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4D-710 Gene Therapy for Adults with Cystic Fibrosis

This study is testing an investigational gene therapy called 4D-710 for adults with cystic fibrosis (CF) lung disease. 4D-710 works by delivering a corrected version of the CFTR gene, which is faulty in people with CF, to the lungs. You may be able to join if you are 18 years or older, have a confirmed diagnosis of CF with lung disease, and meet specific genetic criteria (bi-allelic mutations in the CFTR gene, or a single mutation with CF lung disease). The main goal of this study is to see how safe 4D-710 is and what side effects it might cause over 60 months. This study is currently recruiting about 30 participants, but its overall status is unclear.

Study design
This is a Phase 1/2, open-label study, meaning both you and the study team will know you are receiving 4D-710. It is designed to test a single dose of the gene therapy in adults with cystic fibrosis.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Your safety will be monitored for 60 months after receiving the treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05248230

4D-710 in Adult Patients With Cystic Fibrosis

Recruiting
PHASE2Ages 18+InterventionalTreatment
4D Molecular Therapeutics
~30 participants
Updated 2026-07-06 on ClinicalTrials.gov
What's tested:4D-710

At a glance

Recruiting sites
18 of 20 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence and severity of adverse events
Measured over 60 Months

Conditions

Cystic Fibrosis Lung

Explore related trials

Cystic Fibrosis

Where it's being run

20 sites across 16 states
Florida2
Massachusetts2
Ohio2
Pennsylvania2
Alabama1
Arizona1
California1
Colorado1

Study leadership

  • Sam Moskowitz MD · STUDY_DIRECTOR · 4D Molecular Therapeutics

Who to contact

4DMT Patient Advocacy
[email protected]
(888) 748-8881
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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
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 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 4. 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

ALT, AST or GGT ≥ 3 × the upper limit of normal (ULN)
Total bilirubin ≥ 2 × ULN
Hemoglobin \< 10 g/dL 13. Requirement for continuous or night-time oxygen supplementation 14. Known CF liver disease with evidence of multilobular cirrhosis 15. History of thrombosis (excluding catheter-related thrombosis) or conditions associated with increased risk of thrombosis

What this trial measures

  • Incidence and severity of adverse events60 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.