Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at UCSF
Dates
study started
completion around

Description

Summary

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

Official Title

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

Details

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) advanced 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.

Keywords

Cystic Fibrosis Lung, CF, Cystic Fibrosis, Gene Therapy, Pulmonary Fibrosis, Fibrosis

Eligibility

You can join if…

Open to people ages 18 years and up

(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 (elexacaftor/tezacaftor/ivacaftor) for a minimum of 60 days prior to Screening and agree to maintain current regimen through the 12-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

You CAN'T join if...

(Primary and Sub Study):

  1. Any prior gene therapy for any indication (Exception: mRNA-based 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. 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 multilobular cirrhosis
  14. History of thrombosis (excluding catheter-related thrombosis) or conditions associated with increased risk of thrombosis

Locations

  • University of California San Francisco accepting new patients
    San Francisco California 94143 United States
  • University of Washington Medical Center accepting new patients
    Seattle Washington 98195 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
4D Molecular Therapeutics
ID
NCT05248230
Phase
Phase 1/2 research study
Study Type
Interventional
Participants
Expecting 40 study participants
Last Updated