An Open-label, Phase 1/2 Trial of Gene Therapy 4D-710 in Adults With Cystic Fibrosis
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
• 18 years and older
• Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including:
‣ Sweat chloride ≥ 60 mmol/L
⁃ Mutation Status
• Bi-allelic mutations in the CFTR gene, or
∙ Single mutation in the CFTR gene and clinical manifestations of CF lung disease
⁃ 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 ≤ 90% of predicted (per Global Lung Function Initiative) at Screening
• Resting oxygen saturation ≥ 92% on room air at Screening
• 18 years and older
• Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including:
‣ Sweat chloride ≥ 60 mmol/L
⁃ Mutation Status
• Bi-allelic mutations in the CFTR gene, or
∙ Single mutation in the CFTR gene and clinical manifestations of CF lung disease
• 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
• 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