Diabetic Macular EdemaPhase 2ELAAVATENCT06942520
Phase 2 Trial of RGX-314 in Adults With Center Involved - Diabetic Macular Edema (CI - DME)
A Phase 2 study testing RGX-314 Dose 1, RGX-314 Dose 2 and Aflibercept (2.0 mg) in people with Diabetic Macular Edema, run by Sierra Eye Associates at 1 site in United States.
Trial summary
Phase 2 open label, randomized, active controlled, dose-ranging trial in adults with Center Involved - Diabetic Macular Edema (CI - DME) Read more about this study
- Who is it for?
- Ages 25 to 89 years
- Conditions the study is for
- Diabetic Macular Edema
- What is being tested?
- RGX-314 Dose 1, RGX-314 Dose 2 and Aflibercept (2.0 mg)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Mar 2025to Dec 2027
- Phase
- Phase 2
- 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 25 to 89 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Type I or Type II diabetes mellitus with center involving diabetic macular edema with previous treatment
- HbA1c ≤12%
- BCVA in study eye 78 to 25 ETDRS letters (~20/32 to 20/320) at screening
- Decreased visual acuity attributable primarily due to CI - DME
- Demonstrate clinical response to aflibercept injection in the study eye
- Provide written informed consent
Who cannot join
- Women of childbearing potential
- Neovascularization in the study eye from a cause other than DR
- Evidence in the study eye of optic nerve pallor on clinical examination
- History of pan retinal photocoagulation in the study eye
- Any concurrent ocular condition in the study eye other than CI-DME that could require surgical intervention within 6 months or any condition in the study eye that may increase the risk to the participant, require either medical or surgical intervention during the study to prevent or treat vision loss, or interfere with the study procedures or assessments
- Presence of an implant in the study eye at screening (excluding intraocular lens)
Show all 13Show fewer
- Any condition in the investigator's opinion that could limit VA improvement in the study eye
- Active or history of glaucoma, steroid response, or ocular hypertension
- Any prior intravitreal steroid injection in the study eye within 6 months prior to screening, administration in the study eye of Ozudrex® within 12 months prior to screening, or administration in the study eye if Iluven® within 36 months prior to screening
- Diabetic macular edema diagnosis ≥ 7 years
- History of chronic renal failure requiring dialysis or kidney transplant
- Participation in any other gene therapy study or receipt of any investigational product within 30 days prior to enrollment or 5 half-lives of the investigational product, whichever is longer, or any plans to use an investigational product within 6 months following enrollment
- Note: Other inclusion/exclusion criteria apply
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
Sierra Eye Associates
Reno, Nevada, 89502
Clinical Research Study Coordinator, 775-329-0286, jvannavong@sierraeyeassociates.com
Arshad M Khanani, MD
About this study
Official title: A Randomized, Open Label, Controlled, Phase 2 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 AAV Gene Therapy Administered Via Subretinal Delivery in Participants With Center Involved Diabetic Macular Edema
Phase 2 open label, randomized, active controlled, dose-ranging trial in adults with Center Involved - Diabetic Macular Edema (CI - DME)
Read the detailed description
This Phase 2 trial is an open label, randomized, active controlled, dose-ranging trial to allow for preliminary safety and efficacy data on subretinal (SR) delivery of RGX-314 in participants with CI - DME
- Conditions
- Diabetic Macular Edema
- Being tested
- RGX-314 Dose 1, RGX-314 Dose 2, Aflibercept (2.0 mg)
- Keywords
- diabetic macular edema, gene therapy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RGX-314 Dose 1Genetic
AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)
RGX-314 Dose 2Genetic
AAV8 vector containing a transgene for anti-VEGF fab (Dose 2)
Aflibercept (2.0 mg)Biological
Commercially available Active Comparator
| Study group | Role | What they receive |
|---|---|---|
| RGX-314 Treatment Arm (Dose 1) | Experimental | Genetic: RGX-314 Dose 1
- AAV8 vector containing a transgene for anti-VEGF fab (Dose 1) Receives: Genetic: RGX-314 Dose 1 |
| Treatment Arm (Dose 2) | Experimental | Genetic: RGX-314 Dose 2
-AAV8 vector containing a transgene for anti-VEGF fab (Dose 2) Receives: Genetic: RGX-314 Dose 2 |
| Aflibercept Treatment Arm | Active comparator | Biological: Aflibercept IVT
* Commercially available Active Comparator
* Other Names: Eylea Receives: Biological: Aflibercept (2.0 mg) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean change from baseline in BCVA in the study eye at Week 52
To evaluate the effect of RGX-314 on BCVA at Week 52
Measured: 52 Weeks
Secondary outcomes (15)
Mean change from baseline in BCVA in the study eye over time
To evaluate the effect of RGX-314 on BCVA over time
Measured: 52 Weeks
Proportion of participants with BCVA of 20/40 or better (ETDRS ≥ 69 letters) in the study eye over time
To evaluate the effect of RGX-314 on BCVA over time
Measured: 52 Weeks
Mean change from baseline in central subfield thickness (CST) in the study at week 52
To evaluate the effect of RGX-314 on anatomic outcomes and CST (as determined by SD OCT measurement) at Week 52
Measured: 52 Weeks
Mean change from baseline in CST in the study eye on SD-OCT at Week 26 and Week 52
To evaluate the effect of RGX-314 on anatomic outcomes and CST (as determined by SD OCT measurement) at Week 52
Measured: 52 Weeks
Proportion of participants with an absence of CI-DME in the study eye at Week 26 and Week 52
To evaluate the effect of RGX-314 on anatomic outcomes and CST (as determined by SD OCT measurement) at Week 52
Measured: 52 Weeks
Proportion of participants achieving a 2-step or greater improvement or any improvement (a 1-step or greater improvement) in DR in the study eye by ETDRS DRSS on 4 widefield digital stereoscopic fundus photography at Week 52
To evaluate the effect of RGX-314 on DR by the ETDRS DRSS at Week 52
Measured: 52 Weeks
Incidences of overall and ocular adverse events (AEs)
To assess the safety and tolerability of RGX-314
Measured: 52 Weeks
Proportion of participants requiring any additional intervention for ocular diabetic complications to Week 52
To evaluate the need for additional standard of care intervention due to ocular diabetic complications
Measured: 52 Weeks
Proportion of participants with any sight-threatening ocular diabetic complications to Week 52
To evaluate the need for additional standard of care intervention due to ocular diabetic complications
Measured: 52 Weeks
Proportion of participants developing ocular diabetic complications requiring treatment per standard of care (anti-VEGF treatment, steroid treatment, pan retinal photocoagulation, laser, or surgery) through Week 52
To evaluate the need for additional standard of care intervention due to ocular diabetic complications
Measured: 52 Weeks
Proportion of participants developing ocular diabetic complications (e.g., neovascularization due to DR) requiring treatment per standard of care (pan retinal photocoagulation or anti-VEGF treatment) through Week 52
To evaluate the need for additional standard of care intervention due to ocular diabetic complications
Measured: 52 Weeks
Proportion of participants developing ocular diabetic complications (e.g., retinal detachments) requiring surgical intervention per standard of care through Week 52
To evaluate the need for additional standard of care intervention due to ocular diabetic complications
Measured: 52 Weeks
Aqueous RGX-314 transgene (TP) concentration at assessed time points
To measure aqueous RGX-314 TP concentrations
Measured: 52 Weeks
Proportion of participants with visual stability in the study eye from baseline to Week 52
To evaluate the effect of RGX-314 on vision outcomes
Measured: 52 Weeks
Proportion of participants with vision gain or vision loss ≥ to 5, 10, or 15 ETDRS letters in the study eye from baseline to Week 52
To evaluate the effect of RGX-314 on vision outcomes
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 posted24 Apr 2025
- Study start18 Mar 2025
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diabetic Macular EdemaStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sierra Eye Associates
- Sponsor type
- University, hospital or other
- Collaborators
- REGENXBIO Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06942520
- Sponsor's protocol ID
- SEA1979
- Acronym
- ELAAVATE
- Status last verified
- Apr 2025
- First submitted
- 3 Dec 2024
- Last updated
- 24 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Open Label, Controlled, Phase 2 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 AAV Gene Therapy Administered Via Subretinal Delivery in Participants With Center Involved Diabetic Macular Edema
- ClinicalTrials.gov ID
- NCT06942520
- Acronym / sponsor ID
- ELAAVATE / SEA1979
- Conditions
- Diabetic Macular Edema
- Keywords
- diabetic macular edema, gene therapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Sierra Eye AssociatesUniversity, hospital or other
- Collaborators
- REGENXBIO Inc.
- Study start
- 18 Mar 2025 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 24 Apr 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.
Phase 2 open label, randomized, active controlled, dose-ranging trial in adults with Center Involved - Diabetic Macular Edema (CI - DME)
Detailed description
This Phase 2 trial is an open label, randomized, active controlled, dose-ranging trial to allow for preliminary safety and efficacy data on subretinal (SR) delivery of RGX-314 in participants with CI - DME
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| RGX-314 Treatment Arm (Dose 1) | Experimental | Genetic: RGX-314 Dose 1Genetic: RGX-314 Dose 1 - AAV8 vector containing a transgene for anti-VEGF fab (Dose 1) |
| Treatment Arm (Dose 2) | Experimental | Genetic: RGX-314 Dose 2Genetic: RGX-314 Dose 2 -AAV8 vector containing a transgene for anti-VEGF fab (Dose 2) |
| Aflibercept Treatment Arm | Active comparator | Biological: Aflibercept (2.0 mg)Biological: Aflibercept IVT * Commercially available Active Comparator * Other Names: Eylea |
Who may qualify
- Ages
- 25 to 89 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Type I or Type II diabetes mellitus with center involving diabetic macular edema with previous treatment
- HbA1c ≤12%
- BCVA in study eye 78 to 25 ETDRS letters (~20/32 to 20/320) at screening
- Decreased visual acuity attributable primarily due to CI - DME
- Demonstrate clinical response to aflibercept injection in the study eye
Key exclusion criteria (13 listed)
- Women of childbearing potential
- Neovascularization in the study eye from a cause other than DR
- Evidence in the study eye of optic nerve pallor on clinical examination
- History of pan retinal photocoagulation in the study eye
- Any concurrent ocular condition in the study eye other than CI-DME that could require surgical intervention within 6 months or any condition in the study eye that may increase the risk to the participant, require either medical or surgical intervention during the study to prevent or treat vision loss, or interfere with the study procedures or assessments
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 |
|---|---|---|
| RGX-314 Dose 1AAV8 vector containing a transgene for anti-VEGF fab (Dose 1) | Genetic | RGX-314 Treatment Arm (Dose 1) |
| RGX-314 Dose 2AAV8 vector containing a transgene for anti-VEGF fab (Dose 2) | Genetic | Treatment Arm (Dose 2) |
| Aflibercept (2.0 mg)Commercially available Active Comparator | Biological | Aflibercept Treatment Arm |
Primary outcome
Mean change from baseline in BCVA in the study eye at Week 52
Time frame: 52 Weeks
To evaluate the effect of RGX-314 on BCVA at Week 52
15 secondary outcomes
Mean change from baseline in BCVA in the study eye over time
Time frame: 52 Weeks
Proportion of participants with BCVA of 20/40 or better (ETDRS ≥ 69 letters) in the study eye over time
Time frame: 52 Weeks
Mean change from baseline in central subfield thickness (CST) in the study at week 52
Time frame: 52 Weeks
Mean change from baseline in CST in the study eye on SD-OCT at Week 26 and Week 52
Time frame: 52 Weeks
Proportion of participants with an absence of CI-DME in the study eye at Week 26 and Week 52
Time frame: 52 Weeks
Proportion of participants achieving a 2-step or greater improvement or any improvement (a 1-step or greater improvement) in DR in the study eye by ETDRS DRSS on 4 widefield digital stereoscopic fundus photography at Week 52
Time frame: 52 Weeks
Incidences of overall and ocular adverse events (AEs)
Time frame: 52 Weeks
Proportion of participants requiring any additional intervention for ocular diabetic complications to Week 52
Time frame: 52 Weeks
Proportion of participants with any sight-threatening ocular diabetic complications to Week 52
Time frame: 52 Weeks
Proportion of participants developing ocular diabetic complications requiring treatment per standard of care (anti-VEGF treatment, steroid treatment, pan retinal photocoagulation, laser, or surgery) through Week 52
Time frame: 52 Weeks
Proportion of participants developing ocular diabetic complications (e.g., neovascularization due to DR) requiring treatment per standard of care (pan retinal photocoagulation or anti-VEGF treatment) through Week 52
Time frame: 52 Weeks
Proportion of participants developing ocular diabetic complications (e.g., retinal detachments) requiring surgical intervention per standard of care through Week 52
Time frame: 52 Weeks
Aqueous RGX-314 transgene (TP) concentration at assessed time points
Time frame: 52 Weeks
Proportion of participants with visual stability in the study eye from baseline to Week 52
Time frame: 52 Weeks
Proportion of participants with vision gain or vision loss ≥ to 5, 10, or 15 ETDRS letters in the study eye from baseline to Week 52
Time frame: 52 Weeks
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 24 Apr 2025.
- Individual participant dataSharing planned: Undecided
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Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With and Without Diabetic Retinopathy
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RGX-314 Dose 1, RGX-314 Dose 2 and Aflibercept (2.0 mg) in people with Diabetic Macular Edema, run by Sierra Eye Associates 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 6 inclusion and 13 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 24 Apr 2025. 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Research Study Coordinator (775-329-0286, jvannavong@sierraeyeassociates.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean change from baseline in BCVA in the study eye at Week 52 (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 24 Apr 2025.