Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
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|
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.
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.
This trial id was obtained from ClinicalTrials.gov, 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|
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
|4D Molecular Therapeutics|
The person who is responsible for the scientific and technical direction of the entire clinical study.
|Mitra Tavakkoli, MD, PharmD|
|Principal Investigator Affiliation||4D Molecular Therapeutics|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
The disease, disorder, syndrome, illness, or injury that is being studied.
|Cystic Fibrosis Lung|
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.
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.