Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic Fibrosis (RESTORE-CF)

This is an interventional treatment trial for Cystic Fibrosis focused on measuring Cystic Fibrosis, CF Read More

Inclusion Criteria:

• Confirmed diagnosis of CF as defined by both of the following:

  • Two CF disease-causing CFTR mutations in Class I or II (genotype confirmed at the screening visit).
  • Chronic sinopulmonary disease and/or gastrointestinal/nutritional abnormalities consistent with CF disease.
• Clinically stable CF disease, as judged by the investigator.
• FEV1 ≥50% and ≤90% of the predicted normal for age, gender, and height at screening.
• Resting oxygen saturation ≥92% on room air (pulse oximetry).

Exclusion Criteria:

• An acute upper or lower respiratory infection, pulmonary exacerbation, clinically significant episode of hemoptysis or change in chronic respiratory medications (including antibiotics) for CF lung disease within 28 days prior to dosing with investigational product on Day 1.
• Receiving treatment with ivacaftor monotherapy (KALYDECO)
• For all groups except Daily dosing: Receiving treatment with triple combination therapy (TRIKAFTA).
• Subjects with a Class III, IV, or V CFTR gene mutation in at least 1 allele.
• Infection with highly virulent bacteria associated with accelerated decline in pulmonary function and/or decreased survival (e.g., Burkholderia cenocepacia, Burkholderia dolosa, Mycobacterium abscessus).

Treatment with ORKAMBI or SYMDEKO is not an exclusion for this study.