search
Back to results

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

Primary Purpose

Neovascular (Wet) Age-Related Macular Degeneration

Status
Recruiting
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
4D-150 IVT
Aflibercept IVT
Sponsored by
4D Molecular Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Neovascular (Wet) Age-Related Macular Degeneration focused on measuring 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

Eligibility Criteria

50 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Barnet Delaney Perkins Eye CenterRecruiting
  • California Retina ConsultantsRecruiting
  • Retinal Consultants Medical GroupRecruiting
  • Colorado Retina AssociatesRecruiting
  • Rand Eye InstituteRecruiting
  • Vitreo Retinal AssociatesRecruiting
  • Florida Eye AssociatesRecruiting
  • Retinal Specialty InstituteRecruiting
  • Retina Vitreous Associates of FloridaRecruiting
  • University Retina and Macula AssociatesRecruiting
  • Retina Partners MidwestRecruiting
  • Cumberland Valley Retina ConsultantsRecruiting
  • Ophthalmic Consultants of Boston & Boston Eye Surgery and Laser CenterRecruiting
  • Sierra Eye AssociatesRecruiting
  • Western Carolina Retinal AssociatesRecruiting
  • Verum Research, LLCRecruiting
  • Mid Atlantic RetinaRecruiting
  • Palmetto Retina Center, LLCRecruiting
  • Tennessee RetinaRecruiting
  • Austin Retina Associates
  • Austin Clinical ResearchRecruiting
  • Retina Consultants of TexasRecruiting
  • Valley Retina Institute, PARecruiting
  • Retina Consultants of TexasRecruiting
  • Pacific Northwest Retina LLCRecruiting
  • Emanuelli Research and Development Center, LLCRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm 7

Arm Type

Experimental

Experimental

Experimental

Active Comparator

Experimental

Experimental

Experimental

Arm Label

4D-150 Dose Escalation up to 4 dose levels

4D-150 Dose Expansion Dose 1

4D-150 Dose Expansion Dose 2

4D-150 Dose Expansion Control

4D-150 Steroid Optimization

4D-150 Population Extension Dose 1

4D-150 Population Extension Dose 2

Arm Description

4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.

4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.

4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.

Aflibercept at a fixed regimen will be administered.

4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.

4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.

4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.

Outcomes

Primary Outcome Measures

Incidence and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs), including clinically significant changes in safety parameters

Secondary Outcome Measures

Time to receiving the first supplemental aflibercept injection
Percentage of subjects requiring supplemental aflibercept injections over 52 weeks
Number of supplemental aflibercept injections over 52 weeks
Change from baseline in BCVA over time (up to 52 weeks) as assessed using the ETDRS Visual Acuity Chart
Change from baseline in central subfield thickness (CST) over time (up to 52 weeks) measured by spectral domain optical coherence tomography (SD-OCT)

Full Information

First Posted
January 5, 2022
Last Updated
August 29, 2023
Sponsor
4D Molecular Therapeutics
search

1. Study Identification

Unique Protocol Identification Number
NCT05197270
Brief Title
4D-150 in Patients With Neovascular (Wet) Age-Related Macular Degeneration
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
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
December 9, 2021 (Actual)
Primary Completion Date
November 2024 (Anticipated)
Study Completion Date
November 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
4D Molecular Therapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Phase 1/2 dose-escalation and randomized, controlled, masked expansion trial in adults with wet AMD undergoing active anti-VEGF treatment
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 consists of Dose Escalation, Dose Expansion, Steroid Optimization and Population Extension Cohorts. 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. Only subjects that received 4D-150 will then enter a long-term follow-up (LTFU) period to assess long-term safety of 4D-150 gene therapy and duration of clinical activity through year 5 (60 months).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neovascular (Wet) Age-Related Macular Degeneration
Keywords
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
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=3-5 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). In Steroid Optimization (n=up to 40) and Population Extension (n=up to 45) Cohorts, subjects will be assigned sequentially to receive 4D-150 at doses cleared by the DSMC.
Masking
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 preparer/pharmacist will be unmasked to treatment assignment. Steroid Optimization and Population Extension will be open-label.
Allocation
Randomized
Enrollment
150 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
4D-150 Dose Escalation up to 4 dose levels
Arm Type
Experimental
Arm Description
4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.
Arm Title
4D-150 Dose Expansion Dose 1
Arm Type
Experimental
Arm Description
4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.
Arm Title
4D-150 Dose Expansion Dose 2
Arm Type
Experimental
Arm Description
4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.
Arm Title
4D-150 Dose Expansion Control
Arm Type
Active Comparator
Arm Description
Aflibercept at a fixed regimen will be administered.
Arm Title
4D-150 Steroid Optimization
Arm Type
Experimental
Arm Description
4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.
Arm Title
4D-150 Population Extension Dose 1
Arm Type
Experimental
Arm Description
4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.
Arm Title
4D-150 Population Extension Dose 2
Arm Type
Experimental
Arm Description
4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.
Intervention Type
Biological
Intervention Name(s)
4D-150 IVT
Intervention Description
4D-150: AAV-based gene therapy comprised of miRNA targeting VEGF-C and codon-optimized sequence encoding aflibercept
Intervention Type
Biological
Intervention Name(s)
Aflibercept IVT
Intervention Description
Commercially available Active Comparator Other Name: Eylea
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
Time to receiving the first supplemental aflibercept injection
Time Frame
52 weeks
Title
Percentage of subjects requiring supplemental aflibercept injections over 52 weeks
Time Frame
52 weeks
Title
Number of supplemental aflibercept injections over 52 weeks
Time Frame
52 weeks
Title
Change from baseline in BCVA over time (up to 52 weeks) as assessed using the ETDRS Visual Acuity Chart
Time Frame
52 weeks
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: ≥50 years of age Diagnosed with macular CNV secondary to AMD BCVA ETDRS Snellen equivalent for dose escalation between ~20/32 and ~20/320, or for dose expansion and population extension between ~20/25 and~20/200, for steroid optimization between ~20/25 and ~20/640 Currently receiving anti-VEGF treatment in the study eye and has demonstrated a clinical response consistent with anti-VEGF activity within 12 months prior to screening Exclusion Criteria: Any condition preventing visual acuity improvement in the study eye Prior treatment with photodynamic therapy or retinal laser in the study eye 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
4DMT Patient Advocacy
Phone
(888) 748-8881
Email
clinicaltrials@4DMT.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Chyong Nien, MD
Organizational Affiliation
4D Molecular Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
Barnet Delaney Perkins Eye Center
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Lori Slonecker
Email
Lori.Slonecker@bdpec.com
First Name & Middle Initial & Last Name & Degree
Suhail Alam, M.D.
Facility Name
California Retina Consultants
City
Oxnard
State/Province
California
ZIP/Postal Code
93036
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ellie Gonzalez
Email
ellie@californiaretina.com
First Name & Middle Initial & Last Name & Degree
Dante Pieramici, M.D.
Facility Name
Retinal Consultants Medical Group
City
Sacramento
State/Province
California
ZIP/Postal Code
95841
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Mira Cukrov
Email
cukrovm@retinalmd.com
First Name & Middle Initial & Last Name & Degree
Joel Pearlman, M.D., Ph.D.
Facility Name
Colorado Retina Associates
City
Lakewood
State/Province
Colorado
ZIP/Postal Code
80288
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sara Shaw
Email
sshaw@retinacolorado.com
First Name & Middle Initial & Last Name & Degree
Murtaza Adam, MD
Facility Name
Rand Eye Institute
City
Deerfield Beach
State/Province
Florida
ZIP/Postal Code
33064
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Damian Stega
Email
dstega@randeye.com
First Name & Middle Initial & Last Name & Degree
Carl Danzig, MD
Facility Name
Vitreo Retinal Associates
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32607
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jing Yang
Email
jingzhang@vra-pa.com
First Name & Middle Initial & Last Name & Degree
Christine Kay, M.D.
Facility Name
Florida Eye Associates
City
Melbourne
State/Province
Florida
ZIP/Postal Code
32901
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yvonne Santiago
Email
ysantiago@floridaeyeassociates.com
First Name & Middle Initial & Last Name & Degree
Vrinda Hershberger, M.D., Ph.D.
Facility Name
Retinal Specialty Institute
City
Pensacola
State/Province
Florida
ZIP/Postal Code
32503
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Vera Watkins, R.N.
Email
vwatkins@retinaspecialty.com
First Name & Middle Initial & Last Name & Degree
Sunil Gupta, M.D.
Facility Name
Retina Vitreous Associates of Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33607
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Allison Calvanese
Email
acalvanese@rvaf.com
First Name & Middle Initial & Last Name & Degree
David Eichenbaum, M.D.
Facility Name
University Retina and Macula Associates
City
Oak Forest
State/Province
Illinois
ZIP/Postal Code
60452
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Breanne Kirby
Email
bkirby@uretina.com
First Name & Middle Initial & Last Name & Degree
Veeral Sheth, M.D.
Facility Name
Retina Partners Midwest
City
Carmel
State/Province
Indiana
ZIP/Postal Code
46290
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Lorraine White
Email
startup@midwesteye.com
First Name & Middle Initial & Last Name & Degree
Raj Maturi, MD
Facility Name
Cumberland Valley Retina Consultants
City
Hagerstown
State/Province
Maryland
ZIP/Postal Code
21740
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Brittany Carson
Email
BrittanyC@retinacare.net
First Name & Middle Initial & Last Name & Degree
Allen Hu, M.D.
Facility Name
Ophthalmic Consultants of Boston & Boston Eye Surgery and Laser Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sandy Chyong
Email
schong@eyeboston.com
First Name & Middle Initial & Last Name & Degree
Jeffrey Heier, MD
Facility Name
Sierra Eye Associates
City
Reno
State/Province
Nevada
ZIP/Postal Code
89502
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Naisha Shamim
Email
nshamim@sierraeyeassociates.com
First Name & Middle Initial & Last Name & Degree
Arshad Khanani, M.D., M.A.
Facility Name
Western Carolina Retinal Associates
City
Asheville
State/Province
North Carolina
ZIP/Postal Code
28803
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
McCayla Hall
Email
mchall@aea1961.com
First Name & Middle Initial & Last Name & Degree
Cameron Stone, MD
Facility Name
Verum Research, LLC
City
Eugene
State/Province
Oregon
ZIP/Postal Code
97401
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ryan Lebien
Email
rlebien@versumresearch.com
First Name & Middle Initial & Last Name & Degree
Albert Edwards, MD
Facility Name
Mid Atlantic Retina
City
Bethlehem
State/Province
Pennsylvania
ZIP/Postal Code
18017
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ranya Girgis
Email
dstega@randeye.com
First Name & Middle Initial & Last Name & Degree
Ajay Kuriyan, MD
Facility Name
Palmetto Retina Center, LLC
City
West Columbia
State/Province
South Carolina
ZIP/Postal Code
29169
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Pamala Tims
Email
ptims@palmettoretina.com
First Name & Middle Initial & Last Name & Degree
John Wells, MD
Facility Name
Tennessee Retina
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37203
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Lisa Walden
Email
LWalden@tnretina.com
First Name & Middle Initial & Last Name & Degree
Carl Awh, MD
Facility Name
Austin Retina Associates
City
Austin
State/Province
Texas
ZIP/Postal Code
78705
Country
United States
Individual Site Status
Withdrawn
Facility Name
Austin Clinical Research
City
Austin
State/Province
Texas
ZIP/Postal Code
78750
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ivana Gunderson
Email
igunderson@retinaresearchcenter.net
First Name & Middle Initial & Last Name & Degree
Fuad Makkouk, M.D.
Facility Name
Retina Consultants of Texas
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Chelsey Moore
Email
chelsey.moore@retinaconsultantstexas.com
First Name & Middle Initial & Last Name & Degree
David Brown, M.D.
Facility Name
Valley Retina Institute, PA
City
McAllen
State/Province
Texas
ZIP/Postal Code
78503
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yesenia Salinas
Email
yesenia.salinas@eyeptcare.com
First Name & Middle Initial & Last Name & Degree
Victor Gonzalez, M.D.
Facility Name
Retina Consultants of Texas
City
The Woodlands
State/Province
Texas
ZIP/Postal Code
77384
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kelly Reddin
Email
kelly.reddin@retinaconsultantstexas.com
First Name & Middle Initial & Last Name & Degree
Charles Wykoff, M.D., Ph.D.
Facility Name
Pacific Northwest Retina LLC
City
Bellevue
State/Province
Washington
ZIP/Postal Code
98004
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Erdan Sun
Email
e.sun@pnwretina.com
First Name & Middle Initial & Last Name & Degree
Todd Klesert, MD
Facility Name
Emanuelli Research and Development Center, LLC
City
Arecibo
ZIP/Postal Code
00612
Country
Puerto Rico
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Diane Perez
Email
dperez@erdpr.com
First Name & Middle Initial & Last Name & Degree
Andres Emanuelli, MD

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

We'll reach out to this number within 24 hrs