreal Injection for Neovascular Age-Related Macular Degeneration (nAMD)

Conditions
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Age-Related Macular Degeneration

Intervention / Treatment
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* Drug: BBC1501


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Able to provide voluntary written informed consent on the approved ICF, understand the study requirements, and are willing to follow and complete all the study required procedures. Male or female aged ≥ 50 years. Diagnosed with nAMD in the study eye as confirmed by fundus fluorescein angiography (FFA). Active CNV lesions, secondary to nAMD as confirmed with SD-OCT (or SS-OCT), FFA and fundus photography (FP) in the study eye. Best corrected visual acuity (BCVA) between 73 and 34 letters, inclusive, in the study eye using ETDRS testing. Presence of intra and/or subretinal fluid as identified by SD-OCT (or SS-OCT) attributable to active CNV in the study eye. Participants who have failed prior treatment in the study eye with any IVT anti VEGF medication with a washout period of four weeks prior to first administration of the IMP who in the opinion of the investigator, have disease sufficiently stable to enable this interval. Key Exclusion Criteria: Use of any of the following treatments or anticipated use of any of the following treatments to the study eye: Intravitreal or periocular corticosteroid, within 90 days prior to Visit 1 (Day 1) and throughout the study. Uncontrolled or advanced glaucoma, evidenced by an IOP of > 21 mmHg or cup/disc ratio > 0.8 while on medical therapy, or chronic hypotony (< 6 mmHg) in the study eye. Evidence of any other ocular disease other than nAMD in the study eye that may confound the outcome of the study (eg, active diabetic retinopathy, posterior uveitis, pseudovitelliform macular degeneration, moderate/severe myopia). Need for ocular surgery in the study eye during the course of the study. YAG laser capsulotomy within 30 days prior to Visit 1 (Day 1) in the study eye. Intraocular surgery, including lens removal or laser, within 90 days prior to Visit 1 (Day 1) in the study eye. Ocular or periocular infection in either eye. Pupillary dilation inadequate for quality stereoscopic fundus photography in the study eye. Media opacity that would limit clinical visualization, intravenous fluorescein angiography, or spectral-domain optical coherence tomography (SD-OCT) evaluation in the study eye. History of herpetic infection in the study eye or adnexa. Presence of known active toxoplasmosis, inactive toxoplasmosis or toxoplasmosis scar in either eye. Presence of any form of ocular malignancy including choroidal melanoma in either eye

Ages Eligible for Study
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Minimum Age: 50 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BBC1501 1.25ug<br>Cohort 1; open-label, non-randomized, single administration | Drug: BBC1501<br>* BBC1501 solution for Intravitreal injection<br>|
| Experimental: BBC1501 2.5ug<br>Cohort 2; open-label, non-randomized, single administration | Drug: BBC1501<br>* BBC1501 solution for Intravitreal injection<br>|
| Experimental: BBC1501 5ug<br>Cohort 3; open-label, non-randomized, single administration | Drug: BBC1501<br>* BBC1501 solution for Intravitreal injection<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Assessment of ophthalmic and systemic TEAEs, during study period | To evaluate the safety and tolerability of a single IVT dose of BBC1501 at 4 weeks after dose.To characterize ocular and non-ocular safety by the incidence of treatment-emergent adverse events (AEs) (new or worsening from baseline) summarized categorically by system organ class and/or preferred term. | Every week up to 4 weeks | 
| Assessment of ophthalmic and systemic TEAEs, during study period | To evaluate the safety and tolerability of a single IVT dose of BBC1501 at 12 weeks after dose.To characterize ocular and non-ocular safety by the incidence of treatment-emergent adverse events (AEs) (new or worsening from baseline) summarized categorically by system organ class and/or preferred term. | every 4 weeks up to 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean change in Early Treatment Diabetic Retinopathy Study (ETDRS) BCVA from baseline | Assessment ETDRS change from baseline by Optical Coherence Tomography(OCT) | Baseline, Week4 | 
| Mean change in Early Treatment Diabetic Retinopathy Study (ETDRS) BCVA from baseline | Assessment ETDRS change from baseline by Optical Coherence Tomography(OCT) | Baseline, Week12 | 
| Change in CNV size according to fluorescein angiogram | Assessment CNV size change from baseline by Fundus fluorescein angiography(FFA) | Baseline, Week4 | 
| Change in CNV size according to fluorescein angiogram | Assessment CNV size change from baseline by Fundus fluorescein angiography(FFA) | Baseline, Week12 | 
| Changes in intra-or sub-retinal fluid measured as mean change in central retinal thickness or macula volume | Assessment Central retinal thickness from baseline by Spectral Domain Optical Coherence Tomography (SD-OCT) | Baseline, Week4 | 
| Changes in intra-or sub-retinal fluid measured as mean change in central retinal thickness or macula volume | Assessment Central retinal thickness from baseline by Spectral Domain Optical Coherence Tomography (SD-OCT) | Baseline, Week12 | 
| Number of patients who initiation of rescue therapy during study | Exploratory using rescue therapy during study and follow-up period | Week1, Week12 |