4D-710 in Adult Patients With Cystic Fibrosis
ConditionCystic Fibrosis Lung
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
Sponsor4D Molecular Therapeutics
About this trial
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
Eligibility criteria
Qualifiers
18 years and older
Sweat chloride ≥ 60 mmol/L
Mutation Status
Bi-allelic mutations in the CFTR gene, or
Disqualifiers
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.
Trial design
Treatments tested in this trial
- 4D-710
Treatment groups
30 Participants
are divided into 3 treatment groupsLocations
20Alabama
University of Alabama Child Health Research Unit35233, Birmingham United States
Arizona
The University of Arizona85724, Tucson United States
California
University of California San Francisco94143, San Francisco United States
Colorado
National Jewish Health80206, Denver United States
Sponsors and collaborators
4D Molecular Therapeutics
Lead sponsor
Source ClinicalTrials.gov