Actively Recruiting
4D-710 in Adult Patients With Cystic Fibrosis
Led by 4D Molecular Therapeutics · Updated on 2026-03-11
30
Participants Needed
20
Research Sites
478 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
CONDITIONS
Official Title
4D-710 in Adult Patients With Cystic Fibrosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 18 years and older
- Confirmed diagnosis of cystic fibrosis and CF lung disease with sweat chloride �3e�3d 60 mmol/L
- Bi-allelic mutations in the CFTR gene or a single mutation with clinical signs of CF lung disease
- Ineligible for CFTR modulator therapy or discontinued due to adverse effects
- Forced expiratory volume in 1 second (FEV1) between 50% and 90% of predicted at screening
- Resting oxygen saturation �3e�3d 92% on room air at screening
- Currently on a stable dose of CFTR modulator therapy for at least 60 days prior to screening (sub-study only)
- FEV1 �3e�3d 40% and �3c 70% predicted at screening, and/or experienced at least 2 pulmonary exacerbations requiring IV antibiotics in the past year (sub-study only)
- Agree to maintain current CFTR modulator regimen through the 24-month observation period (sub-study only)
You will not qualify if you...
- Prior gene therapy for any indication (except mRNA-based or antisense oligonucleotide therapies)
- Active Mycobacterium abscessus infection requiring treatment at screening
- Active allergic bronchopulmonary aspergillosis requiring systemic corticosteroids or antifungal therapy
- Smoking or vaping within 2 months prior to screening and unwillingness to abstain during screening and 24-month observation
- Contraindication to systemic corticosteroid therapy
- Chronic use of systemic corticosteroids or immunosuppressants for other conditions
- Hemoglobin A1C �3e�3d 6.5% without diagnosed diabetes, or �3e 7.5% with diagnosed diabetes at screening
- Recent symptomatic hyperglycemia or unstable blood glucose
- Other conditions interfering with corticosteroid management
- Body Mass Index (BMI) less than 16
- Laboratory abnormalities: ALT, AST, or GGT �3e�3d 3 times upper limit of normal; total bilirubin �3e�3d 2 times upper limit; hemoglobin less than 10 g/dL
- Need for continuous or nighttime oxygen supplementation
- Known CF liver disease with multilobular cirrhosis
- History of thrombosis (excluding catheter-related) or increased thrombosis risk conditions
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 20 locations
1
University of Alabama Child Health Research Unit
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
The University of Arizona
Tucson, Arizona, United States, 85724
Actively Recruiting
3
University of California San Francisco
San Francisco, California, United States, 94143
Actively Recruiting
4
National Jewish Health
Denver, Colorado, United States, 80206
Actively Recruiting
5
University of Florida
Gainesville, Florida, United States, 32610
Actively Recruiting
6
University of Miami Hospital
Miami, Florida, United States, 33136
Actively Recruiting
7
Northwestern Memorial Hospital
Chicago, Illinois, United States, 60611
Actively Recruiting
8
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
Actively Recruiting
9
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
Actively Recruiting
10
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Actively Recruiting
11
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
12
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27599
Completed
13
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States, 44146
Actively Recruiting
14
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
15
Penn State Health
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
16
The Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
17
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Actively Recruiting
18
The University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
Actively Recruiting
19
Virginia Commonwealth University Health System
Richmond, Virginia, United States, 23298
Actively Recruiting
20
University of Washington Medical Center
Seattle, Washington, United States, 98195
Actively Recruiting
Research Team
4
4DMT Patient Advocacy
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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