4D-710 in Adult Patients With Cystic Fibrosis
NCT05248230 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 30
Last updated 2026-03-11
Summary
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
Conditions
- Cystic Fibrosis Lung
Interventions
- BIOLOGICAL
-
4D-710
4D-710 is an adeno-associated virus (AAV) gene therapy comprised of an AAV capsid variant (4D-A101) carrying a transgene cassette encoding human cystic fibrosis transmembrane conductance regulator with a deletion in the regulatory domain (CFTRΔR).
Sponsors & Collaborators
-
4D Molecular Therapeutics
lead INDUSTRY
Principal Investigators
-
Sam Moskowitz MD · 4D Molecular Therapeutics
Study Design
- Allocation
- NON_RANDOMIZED
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SEQUENTIAL
Eligibility
- Min Age
- 18 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2022-03-29
- Primary Completion
- 2027-06-30
- Completion
- 2031-06-30
- FDA Drug
- Yes
Countries
- United States
Study Locations
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