Design and Characterization of 4D-710, an Aerosolized Gene Therapy for Cystic Fibrosis Lung Disease.
Melissa A Calton, Roxanne H Croze, Theodore H Sullivan...
https://pubmed.ncbi.nlm.nih.gov/41124321Actively Recruiting
Phase 2
Age: 18Years +
All Genders
ID05248230
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-710 in Adults With Cystic Fibrosis
Led by 4D Molecular Therapeutics · Updated on 2026-03-11
30
Participants Needed
20
Research Sites
208 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate the safety, tolerability, and early effectiveness of 4D-710, an investigational gene therapy, in adults with cystic fibrosis (CF) lung disease who cannot use or tolerate CFTR modulator therapy. It also includes a sub-study to assess this gene therapy in adults with advanced CF lung disease or frequent lung flare-ups while on current modulator treatments. The study is a Phase 1/2, open-label, multicenter trial focused on this population. Participants receive a single inhaled dose of 4D-710, a gene therapy using an adeno-associated virus to deliver a modified CFTR gene. The trial has different groups: those who cannot use modulator therapy will get varying doses to find the best dose for further study, while those on modulator therapy receive selected doses in the sub-study. The treatment is given once, and doses are explored and expanded to identify recommended levels. During the trial, participants are monitored for up to 60 months to track any side effects and overall safety. The main measure is the number and severity of adverse events. Participants will have lung function tests, oxygen level checks, and other health assessments throughout the observation period. The study will gather detailed safety and tolerability data to guide future research on this gene therapy in CF lung disease.
CONDITIONS
Brief Title
4D-710 in Adult Patients With Cystic Fibrosis
Who Can Participate
Age: 18Years +
All Genders
Eligibility Criteria
You may qualify if you...
- 18 years and older
- Confirmed diagnosis of cystic fibrosis (CF) with CF lung disease, including sweat chloride 6 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 modulator therapy due to side effects
- Forced expiratory volume in 1 second (FEV1) between 50% and 90% of predicted at screening
- Resting oxygen saturation 6 92% on room air at screening
- For sub-study: currently on stable CFTR modulator therapy for at least 60 days and willing to maintain this during 24-month observation
- For sub-study: FEV1 6 40% and < 70% predicted, and/or at least 2 pulmonary exacerbations requiring intravenous antibiotics in the past year
You will not qualify if you...
- Prior gene therapy for any condition (except mRNA-based or antisense oligonucleotide treatments)
- Active Mycobacterium abscessus infection needing treatment at screening
- Active allergic bronchopulmonary aspergillosis requiring systemic corticosteroids or antifungal therapy
- Smoking or vaping in the 2 months before screening; must agree to avoid during screening and 24-month observation
- Contraindications to systemic corticosteroid therapy
- Chronic use of systemic corticosteroids or immunosuppressants for other conditions
- Hemoglobin A1C 6.5% if no diabetes diagnosis; > 7.5% if diagnosed with cystic fibrosis related or type I/II diabetes
- Recent symptomatic hyperglycemia or unstable blood glucose as assessed by investigator
- Other conditions interfering with corticosteroid-related hyperglycemia management
- Body Mass Index (BMI) less than 16
- Laboratory abnormalities: ALT, AST, or GGT 3 times upper normal limit; total bilirubin 2 times upper normal limit; hemoglobin less than 10 g/dL
- Need for continuous or night-time oxygen support
- Known CF liver disease with multilobular cirrhosis
- History of thrombosis (excluding catheter-related) or high thrombosis risk conditions
AI-Screening
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Treatment
Duration - Up to 60 months
Participants receive a single inhalational administration of the gene therapy 4D-710 at various dose levels to evaluate safety and preliminary efficacy.
Multiple visits over the treatment period to monitor safety and response
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
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Research Team
4
4DMT Patient Advocacy
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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