Non-Proliferative Diabetic RetinopathyPhase 3HELIOS-3NCT07235085
A Study to Evaluate the Efficacy and Safety of OTX-TKI (Axitinib Implant) in Participants With Non-Proliferative Diabetic Retinopathy
A Phase 3 study testing Intravitreal injection of axitinib hydrogel implant followed by a second intravitreal injection of the axitinib implant at Week 24 in people with Non-Proliferative Diabetic Retinopathy, run by Ocular Therapeutix, Inc. at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Non-Proliferative Diabetic Retinopathy
- What is being tested?
- Intravitreal injection of axitinib hydrogel implant followed by a second intravitreal injection of the axitinib implant at Week 24
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 930 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Nov 2025to Mar 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Cumberland Valley Retina Consultants
Hagerstown, Maryland, 21740
Allen Hu, MD, 7813574000, clinicalaffairs@ocutx.com
About this study
Official 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
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
- Being tested
- Single intravitreal injection of axitinib hydrogel implant followed by a mock (sham) injection procedure at Week 24, Intravitreal injection of axitinib hydrogel implant followed by a second intravitreal injection of the axitinib implant at Week 24, Sham/mock procedure of intravitreal injection followed by a second sham/mock procedure at Week 24
- Keywords
- Non-Proliferative Diabetic Retinopathy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Single intravitreal injection of axitinib hydrogel implant followed by a mock (sham) injection procedure at Week 24Drug
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 24Drug
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 24Other
sham/mock intravitreal injection procedure
| Study group | Role | What they receive |
|---|---|---|
| OTX-TKI Q52W (single dose) | Experimental | OTX-TKI 0.45 mg via intravitreal (IVT) administration at Day 1 and sham procedure at Week 24 Receives: Drug: Single intravitreal injection of axitinib hydrogel implant followed by a mock (sham) injection procedure at Week 24 |
| OTX-TKI Q24W (2 doses) | Experimental | OTX-TKI 0.45 mg via IVT administration at Day 1 and Week 24 Receives: Drug: Intravitreal injection of axitinib hydrogel implant followed by a second intravitreal injection of the axitinib implant at Week 24 |
| Sham Q24W (control) | Sham comparator | Sham procedure at Day 1 and Week 24 Receives: Other: Sham/mock procedure of intravitreal injection followed by a second sham/mock procedure at Week 24 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
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.
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2025
- Study start17 Nov 2025
- Primary completionMar 2027 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Non-Proliferative Diabetic RetinopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 930 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ocular Therapeutix, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Allen Hu, MD, Principal investigator, Cumberland Valley Retina Consultants
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07235085
- Sponsor's protocol ID
- OTX-TKI-2025-NPDR-302
- Acronym
- HELIOS-3
- Status last verified
- Mar 2026
- First submitted
- 14 Nov 2025
- Last updated
- 16 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT07235085
- Acronym / sponsor ID
- HELIOS-3 / OTX-TKI-2025-NPDR-302
- Conditions
- Non-Proliferative Diabetic Retinopathy
- Keywords
- Non-Proliferative Diabetic Retinopathy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 930 participants (estimated)
- Lead sponsor
- Ocular Therapeutix, Inc.Industry (pharma, biotech, devices)
- Study start
- 17 Nov 2025 (actual)
- Primary completion
- Mar 2027 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 19 Nov 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| OTX-TKI Q52W (single dose) | Experimental | Drug: Single intravitreal injection of axitinib hydrogel implant followed by a mock (sham) injection procedure at Week 24OTX-TKI 0.45 mg via intravitreal (IVT) administration at Day 1 and sham procedure at Week 24 |
| OTX-TKI Q24W (2 doses) | Experimental | Drug: Intravitreal injection of axitinib hydrogel implant followed by a second intravitreal injection of the axitinib implant at Week 24OTX-TKI 0.45 mg via IVT administration at Day 1 and Week 24 |
| Sham Q24W (control) | Sham comparator | Other: Sham/mock procedure of intravitreal injection followed by a second sham/mock procedure at Week 24Sham procedure at Day 1 and Week 24 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (5 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Single intravitreal injection of axitinib hydrogel implant followed by a mock (sham) injection procedure at Week 24OTX-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). | Drug | OTX-TKI Q52W (single dose) |
| Intravitreal injection of axitinib hydrogel implant followed by a second intravitreal injection of the axitinib implant at Week 24OTX-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). | Drug | OTX-TKI Q24W (2 doses) |
| Sham/mock procedure of intravitreal injection followed by a second sham/mock procedure at Week 24sham/mock intravitreal injection procedure | Other | Sham Q24W (control) |
Primary outcome
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Mar 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Allen Hu, MD principal investigator
Cumberland Valley Retina Consultants
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Intravitreal injection of axitinib hydrogel implant followed by a second intravitreal injection of the axitinib implant at Week 24 in people with Non-Proliferative Diabetic Retinopathy, run by Ocular Therapeutix, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Project Manager (7813574000, clinicalaffairs@ocutx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Mar 2026.