Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
40 participants
INTERVENTIONAL
2022-03-29
2030-01-31
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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4D-710 Phase 1: Dose Exploration
Participants 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
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).
4D-710 Phase 2: Dose Expansion
Participants will receive a single inhalational administration of 4D-710 at the dose level(s) selected for dose expansion.
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).
4D-710 Dose Exploration (Sub-Study)
Participants who are on currently available CFTR modulator therapy will receive a dose of 4D-710 at various dose levels.
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).
Interventions
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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).
Eligibility Criteria
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Inclusion Criteria
2. Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including:
1. Sweat chloride ≥ 60 mmol/L
2. Mutation Status
* Bi-allelic mutations in the CFTR gene, or
* Single mutation in the CFTR gene and clinical manifestations of CF lung disease
3. Ineligible for CFTR modulator therapy, or previously received modulator therapy but discontinued due to adverse effects.
3. Forced expiratory volume in 1 second (FEV1) ≥ 50% and ≤ 90% of predicted (per Global Lung Function Initiative) at Screening
4. Resting oxygen saturation ≥ 92% on room air at Screening
1. 18 years and older
2. Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including:
1. Sweat chloride ≥ 60 mmol/L
2. Mutation Status
* Bi-allelic mutations in the CFTR gene, or
* Single mutation in the CFTR gene and clinical manifestations of CF lung disease
3. 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
4. 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
Exclusion Criteria
2. Active Mycobacterium abscessus infection requiring ongoing treatment at Screening
3. Active allergic bronchopulmonary aspergillosis requiring management with systemic corticosteroids or antifungal therapy
4. Contraindication to systemic corticosteroid therapy
5. Requires chronic use of systemic corticosteroids or immunosuppressants to treat another condition
6. If no known diagnosis of cystic fibrosis related diabetes (CFRD), Type I, or Type II diabetes: Hemoglobin A1C ≥ 6.5% at Screening
7. If known diagnosis of CFRD, Type I or Type II diabetes: Hemoglobin A1C \> 7.5% at Screening
8. Recent history of symptomatic hyperglycemia or unstable blood glucose levels as per Investigator's assessment
9. Other conditions that, in the Investigator's opinion, may interfere with management of corticosteroid-related hyperglycemia
10. Body Mass Index (BMI) \< 16
11. Laboratory abnormalities at screening:
* ALT, AST or GGT ≥ 3 × the upper limit of normal (ULN)
* Total bilirubin ≥ 2 × ULN
* Hemoglobin \< 10 g/dL
12. Requirement for continuous or night-time oxygen supplementation
13. Known CF liver disease with evidence of multilobular cirrhosis
14. History of thrombosis (excluding catheter-related thrombosis) or conditions associated with increased risk of thrombosis
18 Years
ALL
No
Sponsors
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4D Molecular Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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Alan H. Cohen, MD
Role: STUDY_DIRECTOR
4D Molecular Therapeutics
Locations
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University of Alabama Child Health Research Unit
Birmingham, Alabama, United States
The University of Arizona
Tucson, Arizona, United States
University of California San Francisco
San Francisco, California, United States
National Jewish Health
Denver, Colorado, United States
University of Florida
Gainesville, Florida, United States
University of Miami Hospital
Miami, Florida, United States
Northwestern Memorial Hospital
Chicago, Illinois, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Johns Hopkins Hospital
Baltimore, Maryland, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Penn State Health
Hershey, Pennsylvania, United States
The Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States
The University of Texas Southwestern Medical Center
Dallas, Texas, United States
Virginia Commonwealth University Health System
Richmond, Virginia, United States
University of Washington Medical Center
Seattle, Washington, United States
Countries
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Central Contacts
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Facility Contacts
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Justin Anderson
Role: primary
Elizabeth Ryan
Role: primary
Legna Betancourt
Role: primary
Jennifer Taylor-Cousar, MD
Role: primary
Michele Gaffigan
Role: backup
Chrystal Bailey
Role: primary
Ivan Whitaker
Role: primary
Rachel Nelson
Role: primary
Lawrence Scott
Role: primary
Kaia Houtman
Role: primary
Margot Hardcastle
Role: primary
Monica Ulles
Role: primary
Tia Rone
Role: primary
Terri Johnson
Role: primary
Diane Kitch
Role: primary
Melissa Molter
Role: primary
Maria McLeod
Role: primary
Margaret K. Lessard
Role: primary
Roshni Prabhu
Role: primary
References
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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.
Other Identifiers
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4D-710-C001
Identifier Type: -
Identifier Source: org_study_id