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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
Sponsor:
Information provided by (Responsible Party):
4D Molecular Therapeutics

Study Description
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Brief Summary:
Phase 2 randomized, active-controlled, double-masked, dose-ranging trial in adults with Diabetic Macular Edema (DME).

Condition or disease Intervention/treatment Phase
Diabetic Macular Edema Diabetic Retinopathy Biological: 4D-150 IVT Biological: Aflibercept IVT Phase 2

Detailed Description:
This Phase 2 trial is a prospective, multicenter, randomized, active-controlled, double-masked dose-ranging trial to evaluate the safety and efficacy of 4D-150 in adults with DME. The trial will be conducted in two parts: Dose Confirmation and Dose Expansion.
Study Design
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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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Edema

Arms and Interventions
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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.
 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.
 Biological: Aflibercept IVT
Commercially available Active Comparator
Other Name: Eylea


Outcome Measures
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Primary Outcome Measures :
  1. Annualized number of aflibercept injections in the study eye [ Time Frame: 52 weeks ]

Secondary Outcome Measures :
  1. Mean cumulative number of aflibercept injections over time [ Time Frame: 52 weeks ]
  2. Change from baseline in BCVA as assessed using the ETDRS Visual Acuity Chart [ Time Frame: 104 weeks ]
  3. Change from baseline in CST measured by spectral domain optical coherence tomography (SD-OCT) [ Time Frame: 104 weeks ]
  4. Percentage of subjects with a ≥2 and ≥3-Step Diabetic Retinopathy Severity (DRS) improvement from baseline on the ETDRS-DRSS [ Time Frame: 104 weeks ]

Other Outcome Measures:
  1. Incidence and severity of treatment-emergent adverse events and serious adverse events, including clinically significant changes in safety parameters [ Time Frame: 104 weeks ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Contacts and Locations
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Information from the National Library of Medicine

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


Contacts
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Contact: 4DMT Patient Advocacy (888) 748-8881 clinicaltrials@4DMT.com

Locations
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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         
Sponsors and Collaborators
4D Molecular Therapeutics
Investigators
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Study Director: Schonmei Lee, MD 4D Molecular Therapeutics
More Information
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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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by 4D Molecular Therapeutics:
Retinal gene therapy
Intravitreal gene therapy
DME
SPECTRA
Additional relevant MeSH terms:
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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