Investigational gene therapy for adults with cystic fibrosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 1/2 trial evaluates the safety, tolerability, and preliminary efficacy of 4D-710, an investigational gene therapy, in adults with cystic fibrosis who have advanced lung disease and are ineligible or unable to tolerate CFTR modulator therapy. The study includes a sub-study for those currently on CFTR modulator therapy experiencing frequent pulmonary exacerbations. Participants will receive a single dose of 4D-710, and the trial will assess various health outcomes related to cystic fibrosis lung disease.

Why it matters

Who can join: the full rules

Key Inclusion Criteria (Primary Study):

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 ≤ 90% of predicted (per Global Lung Function Initiative) at Screening
4. Resting oxygen saturation ≥ 92% on room air at Screening

Key Inclusion Criteria (Sub-Study):

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 for a minimum of 60 days prior to Screening and agree to maintain current regimen through the 24-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

Key Exclusion Criteria (Primary and Sub Study):

1. Any prior gene therapy for any indication (Exception: mRNA-based or antisense oligonucleotide therapies are not exclusionary)
2. Active Mycobacterium abscessus infection requiring ongoing treatment at Screening
3. Active allergic bronchopulmonary aspergillosis requiring management with systemic corticosteroids or antifungal therapy
4. 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.
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 multilobular cirrhosis
15. History of thrombosis (excluding catheter-related thrombosis) or conditions associated with increased risk of thrombosis

Where it's running

• University of Alabama Child Health Research Unit
  Birmingham, Alabama, United States Recruiting
• The University of Arizona
  Tucson, Arizona, United States Recruiting
• University of California San Francisco
  San Francisco, California, United States Recruiting
• National Jewish Health
  Denver, Colorado, United States Recruiting
• University of Florida
  Gainesville, Florida, United States Recruiting
• University of Miami Hospital
  Miami, Florida, United States Recruiting

Study contacts

• Study coordinator: 4DMT Patient Advocacy
• Email: clinicaltrials@4DMT.com
• Phone: (888) 748-8881

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Observing the progression of cystic fibrosis lung disease in young patients

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• Inhaled CFTR mRNA for cystic fibrosis in Chinese patients aged 12 and older

  This trial tests inhaled BMD003, a CFTR mRNA therapy, to see if it's safe and shows early signs of benefit in Chinese people 12 years and up with cystic fibrosis.

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• VX-272 dose escalation in healthy adults

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• Prevalence of smoking, passive smoke exposure, and vaping among adults with cystic fibrosis

  This project will see how common smoking, exposure to secondhand smoke, and vaping are among adults with cystic fibrosis followed at the Strasbourg Adult CF Center.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.