Gene therapy for diabetic macular edema
A Phase 2 Randomized, Active-Controlled, Double-masked Trial of Intravitreal 4D-150 Gene Therapy in Adults With Diabetic Macular Edema (SPECTRA)
PHASE2 · 4D Molecular Therapeutics · NCT05930561
This study is testing a new gene therapy called 4D-150 to see if it can improve vision and retinal health in adults with diabetic macular edema.
Quick facts
| Phase | PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 72 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | 4D Molecular Therapeutics (industry) |
| Drugs / interventions | bevacizumab, sunitinib |
| Locations | 15 sites (Phoenix, Arizona and 14 other locations) |
| Trial ID | NCT05930561 on ClinicalTrials.gov |
What this trial studies
This Phase 2 trial evaluates the safety and efficacy of 4D-150, a retinal gene therapy, in adults with diabetic macular edema (DME). The study is designed as a randomized, active-controlled, double-masked, dose-ranging trial and will be conducted in two parts: Dose Confirmation and Dose Expansion. Participants will receive either 4D-150 or aflibercept through intravitreal injection, with the aim of assessing improvements in visual acuity and retinal health.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older with Type I or Type II diabetes and clinically significant diabetic macular edema affecting their vision.
Not a fit: Patients whose macular edema is caused by factors other than diabetic macular edema will not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could significantly improve vision and quality of life for patients suffering from diabetic macular edema.
How similar studies have performed: Other studies have shown promise with similar gene therapy approaches, indicating potential for success in this area.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * ≥18 years of age * Type I or Type II diabetes mellitus with macular thickening secondary to DME involving the center of the fovea; diagnosis of DME must be within 2 years of Screening * Demonstrate clinical response to on-study aflibercept injection in the study eye. * Decreased visual acuity attributable primarily to DME * BCVA in the study eye between 25 and 83 ETDRS letters, inclusive (\~20/320 and 20/25, respectively) at Screening * Study eye amenable to IVT injection * Sufficient clear ocular media, pupil dilation and fixation in the study eye to permit adequate imaging; ability to perform tests of visual and retinal function and structure; and ability to comply with other protocol-specified procedures * Provide written informed consent Exclusion Criteria: * Macular edema in the study eye considered to be secondary to a cause other than DME * Systemic anti-VEGF treatment (e.g. sunitinib, bevacizumab, pazopanib) within 6 months, or anticipated need for systemic anti-VEGF therapy during study participation * Systemic corticosteroids (oral, intravenous, intramuscular, intra-articular) or other immunosuppressive medications within 3 months * Received an investigational drug, agent, device, or therapy (ocular or non-ocular) in the 3 months (or at least 5 half-lives, whichever is longer) prior to 4D-150 administration (Day 1) * Prior gene therapy (ocular or non-ocular) and/or ocular stem cell therapy in either eye * Any concurrent ocular condition in the study eye that is likely to require surgical intervention (e.g. cataract surgery) during the 2 year (104 week) study duration Note: Other inclusion/exclusion criteria apply.
Where this trial is running
Phoenix, Arizona and 14 other locations
- Retinal Research Institute LLC — Phoenix, Arizona, United States (RECRUITING)
- Retina Consultants of Southern Colorado — Colorado Springs, Colorado, United States (RECRUITING)
- National Ophthalmic Research Institute — Fort Myers, Florida, United States (RECRUITING)
- Retina Specialty Institute — Pensacola, Florida, United States (RECRUITING)
- Retina Vitreous Associates of Florida — Saint Petersburg, Florida, United States (RECRUITING)
- Raj K Maturi MD PC — Carmel, Indiana, United States (RECRUITING)
- Cumberland Valley Retina Consultants — Hagerstown, Maryland, United States (RECRUITING)
- Sierra Eye Associates — Reno, Nevada, United States (RECRUITING)
- Vision Research Center Eye Associates of New Mexico — Albuquerque, New Mexico, United States (RECRUITING)
- Western Carolina Retinal Associates — Asheville, North Carolina, United States (RECRUITING)
- Verum Research, LLC — Eugene, Oregon, United States (RECRUITING)
- Erie Retina Research — Erie, Pennsylvania, United States (RECRUITING)
- Austin Clinical Research — Austin, Texas, United States (RECRUITING)
- Piedmont Eye Center — Lynchburg, Virginia, United States (RECRUITING)
- Emanuelli Research and Development Center — Arecibo, Puerto Rico (RECRUITING)
Study contacts
- Study coordinator: 4DMT Patient Advocacy
- Email: clinicaltrials@4DMT.com
- Phone: (888) 748-8881
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Diabetic Macular Edema, Diabetic Retinopathy, Retinal gene therapy, Intravitreal gene therapy, DME, SPECTRA