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4D-150 in Patients With Neovascular (Wet) Age-Related Macular Degeneration

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05197270
Recruitment Status : Recruiting
First Posted : January 19, 2022
Last Update Posted : October 20, 2022
Sponsor:
Information provided by (Responsible Party):
4D Molecular Therapeutics

Study Description
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Brief Summary:
Phase 1/2 dose-escalation and randomized, controlled, masked expansion trial in adults with wet AMD undergoing active anti-VEGF treatment

Condition or disease Intervention/treatment Phase
Neovascular (Wet) Age-Related Macular Degeneration Biological: 4D-150 IVT Biological: Aflibercept IVT Phase 1 Phase 2

Detailed Description:

This Phase 1/2 trial is a prospective, multicenter, Phase 1/2 dose-escalation and randomized, controlled, masked expansion trial in adults with wet AMD undergoing active anti-VEGF treatment who have demonstrated a clinical response consistent with anti-VEGF activity. The trial will be conducted in 2 parts: Dose Escalation and Dose Expansion.

After receiving one time administration of 4D-150 by intravitreal injection, subjects will undergo assessments at monthly intervals for 24 months to assess safety and efficacy outcomes.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 65 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: In Dose Escalation, the safety and tolerability of multiple dose levels of 4D-150 will be examined following an open-label, 3+3 dose escalation design (n=3 per dose level). In Dose Expansion, subjects (n=50) will be randomized to receive one of 2 dose levels of 4D-150 (n=20 for each dose level) based on results from Dose Escalation, or aflibercept (n=10).
Masking: Single (Outcomes Assessor)
Masking Description: Dose-escalation will be open-label. During Dose Expansion, outcome assessors will be masked to treatment assignment. Other site personnel (including investigator) and subjects will be unmasked to treatment assignment but will be masked to 4D-150 dose level. The Sponsor and its representatives and the site dose preparation/pharmacist will be unmasked to treatment assignment.
Primary Purpose: Treatment
Official Title: A Phase 1/2 Dose-Escalation and Randomized, Controlled, Masked Expansion Trial of Intravitreal 4D-150 Gene Therapy in Adults With Neovascular (Wet) Age-Related Macular Degeneration
Actual Study Start Date : December 9, 2021
Estimated Primary Completion Date : May 30, 2026
Estimated Study Completion Date : September 30, 2026

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: 4D-150 Dose Escalation up to 4 dose levels
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 Dose Expansion Dose 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 Dose Expansion Dose 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 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. Incidence and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs), including clinically significant changes in safety parameters [ Time Frame: 52 weeks ]

Secondary Outcome Measures :
  1. Time to receiving the first supplemental aflibercept injection [ Time Frame: 52 weeks ]
  2. Percentage of subjects requiring supplemental aflibercept injections over 52 weeks [ Time Frame: 52 weeks ]
  3. Number of supplemental aflibercept injections over 52 weeks [ Time Frame: 52 weeks ]
  4. Change from baseline in BCVA over time (up to 52 weeks) as assessed using the ETDRS Visual Acuity Chart [ Time Frame: 52 weeks ]
  5. Change from baseline in central subfield thickness (CST) over time (up to 52 weeks) measured by spectral domain optical coherence tomography (SD-OCT) [ Time Frame: 52 weeks ]

Eligibility Criteria
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Ages Eligible for Study:   50 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • ≥50 years of age
  • Diagnosed with CNV secondary to AMD
  • BCVA ETDRS Snellen equivalent for dose escalation between ~20/50 and ~20/320 (sentinel subjects only) or ~20/32 and ~20/320, or for dose expansion ~20/25 and~20/200
  • Currently receiving anti-VEGF treatment in the study eye (minimum of 6 injections within the last 12 months) and has demonstrated a clinical response consistent with anti-VEGF activity within 12 months prior to screening

Exclusion Criteria:

  • Fibrosis, atrophy, or retinal pigment epithelial tear in the center of the fovea in the study eye, or any condition preventing visual acuity improvement
  • Prior treatment with photodynamic therapy or retinal laser
  • History of uveitis in either eye
  • Any other pre-existing eye conditions or surgical complications that would preclude participation in an interventional clinical trial or interfere with the interpretation of study endpoints
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): NCT05197270


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

Locations
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United States, Arizona
Barnet Delaney Perkins Eye Center Recruiting
Phoenix, Arizona, United States, 85016
Contact: Lori Slonecker       Lori.Slonecker@bdpec.com   
Principal Investigator: Suhail Alam, M.D.         
United States, California
California Retina Consultants Recruiting
Oxnard, California, United States, 93036
Contact: Ellie Gonzalez       ellie@californiaretina.com   
Principal Investigator: Dante Pieramici, M.D.         
Retinal Consultants Medical Group Recruiting
Sacramento, California, United States, 95841
Contact: Mira Cukrov       cukrovm@retinalmd.com   
Principal Investigator: Joel Pearlman, M.D., Ph.D.         
United States, Florida
Vitreo Retinal Associates Recruiting
Gainesville, Florida, United States, 32607
Contact: Jing Yang       jingzhang@vra-pa.com   
Principal Investigator: Christine Kay, M.D.         
Florida Eye Associates Recruiting
Melbourne, Florida, United States, 32901
Contact: Yvonne Santiago       ysantiago@floridaeyeassociates.com   
Principal Investigator: Vrinda Hershberger, M.D., Ph.D.         
Retinal Specialty Institute Recruiting
Pensacola, Florida, United States, 32503
Contact: Vera Watkins, R.N.       vwatkins@retinaspecialty.com   
Principal Investigator: Sunil Gupta, M.D.         
Retina Vitreous Associates of Florida Recruiting
Tampa, Florida, United States, 33607
Contact: Allison Calvanese       acalvanese@rvaf.com   
Principal Investigator: David Eichenbaum, M.D.         
United States, Illinois
University Retina and Macula Associates Recruiting
Oak Forest, Illinois, United States, 60452
Contact: Breanne Kirby       bkirby@uretina.com   
Principal Investigator: Veeral Sheth, M.D.         
United States, Maryland
Cumberland Valley Retina Consultants Recruiting
Hagerstown, Maryland, United States, 21740
Contact: Brittany Carson       BrittanyC@retinacare.net   
Principal Investigator: Allen Hu, M.D.         
United States, Nevada
Sierra Eye Associates Recruiting
Reno, Nevada, United States, 89502
Contact: Naisha Shamim       nshamim@sierraeyeassociates.com   
Principal Investigator: Arshad Khanani, M.D., M.A.         
United States, Texas
Austin Clinical Research Recruiting
Austin, Texas, United States, 78750
Contact: Ivana Gunderson       igunderson@retinaresearchcenter.net   
Principal Investigator: Fuad Makkouk, M.D.         
Retina Consultants of Texas Recruiting
Houston, Texas, United States, 77030
Contact: Chelsey Moore       chelsey.moore@retinaconsultantstexas.com   
Principal Investigator: David Brown, M.D.         
Valley Retina Institute, PA Recruiting
McAllen, Texas, United States, 78503
Contact: Yesenia Salinas       yesenia.salinas@eyeptcare.com   
Principal Investigator: Victor Gonzalez, M.D.         
Retina Consultants of Texas Recruiting
The Woodlands, Texas, United States, 77384
Contact: Kelly Reddin       kelly.reddin@retinaconsultantstexas.com   
Principal Investigator: Charles Wykoff, M.D., Ph.D.         
Sponsors and Collaborators
4D Molecular Therapeutics
Investigators
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Study Director: Chyong Nien, MD 4D Molecular Therapeutics
More Information
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Responsible Party: 4D Molecular Therapeutics
ClinicalTrials.gov Identifier: NCT05197270    
Other Study ID Numbers: 4D-150-C001
First Posted: January 19, 2022    Key Record Dates
Last Update Posted: October 20, 2022
Last Verified: October 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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:
Age-related macular degeneration
AMD
Exudative AMD
Exudative age-related macular degeneration
Neovascular AMD
Neovascular age-related macular degeneration
 Wet age-related macular degeneration
Wet macular degeneration
Wet AMD
wAMD
Retinal gene therapy
Intravitreal gene therapy
Additional relevant MeSH terms:
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Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases
Aflibercept
Angiogenesis Inhibitors
 Angiogenesis Modulating Agents
Growth Substances
Physiological Effects of Drugs
Growth Inhibitors
Antineoplastic Agents