4D-150 in Patients With Diabetic Macular Edema
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ClinicalTrials.gov Identifier: NCT05930561 |
Recruitment Status :
Recruiting
First Posted : July 5, 2023
Last Update Posted : November 13, 2023
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Diabetic Macular Edema Diabetic Retinopathy | Biological: 4D-150 IVT Biological: Aflibercept IVT | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 72 participants |
Allocation: | Non-Randomized |
Intervention Model: | Parallel Assignment |
Intervention Model Description: | In Dose Confirmation (Part 1), subjects (n=12-18) will be sequentially assigned to one of two open-label dose levels of 4D-150 (n=6-9 per dose level). In Dose Expansion (Part 2), subjects (n=54) will be randomized to receive one of two dose levels of 4D-150 (n=18 per dose level) based on results from Dose Confirmation, or aflibercept control (n=18). Subjects will be masked to treatment arm in Dose Expansion (Part 2). |
Masking: | Double (Participant, Outcomes Assessor) |
Masking Description: | No masking in Dose Confirmation (Part 1). In Dose Expansion (Part 2), participants and outcomes assessors will be masked to treatment arm for the duration of the trial. |
Primary Purpose: | Treatment |
Official Title: | A Phase 2 Randomized, Active-Controlled, Double-masked Trial of Intravitreal 4D-150 Gene Therapy in Adults With Diabetic Macular Edema (SPECTRA) |
Actual Study Start Date : | August 9, 2023 |
Estimated Primary Completion Date : | February 29, 2028 |
Estimated Study Completion Date : | July 1, 2028 |
Arm | Intervention/treatment |
---|---|
Experimental: 4D-150 Part 1 Dose Confirmation Dose Level 1
4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.
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Biological: 4D-150 IVT
4D-150: AAV-based gene therapy comprised of miRNA targeting VEGF-C and codon-optimized sequence encoding aflibercept. |
Experimental: 4D-150 Part 1 Dose Confirmation Dose Level 2
4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.
|
Biological: 4D-150 IVT
4D-150: AAV-based gene therapy comprised of miRNA targeting VEGF-C and codon-optimized sequence encoding aflibercept. |
Experimental: 4D-150 Part 2 Dose Expansion Dose Level 1
4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.
|
Biological: 4D-150 IVT
4D-150: AAV-based gene therapy comprised of miRNA targeting VEGF-C and codon-optimized sequence encoding aflibercept. |
Experimental: 4D-150 Part 2 Dose Expansion Dose Level 2
4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.
|
Biological: 4D-150 IVT
4D-150: AAV-based gene therapy comprised of miRNA targeting VEGF-C and codon-optimized sequence encoding aflibercept. |
Active Comparator: 4D-150 Part 2 Dose Expansion Control
Aflibercept at a fixed regimen will be administered.
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Biological: Aflibercept IVT
Commercially available Active Comparator
Other Name: Eylea |
- Annualized number of aflibercept injections in the study eye [ Time Frame: 52 weeks ]
- Mean cumulative number of aflibercept injections over time [ Time Frame: 52 weeks ]
- Change from baseline in BCVA as assessed using the ETDRS Visual Acuity Chart [ Time Frame: 104 weeks ]
- Change from baseline in CST measured by spectral domain optical coherence tomography (SD-OCT) [ Time Frame: 104 weeks ]
- Percentage of subjects with a ≥2 and ≥3-Step Diabetic Retinopathy Severity (DRS) improvement from baseline on the ETDRS-DRSS [ Time Frame: 104 weeks ]
- Incidence and severity of treatment-emergent adverse events and serious adverse events, including clinically significant changes in safety parameters [ Time Frame: 104 weeks ]
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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05930561
Contact: 4DMT Patient Advocacy | (888) 748-8881 | clinicaltrials@4DMT.com |
United States, Arizona | |
Retinal Research Institute LLC | Recruiting |
Phoenix, Arizona, United States, 85053 | |
Contact: Michelle Zunitch Mixhelle.Zunitch@researchavp.com | |
Principal Investigator: Mark Barakat, MD | |
United States, Colorado | |
Retina Consultants of Southern Colorado | Recruiting |
Colorado Springs, Colorado, United States, 80909 | |
Contact: Kari Winger Kwinger@coloradoretina.com | |
Principal Investigator: Stanford Taylor, MD | |
United States, Florida | |
National Ophthalmic Research Institute | Recruiting |
Fort Myers, Florida, United States, 33912 | |
Contact: Lee Toleman leet@eye.md | |
Principal Investigator: Katrina Mears, MD | |
Retina Specialty Institute | Recruiting |
Pensacola, Florida, United States, 32503 | |
Contact: Veronica Wilder VWilder@retinaspecialty.com | |
Principal Investigator: Sunil Gupta, MD | |
Retina Vitreous Associates of FL | Recruiting |
Saint Petersburg, Florida, United States, 33711 | |
Contact: Allison Calvanese ACalvanese@rvaf.com | |
Principal Investigator: David EIchenbaum, MD | |
United States, Indiana | |
Raj K Maturi MD PC | Recruiting |
Carmel, Indiana, United States, 46290 | |
Contact: Lorraine White Lorrainew@midwesteye.com | |
Principal Investigator: Raj Maturi, MD | |
United States, Maryland | |
Cumberland Valley Retina Consultants | Recruiting |
Hagerstown, Maryland, United States, 21740 | |
Contact: Erica Buls ericab@retinacare.net | |
Principal Investigator: Allen Hu, MD | |
United States, Nevada | |
Sierra Eye Associates | Recruiting |
Reno, Nevada, United States, 89502 | |
Contact: Lindsey Godoy LGodoy@midwesteye.com | |
Principal Investigator: Arshad Khanani, MD | |
United States, New Mexico | |
Vision Research Center Eye Associates of New Mexico | Recruiting |
Albuquerque, New Mexico, United States, 87109 | |
Contact: Darlene Harbour EDHarbour@eyenm.com | |
Principal Investigator: John Pitcher III, MD | |
United States, North Carolina | |
Western Carolina Retinal Associates | Recruiting |
Asheville, North Carolina, United States, 28803 | |
Contact: Lea Raymer ldoll@aea1961.com | |
Principal Investigator: Cameron Stone, MD | |
United States, Oregon | |
Verum Research, LLC | Recruiting |
Eugene, Oregon, United States, 97401 | |
Contact: Ryan Lieben rlebien@verumresearch.com | |
Principal Investigator: Albert Edwards, MD | |
United States, Pennsylvania | |
Erie Retina Research | Recruiting |
Erie, Pennsylvania, United States, 16507 | |
Contact: Beth Hess ehess@erieretinaresearch.com | |
Principal Investigator: David Almeida, MD | |
United States, Texas | |
Austin Clinical Research | Recruiting |
Austin, Texas, United States, 78750 | |
Contact: Ivana Gunderson igunderson@austinclinicalresearch.com | |
Principal Investigator: Brian Berger, MD | |
United States, Virginia | |
Piedmont Eye Center | Recruiting |
Lynchburg, Virginia, United States, 24502 | |
Contact: Susan Shelton sshelton@piedmonteye.com | |
Principal Investigator: Golnaz Javey, MD | |
Puerto Rico | |
Emanuelli Research and Development Center | Recruiting |
Arecibo, Puerto Rico, X00612 | |
Contact: Diane Perez dperez@erdpr.com | |
Principal Investigator: Andres Emanuelli, MD |
Study Director: | Schonmei Lee, MD | 4D Molecular Therapeutics |
Responsible Party: | 4D Molecular Therapeutics |
ClinicalTrials.gov Identifier: | NCT05930561 |
Other Study ID Numbers: |
4D-150-C002 |
First Posted: | July 5, 2023 Key Record Dates |
Last Update Posted: | November 13, 2023 |
Last Verified: | November 2023 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Retinal gene therapy Intravitreal gene therapy DME SPECTRA |
Macular Edema Diabetic Retinopathy Edema Macular Degeneration Retinal Degeneration Retinal Diseases Eye Diseases Diabetic Angiopathies Vascular Diseases Cardiovascular Diseases |
Diabetes Complications Diabetes Mellitus Endocrine System Diseases Aflibercept Angiogenesis Inhibitors Angiogenesis Modulating Agents Growth Substances Physiological Effects of Drugs Growth Inhibitors Antineoplastic Agents |