Gene therapy 4D-710 for adults with cystic fibrosis lung disease
Tests a new gene medicine given once to adults with cystic fibrosis lungs.
Plain-language title generated from the registry record
Registry title: 4D-710 in Adult Patients With Cystic Fibrosis
Key facts
- Where
- Baltimore, Birmingham, Boston, Chapel Hill, Charleston, Chicago, Cleveland, Columbus, Dallas, Denver, Gainesville, Hershey, Kansas City, Miami, Philadelphia, Richmond, San Francisco, Seattle, and Tucson, United StatesSee the 20 sites
- Phase
- Phase 2
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with cystic fibrosis and cystic fibrosis lung disease, to test a single dose of the gene therapy 4D-710.
- Age
- 18 years and older
- Sex
- All sexes can join.
- Disease stage
- Confirmed cystic fibrosis with lung disease and sweat chloride of 60 mmol/L or more. Lung function (FEV1) must be 50% to 90% of predicted in the main study, and 40% to under 70% (or at least 2 flare-ups in the last year needing intravenous antibiotics) in the sub-study. Resting oxygen saturation must be 92% or more on room air in the main study.
- Earlier treatment
- Main study: cannot take CFTR modulator therapy, or took it before and stopped because of side effects. Sub-study: on a stable dose of CFTR modulator therapy for at least 60 days before screening. No prior gene therapy of any kind (mRNA or antisense therapies are allowed).
- Biomarker or test result
- Two CFTR gene mutations, or one CFTR mutation together with signs of cystic fibrosis lung disease.
Main reasons for not taking part
- Any earlier gene therapy (except mRNA or antisense therapy)
- Active Mycobacterium abscessus infection needing treatment
- Smoking or vaping in the last 2 months
- Needing long-term steroid or immune-suppressing medicine, or being unable to take steroids
- Needing oxygen all the time or at night, or a body mass index (BMI) below 16
Read by machine on 9 November 2026.
Where in United States
- Johns Hopkins HospitalBaltimore, United StatesTaking people
- University of Alabama Child Health Research UnitBirmingham, United StatesTaking people
- Boston Children's HospitalBoston, United StatesTaking people
- Massachusetts General HospitalBoston, United StatesTaking people
- University of North Carolina at Chapel HillChapel Hill, United StatesNo longer taking part
- Medical University of South CarolinaCharleston, United StatesTaking people
- Northwestern Memorial HospitalChicago, United StatesTaking people
- Rainbow Babies and Children's Hospital / University Hospitals Cleveland Medical CenterCleveland, United StatesTaking people
- Nationwide Children's HospitalColumbus, United StatesTaking people
- The University of Texas Southwestern Medical CenterDallas, United StatesTaking people
- National Jewish HealthDenver, United StatesTaking people
- University of FloridaGainesville, United StatesTaking people
- Penn State HealthHershey, United StatesNo longer taking part
- University of Kansas Medical CenterKansas City, United StatesTaking people
- University of Miami HospitalMiami, United StatesTaking people
- The Hospital of the University of PennsylvaniaPhiladelphia, United StatesTaking people
- Virginia Commonwealth University Health SystemRichmond, United StatesTaking people
- University of California San FranciscoSan Francisco, United StatesTaking people
- University of Washington Medical CenterSeattle, United StatesTaking people
- The University of ArizonaTucson, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- 4D Molecular Therapeutics
- Trial code
- NCT05248230
- Record updated
- 6 July 2026
- Original description
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.Read full original descriptionShow less
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-710 in Adults With Cystic Fibrosis
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
Inclusion: 18 years and older; Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including:; Sweat chloride ≥ 60 mmol/L; Mutation Status; Bi-allelic mutations in the CFTR gene, or; Single mutation in the CFTR gene and clinical manifestations of CF lung disease; Ineligible for CFTR modulator therapy, or previously received modulator therapy but discontinued due to adverse effects; Forced expiratory volume in 1 second (FEV1) ≥ 50% and ≤ 90% of predicted (per Global Lung Function Initiative) at Screening; Resting oxygen saturation ≥ 92% on room air at Screening; 18 years and older; Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including:; Sweat chloride ≥ 60 mmol/L; Mutation Status; Bi-allelic mutations in the CFTR gene, or; Single mutation in the CFTR gene and clinical manifestations of CF lung disease; Currently on a stable dose of CFTR modulator therapy for a minimum of 60 days prior to Screening and agree to maintain current regimen through the 24-month Observation Period; FEV1 ≥ 40% and < 70% predicted (per Global Lung Function Initiative) at Screening, AND/OR experienced at least 2 pulmonary exacerbations in the last year requiring intravenous antibiotics.
Exclusion: Any prior gene therapy for any indication (Exception: mRNA-based or antisense oligonucleotide therapies are not exclusionary); Smoking (e.g. cigarettes, cigars, cannabis) or use of a vaping product during the 2 months prior to screening; must agree to not smoke or use vaping products during Screening and initial 24-month observation period; Contraindication to systemic corticosteroid therapy; Active Mycobacterium abscessus infection requiring ongoing treatment at Screening; Active allergic bronchopulmonary aspergillosis requiring management with systemic corticosteroids or antifungal therapy; Requires chronic use of systemic corticosteroids or immunosuppressants to treat another condition; Other conditions that, in the Investigator's opinion, may interfere with management of corticosteroid-related hyperglycemia; Known CF liver disease with evidence of multilobular cirrhosis; History of thrombosis (excluding catheter-related thrombosis) or conditions associated with increased risk of thrombosis; If no known diagnosis of cystic fibrosis related diabetes (CFRD), Type I, or Type II diabetes: Hemoglobin A1C ≥ 6.5% at Screening; If known diagnosis of CFRD, Type I or Type II diabetes: Hemoglobin A1C > 7.5% at Screening; Recent history of symptomatic hyperglycemia or unstable blood glucose levels as per Investigator's assessment; Laboratory abnormalities at screening:; ALT, AST or GGT ≥ 3 × the upper limit of normal (ULN); Total bilirubin ≥ 2 × ULN; Hemoglobin < 10 g/dL; Body Mass Index (BMI) < 16; Requirement for continuous or night-time oxygen supplementation.