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

  • STATUS
    Recruiting
  • End date
    Aug 16, 2025
  • participants needed
    21
  • sponsor
    4D Molecular Therapeutics
Updated on 16 October 2022
oximetry
forced expiratory volume
cystic fibrosis transmembrane conductance regulator
cftr gene

Summary

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.

Description

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.

Details
Condition Cystic Fibrosis Lung
Treatment 4D-710
Clinical Study IdentifierNCT05248230
Sponsor4D Molecular Therapeutics
Last Modified on16 October 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

18 years and older
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
Forced expiratory volume in 1 second (FEV1) ≥50% and ≤100% of predicted (per Global
Lung Function Initiative) at Screening
Resting oxygen saturation ≥ 92% on room air at Screening

Exclusion Criteria

Any prior gene therapy for any indication
History of positive culture for Burkholderia cenocepacia, B. dolosa, or Mycobacterium abscessus within the past 24 months
Active allergic bronchopulmonary aspergillosis requiring management with systemic corticosteroids or antifungal therapy
Contraindication to systemic corticosteroid therapy
Requires chronic use of systemic corticosteroids or immunosuppressants to treat another condition
If no known diagnosis of cystic fibrosis related diabetes (CFRD), Type I, or Type II diabetes: Hemoglobin A1C ≥6.5% at Screening
Body Mass Index (BMI) <16
If known diagnosis of CFRD, Type I or Type II diabetes: Hemoglobin A1C >7.5% at Screening
Laboratory abnormalities 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
Total bilirubin ≥ 2 × ULN
Hemoglobin < 10 g/dL
Requirement for continuous or night-time oxygen supplementation
ALT, AST or GGT ≥ 3 × the upper limit of normal (ULN)
Known CF liver disease with evidence of cirrhosis
History of thrombosis (excluding catheter-related thrombosis) or conditions associated with increased risk of thrombosis
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