NCT05248230 · 4D Molecular Therapeutics
4D-710 in Adult Patients With Cystic Fibrosis
(CF)
What this study is about
This is a Phase 1/2 conducted at multiple hospitals, where both patients and doctors know the treatment given, single dose trial of 4D-710 experimental gene therapy in adults with cystic fibrosis.
View original scientific description
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
Interventions
BIOLOGICAL
4D-710
4D-710 is an adeno-associated virus (AAV) gene therapy comprised of an AAV capsid variant (4D-A101) carrying a transgene cassette encoding human cystic fibrosis transmembrane conductance regulator with a deletion in the regulatory domain (CFTRΔR).
Primary outcome measures
Incidence and severity of adverse events
Time frame: 60 Months
Safety and tolerability of 4D-710 following a single dose via inhalation, as assessed by incidence and severity of treatment emergent adverse events, serious adverse events, and dose limiting toxicities, including clinically significant changes from baseline to scheduled time points in safety parameters.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- (Primary Study): 1. 18 years and older 2. Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including: 1. Sweat chloride ≥ 60 mmol/L 2. Mutation Status
- Bi-allelic mutations in the CFTR gene, or
- Single mutation in the CFTR gene and clinical manifestations of CF lung disease 3. Ineligible for CFTR modulator therapy, or previously received modulator therapy but discontinued due to adverse effects. 3. Forced expiratory volume in 1 second (FEV1) ≥ 50% and ≤ 90% of predicted (per Global Lung Function Initiative) at Screening 4. Resting oxygen saturation ≥ 92% on room air at Screening Key Inclusion Criteria (Sub-Study): 1. 18 years and older 2. Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including: 1. Sweat chloride ≥ 60 mmol/L 2. Mutation Status
- Bi-allelic mutations in the CFTR gene, or
- Single mutation in the CFTR gene and clinical manifestations of CF lung disease 3. Currently on a
Where
- Birmingham, Alabama
- Tucson, Arizona
- San Francisco, California
- Denver, Colorado
- Gainesville, Florida
- Miami, Florida
- Chicago, Illinois
- Kansas City, Kansas
- Baltimore, Maryland
- Boston, Massachusetts
- Chapel Hill, North Carolina
- Cleveland, Ohio
And 7 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 6, 2026 · Source of record for eligibility and locations