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4D-710 in Adult Patients With Cystic Fibrosis

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.

An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.

Searching Both is inclusive of interventional and observational studies.

Eligible Ages 18 Years and Over
Gender All
More Inclusion & Exclusion Criteria


Inclusion Criteria:

1. 18 years and older. 2. Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including:
  • - Bi-allelic mutations in the CFTR gene, and.
  • - 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. History of positive culture for Burkholderia cenocepacia, B. dolosa, or Mycobacterium abscessus within the past 24 months. 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 cirrhosis. 14. History of thrombosis (excluding catheter-related thrombosis) or conditions associated with increased risk of thrombosis

Trial Details

Trial ID:

This trial id was obtained from, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.


Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 1/Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

4D Molecular Therapeutics
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Susan Limb, MD
Principal Investigator Affiliation 4D Molecular Therapeutics
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Overall Status Recruiting
Countries United States

The disease, disorder, syndrome, illness, or injury that is being studied.

Cystic Fibrosis Lung
Additional Details

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.

Arms & Interventions


Experimental: 4D-710 Dose Exploration Cohort 1

Single inhalational administration of 4D-710 Dose Level 1

Experimental: 4D-710 Dose Exploration Cohort 2

Single inhalational administration of 4D-710 Dose Level 2

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).

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

National Jewish Health, Denver, Colorado




National Jewish Health

Denver, Colorado, 80206

Site Contact

Jennifer Taylor-Cousar, MD

(888) 748-8881

University of Kansas Medical Center, Kansas City, Kansas




University of Kansas Medical Center

Kansas City, Kansas, 66160

Site Contact

Lawrence Scott

(888) 748-8881

Boston Children's Hospital, Boston, Massachusetts




Boston Children's Hospital

Boston, Massachusetts, 02115

Cleveland, Ohio




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

Cleveland, Ohio, 44146

Site Contact

Laura Batke

(888) 748-8881

Penn State Health, Hershey, Pennsylvania




Penn State Health

Hershey, Pennsylvania, 17033

Philadelphia, Pennsylvania




The Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104

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