Status:
RECRUITING
4D-710 in Adult Patients With Cystic Fibrosis
Lead Sponsor:
4D Molecular Therapeutics
Conditions:
Cystic Fibrosis Lung
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
Detailed Description
This Phase 1/2 trial will evaluate the safety, tolerability, and preliminary efficacy of 4D-710, an investigational gene therapy, in adults with cystic fibrosis (CF) advanced lung disease who are inel...
Eligibility Criteria
Inclusion
- Key Inclusion Criteria (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 Inclusion Criteria (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 (elexacaftor/tezacaftor/ivacaftor) for a minimum of 60 days prior to Screening and agree to maintain current regimen through the 12-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 Exclusion Criteria (Primary and Sub Study):
- Any prior gene therapy for any indication (Exception: mRNA-based 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
- 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
Exclusion
Key Trial Info
Start Date :
March 29 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
January 1 2030
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT05248230
Start Date
March 29 2022
End Date
January 1 2030
Last Update
December 11 2025
Active Locations (19)
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1
University of Alabama Child Health Research Unit
Birmingham, Alabama, United States, 35233
2
The University of Arizona
Tucson, Arizona, United States, 85724
3
University of California San Francisco
San Francisco, California, United States, 94143
4
National Jewish Health
Denver, Colorado, United States, 80206