Evaluation of OLX10212 in Patients With Neovascular Age-related Macular Degeneration

NCT05643118 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: OLX10212-01
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 5

Brief Summary

This is a Phase 1, multicenter, open-label, single- and multi-dose, dose-escalating study of OLX10212 in patients with neovascular age-related macular degeneration (AMD). This study is composed of 2 parts: Part A and Part B. Part A is a single ascending dose study, and Part B is a multiple ascending dose study. The primary objective is to evaluate the safety and tolerability of single and multiple intravitreal injection(s) of OLX10212 in patients with neovascular AMD. The exploratory objectives are to evaluate the preliminary efficacy of single and multiple intravitreal injection(s) of OLX10212 in patients with neovascular AMD, and to evaluate the pharmacokinetics (PK) of single and multiple intravitreal injection(s) of OLX10212 in patients with neovascular AMD.

Conditions

Neovascular Age-related Macular Degeneration

Keywords

Neovascular age-related macular degeneration; OLX10212; siRNA; intravitreal injection; safety and tolerability; preliminary efficacy

Outcome Measures

Primary Outcomes (6)

• Best-corrected visual acuity (BCVA)

  Visual acuity using an ETDRS chart

  28 days after last dose administration

• Intraocular pressure (IOP)

  Millimeters of mercury (mmHg)

  28 days after last dose administration

• Slit lamp

  Anterior segment of the eye examination

  28 days after last dose administration

• Fundus examination

  Posterior segment of the eye examination

  28 days after last dose administration

• Spectral-domain optical coherence tomography (SD-OCT)

  Evaluation of retinal characteristics

  28 days after last dose administration

• Fluorescein angiography (FA)

  Evaluation of retinal vasculature

  28 days after last dose administration

Other Outcomes (5)

• Spectral-domain optical coherence tomography

  Week 24

• Fluorescein angiography

  Week 24

• Cmax

  Day 0, Day 1, Day 2, and Day 3 for Part A and Day 0, Day 1, Day 2, Day 3, Day 28, Day 29, Day 30, Day 31,Day 56, Day 57, Day 58, and Day 59 for Part B

Study Arms (7)

Part A 100 μg/eye/50 μL

EXPERIMENTAL

study eye treated with 100 μg (94.3 μg free acid) of OLX10212

Genetic: OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)

Part A 250 μg/eye/50 μL

EXPERIMENTAL

study eye treated with 250 μg (235.8 μg free acid) of OLX10212

Genetic: OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)

Part A 500 μg/eye/50 μL

EXPERIMENTAL

study eye treated with 500 μg (471.5 μg free acid) of OLX10212

Genetic: OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)

Part A 750 μg/eye/50 μL

EXPERIMENTAL

study eye treated with 750 μg (707.3 μg free acid) of OLX10212

Genetic: OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)

Part A 950 μg/eye/50 μL

EXPERIMENTAL

study eye treated with 950 μg (895.9 μg free acid) of OLX10212

Genetic: OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)

Part B 750 μg/eye/50 μL

EXPERIMENTAL

study eye treated with a total of 3 intravitreal injections of 750 μg (707.3 μg free acid) of OLX10212 each 28 days apart

Genetic: OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)

Part B 950 μg/eye/50 μL

EXPERIMENTAL

study eye treated with a total of 3 intravitreal injections of 950 μg (895.9 μg free acid) of OLX10212 each 28 days apart

Genetic: OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)

Interventions

OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA) GENETIC

Clear colorless solution dissolved in 1X PBS and injected intravitreally

Also known as: OLX10212

Part A 100 μg/eye/50 μL; Part A 250 μg/eye/50 μL; Part A 500 μg/eye/50 μL; Part A 750 μg/eye/50 μL; Part A 950 μg/eye/50 μL; Part B 750 μg/eye/50 μL; Part B 950 μg/eye/50 μL

Eligibility Criteria

• Age: 50 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Men and women ≥50 years of age
• Primary subfoveal CNV lesions secondary to AMD, including juxtafoveal lesions that affect the fovea, as evidenced by FA in the study eye
• CNV must be ≥50% of the total lesion size in the study eye
• ETDRS BCVA score ranging from 20/60 to 20/400 in the study eye
• Clear ocular media and adequate pupillary dilation (able to dilate pupil to ≥4 mm using standard mydriatics) in the study eye to permit good stereoscopic fundus photography
• Retinal thickness ≥200 μm in the macular region of the study eye as measured by SD-OCT, and active neovascular AMD, in the opinion of the Investigator
• Willing, committed, and able to return for all clinic visits and complete all study-related procedures
• Able to read (or if unable to read due to visual impairment, be read to verbatim by the person administering the informed consent or by a family member), understand, and willing to sign the informed consent form

You may not qualify if:

• Any prior systemic treatment for neovascular AMD in either eye, except dietary supplements or vitamins or systemic anti-VEGF therapy, or planned use at any time during the study
• Any prior treatment in the study eye with another investigational agent to treat neovascular AMD within 6 months prior to Day 0 or planned use at any time during the study
• Prior treatment with anti-VEGF agents as follows:
• Anti-VEGF therapy in the study eye within 4 weeks prior to Day 0
• Anti-VEGF therapy in the study eye at any time to which there was no response, as defined by the presence of at least 1 of the following conditions: (1) persistent (plasma) fluid exudation, (2) unresolved or new hemorrhage, and (3) progressive lesion fibrosis
• Anti-VEGF therapy in the fellow eye with an investigational agent (not FDA approved, unless it is bevacizumab) within 3 months prior to Day 0 (prior treatment with an FDA approved anti-VEGF therapy in the fellow eye is allowed at any time)
• Systemic anti-VEGF therapy, investigational or FDA approved, within 3 months prior to Day 0 or planned use at any time during the study
• Scar or fibrosis in the study eye involving \>50% of the total lesion size
• Retinal pigment epithelial tears or rips in the study eye involving the macula within 6 months prior to Day 0
• History of any vitreous hemorrhage in the study eye within 4 weeks prior to Day 0
• Presence of other causes of CNV in the study eye, including pathologic myopia, ocular histoplasmosis syndrome, angioid streaks, choroidal rupture, or multifocal choroiditis
• Clinical evidence of moderate or severe diabetic retinopathy, diabetic macular edema, or any other inflammatory or occlusive vascular disease affecting the retina (other than AMD) in either eye
• History of stage ≥2 macular hole in the study eye
• Any prior intraocular or periocular surgery on the study eye within 3 months prior to Day 0 (lid surgery is allowed if it took place at least 1 month prior to Day 0 and is unlikely to interfere with OLX10212 injection). Prior vitrectomy in the study eye, surgery for retinal detachment in the study eye, and prior trabeculectomy or other filtration surgery in the study eye are not permitted at any time
• Uncontrolled glaucoma (defined as IOP ≥25 mmHg despite treatment with antiglaucoma medication) in the study eye

Sponsors & Collaborators

• Olix Pharmaceuticals, Inc.: lead
• Trial Runners, LLC: collaborator
• Avance Clinical Pty Ltd.: collaborator

Study Sites (5)

California Retina Consultants

Santa Maria, California, 93434, United States

Location

University Retina

Oak Forest, Illinois, 60452, United States

Location

The Retina Institute

St Louis, Missouri, 63128, United States

Location

Ophthalmic Consultants of the Capital Region

Troy, New York, 12180, United States

Location

Texas Retina Consultants

Bellaire, Texas, 77401, United States

Location

Related Publications (1)

• Hwang J, Chang C, Kim JH, Oh CT, Lee HN, Lee C, Oh D, Lee C, Kim B, Hong SW, Lee DK. Development of Cell-Penetrating Asymmetric Interfering RNA Targeting Connective Tissue Growth Factor. J Invest Dermatol. 2016 Nov;136(11):2305-2313. doi: 10.1016/j.jid.2016.06.626. Epub 2016 Jul 15.

  PMID: 27427487 BACKGROUND

Study Officials

• Toni Bransford, MD

  Olix Pharmaceuticals, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 10, 2022
• First Posted: December 8, 2022
• Study Start: January 4, 2023
• Primary Completion: November 25, 2024
• Study Completion: November 10, 2025
• Last Updated: December 11, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)