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NCT Number: NCT07235085

A Study to Evaluate the Efficacy and Safety of OTX-TKI (Axitinib Implant) in Participants With Non-Proliferative Diabetic Retinopathy

The purpose of this trial is to study the safety and effectiveness of OTX-TKI (axitinib intravitreal hydrogel) for the treatment of Non-Proliferative Diabetic Retinopathy.

OTX-TKI is an intravitreal hydrogel embedded with axitinib. When the OTX-TKI hydrogel is administered into the vitreous cavity of the eye, the hydrogel begins to slowly break down, which allows the axitinib to be slowly released over time.

This clinical trial is comparing OTX-TKI to a "sham" injection procedure. The sham injection is a mock injection procedure, but nothing will actually be inserted in the eye as there is no needle on the sham injector. Only one eye ("study eye") will be treated with study treatment.

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Key information

Conditions

Non-Proliferative Diabetic Retinopathy

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Cumberland Valley Retina Consultants

Hagerstown, Maryland, 21740, United States

Location status: Recruiting

Location contact

Allen Hu, MD

CONTACT

[email protected]

7813574000

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Male or female who is at least 18 years of age at the time of signing the informed consent form (ICF)
  • History of or newly diagnosed with type 1 or 2 diabetes mellitus and have moderately severe to severe NPDR (DRSS levels 47 or 53), confirmed by the Central Reading Center (CRC) based on the images obtained at the Screening visit
  • BCVA Early Treatment Diabetic Retinopathy Study (ETDRS) letter score of ≥ 69 letters (approximate Snellen equivalent of 20/40 or better) in the study eye
  • Willing and able to comply with clinic visits and study-related procedures
  • Provide signed informed consent

Exclusion criteria

  • Presence of center-involved diabetic macular edema (CI-DME) defined per protocol via optical coherence tomography (SD-OCT) in the study eye, obtained at the Screening visit
  • Evidence of a rhegmatogenous retinal detachment or visually significant/severe epiretinal membrane, vitreomacular traction syndrome, macular hole, tear of the retinal pigment epithelium in the macula, or other macular pathology in the study eye deemed visually significant by the Investigator
  • In the study eye, any panretinal photocoagulation (PRP) treatment prior to baseline, or received focal or grid laser photocoagulation within 1000 microns of the central subfield of the macula within 6 months prior to baseline
  • IVT anti-VEGF treatment in the study eye within 6 months or port delivery system (PDS) with ranibizumab in the study eye at any time prior to baseline (Day 1)
  • (There are additional exclusion criteria)

Treatment and study plan

Single intravitreal injection of axitinib hydrogel implant followed by a mock (sham) injection procedure at Week 24

Drug

OTX-TKI 0.45 mg is a dried polyethylene glycol (PEG)-based hydrogel containing dispersed drug particles of the small molecule axitinib, a tyrosine kinase inhibitor (TKI).

Intravitreal injection of axitinib hydrogel implant followed by a second intravitreal injection of the axitinib implant at Week 24

Drug

OTX-TKI 0.45 mg is a dried polyethylene glycol (PEG)-based hydrogel containing dispersed drug particles of the small molecule axitinib, a tyrosine kinase inhibitor (TKI).

Sham/mock procedure of intravitreal injection followed by a second sham/mock procedure at Week 24

Other

sham/mock intravitreal injection procedure

Primary outcomes

  1. DRSS 2-step change status at Week 52 from baseline in the study eye (≥ 2 step improvement, ≥ 2-step worsening, < 2-step change in either direction)

    Time frame: 52 weeks

    The Diabetic Retinopathy Severity Scale (DRSS) measures the severity of the diabetic retinopathy in patients by grading retinal images obtained by color fundus photography. Severity is determined by the presence of specific signs.

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Project Manager

CONTACT

[email protected]

7813574000

Sponsors and collaborators

Lead sponsor

Ocular Therapeutix, Inc.

Industry

Registry information

Official study title

A Phase 3, Multicenter, Double-Masked, Randomized, 3-Arm Parallel Group Study to Evaluate the Efficacy and Safety of Intravitreal OTXTKI (Axitinib Implant) in Participants With Non-Proliferative Diabetic Retinopathy

Acronym: HELIOS-3

Important dates

Study start
2025
Primary completion
2027
Study completion
2027
First posted
Nov 19, 2025
Registry last updated
Mar 16, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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