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Cystic Fibrosis LungPhase 2CFNCT05248230

4D-710 in Adult Patients With Cystic Fibrosis

A Phase 2 study testing 4D-710 in people with Cystic Fibrosis Lung, run by 4D Molecular Therapeutics at 20 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Cystic Fibrosis Lung
What is being tested?
4D-710
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
20 sitesUnited States
Study dates
29 Mar 2022to Jun 2031
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 18 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
Show all 17Show fewer
  • 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
  • 18 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
  • Currently on a stable dose of CFTR modulator therapy for a minimum of 60 days prior to Screening and agree to maintain current regimen through the 24-month Observation Period
  • 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

Who cannot join

  • 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.
  • Contraindication to systemic corticosteroid therapy
  • Requires chronic use of systemic corticosteroids or immunosuppressants to treat another condition
Show all 18Show fewer
  • 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
  • Known CF liver disease with evidence of multilobular cirrhosis
  • History of thrombosis (excluding catheter-related thrombosis) or conditions associated with increased risk of thrombosis

Trial locations and contacts

20 sites in 1 country. Contact the study team or a site near you to ask about taking part.

4DMT Patient Advocacy contact

(888) 748-8881

clinicaltrials@4DMT.com

20 sites in 1 country

United States20 sites, 18 recruiting

  • Johns Hopkins Hospital

    Baltimore, Maryland, 21287

    Kaia Houtman, khoutma1@jh.edu

    Noah Lechtzin, MD

    Recruiting

  • University of Alabama Child Health Research Unit

    Birmingham, Alabama, 35233

    Justin Anderson, justinanderson@uabmc.edu

    George M. Solomon, MD

    Recruiting

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

    Margot Hardcastle, mhardcastle@mgh.harvard.edu

    Allen Lapey, MD

    Recruiting

  • Boston Children's Hospital

    Boston, Massachusetts, 02115

    Monica Ulles, monica.ulles@childrens.harvard.edu

    Alicia Casey, MD

    Recruiting

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27599

    Completed

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

    Cameron Mathison, mathisoc@musc.edu

    Patrick Flume, MD

    Recruiting

  • Northwestern Memorial Hospital

    Chicago, Illinois, 60611

    Rachel Nelson, rachel.nelson@northwestern.edu

    Manu Jain, MD

    Recruiting

  • Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44146

    Tia Rone, Tia.Rone@uhhospitals.org

    Alex Gifford, MD

    Recruiting

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205

    Terri Johnson, terri.johnson@nationwidechildrens.org

    Karen S. McCoy, MD

    Recruiting

  • The University of Texas Southwestern Medical Center

    Dallas, Texas, 75390

    Maria McLeod, maria.mcleod@utsouthwestern.edu

    Raksha Jain, MD

    Recruiting

  • National Jewish Health

    Denver, Colorado, 80206

    Jennifer Taylor-Cousar, MD, TaylorCousarJ@NJHealth.org

    Michele Gaffigan, gaffiganm@njhealth.org

    Recruiting

  • University of Florida

    Gainesville, Florida, 32610

    Chrystal Bailey, cbailey1@peds.ufl.edu

    Cesar Trillo-Alvarez, MD

    Recruiting

  • Penn State Health

    Hershey, Pennsylvania, 17033

    Withdrawn

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160

    Lawrence Scott, lscott2@kumc.edu

    Joel Mermis, MD

    Recruiting

  • University of Miami Hospital

    Miami, Florida, 33136

    Ivan Whitaker, 305-799-9209, yiw2@miami.edu

    Maria G Tupayachi Ortiz, MD

    Recruiting

  • The Hospital of the University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

    Melissa Molter, melissa.molter@pennmedicine.upenn.edu

    Daniel Dorgan, MD

    Recruiting

  • Virginia Commonwealth University Health System

    Richmond, Virginia, 23298

    Margaret K. Lessard, margaret.lessard@vcuhealth.org

    Nauman A. Chaudary, MD

    Recruiting

  • University of California San Francisco

    San Francisco, California, 94143

    Legna Betancourt, legna.betancourt@ucsf.edu

    Jonathan Singer, MD

    Recruiting

  • University of Washington Medical Center

    Seattle, Washington, 98195

    Roshni Prabhu, rprabhu@medicine.washington.edu

    Christopher Goss, MD

    Recruiting

  • The University of Arizona

    Tucson, Arizona, 85724

    Elizabeth Ryan, elizabethryan@arizona.edu

    Cori L Daines, MD

    Recruiting

Other trials in United States

About this study

Official title: An Open-label, Phase 1/2 Trial of Gene Therapy 4D-710 in Adults With Cystic Fibrosis

This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.

Read the detailed description

This Phase 1/2 trial will evaluate the safety, tolerability, and preliminary efficacy of 4D-710, an investigational gene therapy, in adults with cystic fibrosis (CF) lung disease who are ineligible or unable to tolerate CFTR modulator therapy. A sub-study will evaluate 4D-710 in a cohort of adults with CF advanced lung disease and/or frequent pulmonary exacerbation (PE) while on currently available CFTR modulator therapy.

Being tested
4D-710
Keywords
CF, Cystic Fibrosis, Gene Therapy
Medical subject headings
Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • 4D-710Biological

    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).

Study groups in NCT05248230
Study groupRoleWhat they receive
4D-710 Phase 1: Dose ExplorationExperimentalParticipants 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.
Receives: Biological: 4D-710
4D-710 Phase 2: Dose ExpansionExperimentalParticipants will receive a single inhalational administration of 4D-710 at the dose level(s) selected for dose expansion.
Receives: Biological: 4D-710
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.
Receives: Biological: 4D-710

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: 60 Months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Feb 2022
  2. Study start29 Mar 2022
  3. Primary completionJun 2027 (estimated)
  4. Study completionJun 2031 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05248230
Sponsor's protocol ID
4D-710-C001
Acronym
CF
Status last verified
Jul 2026
First submitted
10 Feb 2022
Last updated
6 Jul 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • 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. 2026 Jul 1;74(7):837-846. doi: 10.1165/rcmb.2025-0243MA.

    PubMed 41124321

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing 4D-710 in people with Cystic Fibrosis Lung, run by 4D Molecular Therapeutics at 20 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 17 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 6 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 20 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact 4DMT Patient Advocacy ((888) 748-8881, clinicaltrials@4DMT.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence and severity of adverse events (time frame: 60 Months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05248230, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jul 2026.

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