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Trial record 1 of 2 for:    4D | cystic fibrosis
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4D-710 in Adult Patients With Cystic Fibrosis (CF)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05248230
Recruitment Status : Recruiting
First Posted : February 21, 2022
Last Update Posted : February 6, 2023
Sponsor:
Information provided by (Responsible Party):
4D Molecular Therapeutics

Study Description
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Brief Summary:
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with CF who are ineligible for or unable to tolerate CFTR modulator therapy.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Lung Biological: 4D-710 Phase 1 Phase 2

Detailed Description:
This Phase 1/2 trial will evaluate the safety, tolerability, and preliminary efficacy of 2 dose levels of 4D-710, an investigational gene therapy, in adults with cystic fibrosis lung disease who are ineligible or unable to tolerate CFTR modulator therapy.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 21 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Phase 1/2 Trial of Gene Therapy 4D-710 in Adults With Cystic Fibrosis
Actual Study Start Date : March 29, 2022
Estimated Primary Completion Date : August 2024
Estimated Study Completion Date : August 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: 4D-710 Dose Exploration Cohort 1
Single inhalational administration of 4D-710 Dose Level 1
 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).
Experimental: 4D-710 Dose Exploration Cohort 2
Single inhalational administration of 4D-710 Dose Level 2
 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).
Experimental: 4D-710 Dose Expansion Cohort
Single inhalational administration of 4D-710 at the selected dose
 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).


Outcome Measures
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Primary Outcome Measures :
  1. Incidence and severity of adverse events [ Time Frame: 24 Months ]
    Safety and tolerability of 4D-710 following a single inhalation dose, as assessed by incidence and severity of adverse events, serious adverse events and dose limiting toxicities, including clinically significant changes from baseline to scheduled time points in safety parameters.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  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. 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 ≤100% of predicted (per Global Lung Function Initiative) at Screening
  4. Resting oxygen saturation ≥ 92% on room air at Screening

Key Exclusion Criteria:

  1. Any prior gene therapy for any indication
  2. Active Mycobacterium abscessus infection requiring ongoing treatment at Screening
  3. Active allergic bronchopulmonary aspergillosis requiring management with systemic corticosteroids or antifungal therapy
  4. Two or more pulmonary exacerbations requiring treatment with intravenous (IV) antibiotics within 6 months prior to Screening
  5. Contraindication to systemic corticosteroid therapy
  6. Requires chronic use of systemic corticosteroids or immunosuppressants to treat another condition
  7. If no known diagnosis of cystic fibrosis related diabetes (CFRD), Type I, or Type II diabetes: Hemoglobin A1C ≥6.5% at Screening
  8. If known diagnosis of CFRD, Type I or Type II diabetes: Hemoglobin A1C >7.5% at Screening
  9. Recent history of symptomatic hyperglycemia or unstable blood glucose levels as per Investigator's assessment
  10. Other conditions that, in the Investigator's opinion, may interfere with management of corticosteroid-related hyperglycemia
  11. Body Mass Index (BMI) <16

  12. Laboratory abnormalities at screening:

    • ALT, AST or GGT ≥ 3 × the upper limit of normal (ULN)
    • Total bilirubin ≥ 2 × ULN
    • Hemoglobin < 10 g/dL
  13. Requirement for continuous or night-time oxygen supplementation
  14. Known CF liver disease with evidence of cirrhosis
  15. History of thrombosis (excluding catheter-related thrombosis) or conditions associated with increased risk of thrombosis
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05248230


Contacts
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Contact: 4DMT Patient Advocacy (888) 748-8881 clinicaltrials@4DMT.com

Locations
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United States, Alabama
University of Alabama Child Health Research Unit Recruiting
Birmingham, Alabama, United States, 35233
Contact: Heather Hathorne       hyhathorne@uabmc.edu   
Contact: Kathryn Monroe       kathrynmonroe@uabmc.edu   
Principal Investigator: George M. Solomon, MD         
United States, Colorado
National Jewish Health Recruiting
Denver, Colorado, United States, 80206
Contact: Jennifer Taylor-Cousar, MD       TaylorCousarJ@NJHealth.org   
Contact: Alexandra Wilson       wilsona@njhealth.org   
Principal Investigator: Jennifer Taylor-Cousar, MD         
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Lawrence Scott       lscott2@kumc.edu   
Principal Investigator: Joel Mermis, MD         
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Margot Hardcastle       mhardcastle@mgh.harvard.edu   
Principal Investigator: Allen Lapey, MD         
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Monica Ulles       monica.ulles@childrens.harvard.edu   
Principal Investigator: Henry Dorkin, MD         
United States, North Carolina
University of North Carolina at Chapel Hill Recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: Alexandria Nesbit       alexandria_nesbit@med.unc.edu   
Contact: Margret Powell       margret_powell@med.unc.edu   
Principal Investigator: Scott Donaldson, MD         
United States, Ohio
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center Recruiting
Cleveland, Ohio, United States, 44146
Contact: Laura Batke       Laura.Batke@UHhospitals.org   
Contact: Kathleen Hilliard       kathleen.hilliard@uhhospitals.org   
Principal Investigator: Alex Gifford, MD         
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
Contact: Terri Johnson       terri.johnson@nationwidechildrens.org   
Principal Investigator: Karen S. McCoy, MD         
United States, Pennsylvania
Penn State Health Recruiting
Hershey, Pennsylvania, United States, 17033
Contact: Diane Kitch       dkitch@pennstatehealth.psu.edu   
Principal Investigator: Judie A. Howrylak, MD, PhD         
The Hospital of the University of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Melissa Molter       melissa.molter@pennmedicine.upenn.edu   
Principal Investigator: Daniel Dorgan, MD         
United States, Texas
The University of Texas Southwestern Medical Center Recruiting
Dallas, Texas, United States, 75390
Contact: Ashley Keller       ashley.keller@utsouthwestern.edu   
Principal Investigator: Raksha Jain, MD         
United States, Virginia
Virginia Commonwealth University Health System Recruiting
Richmond, Virginia, United States, 23298
Contact: Margaret K. Lessard       margaret.lessard@vcuhealth.org   
Principal Investigator: Nauman A. Chaudary, MD         
Sponsors and Collaborators
4D Molecular Therapeutics
Investigators
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Study Director: Susan Limb, MD 4D Molecular Therapeutics
More Information
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Responsible Party: 4D Molecular Therapeutics
ClinicalTrials.gov Identifier: NCT05248230    
Other Study ID Numbers: 4D-710-C001
First Posted: February 21, 2022    Key Record Dates
Last Update Posted: February 6, 2023
Last Verified: February 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by 4D Molecular Therapeutics:
CF
Cystic Fibrosis
Gene Therapy
Additional relevant MeSH terms:
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Cystic Fibrosis
Pulmonary Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
 Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Lung Diseases, Interstitial