Actively Recruiting
An Open-label, Phase 12 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 12, 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
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
Research Team
4
4DMT Patient Advocacy
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