4D-710 in Adult Patients With Cystic Fibrosis
CF
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-710 in Adults With Cystic Fibrosis
1 other identifier
interventional
30
1 country
20
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Mar 2022
Longer than P75 for phase_2
20 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 10, 2022
CompletedFirst Posted
Study publicly available on registry
February 21, 2022
CompletedStudy Start
First participant enrolled
March 29, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 1, 2031
March 11, 2026
March 1, 2026
5.2 years
February 10, 2022
March 9, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Incidence and severity of adverse events
Safety and tolerability of 4D-710 following a single dose via inhalation, as assessed by incidence and severity of treatment emergent adverse events, serious adverse events, and dose limiting toxicities, including clinically significant changes from baseline to scheduled time points in safety parameters.
60 Months
Study Arms (3)
4D-710 Phase 1: Dose Exploration
EXPERIMENTALParticipants who are ineligible for or intolerant of modulator therapy will receive one of various dose levels of 4D-710 to identify recommended phase 2 dose(s) for further evaluation.
4D-710 Phase 2: Dose Expansion
EXPERIMENTALParticipants will receive a single inhalational administration of 4D-710 at the dose level(s) selected for dose expansion.
4D-710 Dose Exploration (Sub-Study)
EXPERIMENTALParticipants who are on currently available CFTR modulator therapy will receive a dose of 4D-710 at various dose levels.
Interventions
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).
Eligibility Criteria
You may qualify if:
- 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
- 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
- +2 more criteria
You may not qualify if:
- 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
- +2 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (20)
University of Alabama Child Health Research Unit
Birmingham, Alabama, 35233, United States
The University of Arizona
Tucson, Arizona, 85724, United States
University of California San Francisco
San Francisco, California, 94143, United States
National Jewish Health
Denver, Colorado, 80206, United States
University of Florida
Gainesville, Florida, 32610, United States
University of Miami Hospital
Miami, Florida, 33136, United States
Northwestern Memorial Hospital
Chicago, Illinois, 60611, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
Johns Hopkins Hospital
Baltimore, Maryland, 21287, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599, United States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44146, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Penn State Health
Hershey, Pennsylvania, 17033, United States
The Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
The University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
Virginia Commonwealth University Health System
Richmond, Virginia, 23298, United States
University of Washington Medical Center
Seattle, Washington, 98195, United States
Related Publications (1)
Calton MA, Croze RH, Sullivan TH, Collins SA, Tucker S, Whittlesey KJ, Kim DH, Nye JA, Beliakoff G, Quezada M, Burns C, Schmitt C, Klein A, Jia V, Kovacs L, Lauko D, Yoh K, Nguyen K, Barglow K, Gonzales J, Khoday D, Mason T, Delaria K, Bashour K, Kotterman M, Schaffer D, Song A, Francis P, Taylor-Cousar JL, Kirn D. Design and Characterization of 4D-710, an Aerosolized Gene Therapy for Cystic Fibrosis Lung Disease. Am J Respir Cell Mol Biol. 2025 Oct 22. doi: 10.1165/rcmb.2025-0243MA. Online ahead of print.
PMID: 41124321DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Sam Moskowitz MD
4D Molecular Therapeutics
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 10, 2022
First Posted
February 21, 2022
Study Start
March 29, 2022
Primary Completion (Estimated)
June 1, 2027
Study Completion (Estimated)
June 1, 2031
Last Updated
March 11, 2026
Record last verified: 2026-03