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

NCT ID: NCT07235085

Last Updated: 2025-11-19

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 930 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-11-17
• Study Completion Date: 2027-03-31

Brief Summary

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.

Conditions

Non-Proliferative Diabetic Retinopathy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

OTX-TKI Q52W (single dose)

OTX-TKI 0.45 mg via intravitreal (IVT) administration at Day 1 and sham procedure at Week 24

Group Type: EXPERIMENTAL

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

Intervention Type: 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).

OTX-TKI Q24W (2 doses)

OTX-TKI 0.45 mg via IVT administration at Day 1 and Week 24

Group Type: EXPERIMENTAL

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

Intervention Type: 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 Q24W (control)

Sham procedure at Day 1 and Week 24

Group Type: SHAM_COMPARATOR

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

Intervention Type: OTHER

sham/mock intravitreal injection procedure

Interventions

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

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).

Intervention Type: DRUG

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

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).

Intervention Type: DRUG

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

sham/mock intravitreal injection procedure

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

1. Male or female who is at least 18 years of age at the time of signing the informed consent form (ICF)

2. 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

3. BCVA Early Treatment Diabetic Retinopathy Study (ETDRS) letter score of ≥ 69 letters (approximate Snellen equivalent of 20/40 or better) in the study eye

4. Willing and able to comply with clinic visits and study-related procedures

5. Provide signed informed consent

Exclusion Criteria

1. 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

2. 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

3. 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

4. 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)

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Ocular Therapeutix, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Allen Hu, MD

Role: PRINCIPAL_INVESTIGATOR

Cumberland Valley Retina Consultants

Locations

Cumberland Valley Retina Consultants

Hagerstown, Maryland, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Clinical Project Manager

Role: CONTACT

clinicalaffairs@ocutx.com

7813574000

Facility Contacts

Allen Hu, MD

Role: primary

clinicalaffairs@ocutx.com

781-357-4000

Other Identifiers

OTX-TKI-2025-NPDR-302

Identifier Type: -

Identifier Source: org_study_id