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New treatment option for Cystic Fibrosis Lung
Official title 4D-710 in Adult Patients With Cystic Fibrosis
ClinicalTrials.gov ID: NCT05248230
What this study is testing
What is 4D-710?
4D-710 is an investigational medicine, being studied as a potential treatment for cystic fibrosis lung.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Key (Primary Study):
- 18 years and older
- Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including:
- Sweat chloride ≥ 60 mmol/L
- Mutation Status
You likely can't join if
- (Primary and Sub Study):
- Any prior gene therapy for any indication (Exception: mRNA-based or antisense oligonucleotide therapies are not exclusionary)
- Active Mycobacterium abscessus infection requiring ongoing treatment at Screening
- Active allergic bronchopulmonary aspergillosis requiring management with systemic corticosteroids or antifungal therapy
- 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...
- Contraindication to systemic corticosteroid therapy
See the full eligibility criteria
- Key (Primary Study):
- 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 Key (Sub-Study):
- 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 Key
- (Primary and Sub Study):
- Any prior gene therapy for any indication (Exception: mRNA-based or antisense oligonucleotide therapies are not exclusionary)
- Active Mycobacterium abscessus infection requiring ongoing treatment at Screening
- Active allergic bronchopulmonary aspergillosis requiring management with systemic corticosteroids or antifungal therapy
- 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
- Requires chronic use of systemic corticosteroids or immunosuppressants to treat another condition
- 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
- Other conditions that, in the Investigator's opinion, may interfere with management of corticosteroid-related hyperglycemia
- Body Mass Index (BMI) \< 16
- Laboratory abnormalities at screening:
- ALT, AST or GGT ≥ 3 × the upper limit of normal (ULN)
- Total bilirubin ≥ 2 × ULN
- Hemoglobin \< 10 g/dL
- Requirement for continuous or night-time oxygen supplementation
- Known CF liver disease with evidence of multilobular cirrhosis
- History of thrombosis (excluding catheter-related thrombosis) or conditions associated with increased risk of thrombosis
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Tucson, Arizona, United States
- San Francisco, California, United States
- Denver, Colorado, United States
- Gainesville, Florida, United States
- Miami, Florida, United States
- Chicago, Illinois, United States
- Kansas City, Kansas, United States
- Baltimore, Maryland, United States
- Boston, Massachusetts, United States
- Chapel Hill, North Carolina, United States
- Cleveland, Ohio, United States
- Columbus, Ohio, United States
- Hershey, Pennsylvania, United States
- Philadelphia, Pennsylvania, United States
- Charleston, South Carolina, United States
- Dallas, Texas, United States
- Richmond, Virginia, United States
- Seattle, Washington, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Tucson, Arizona, United States; San Francisco, California, United States; Denver, Colorado, United States; Gainesville, Florida, United States; Miami, Florida, United States and 13 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.