A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF)

Eligibility Criteria

• 1. Males or females, age 18 to 65 years at Screening Visit, inclusive
• 2. Diagnosis of CF
• 3. ppFEV1 value between 50-100% (inclusive)
• 4. Resting oxygen saturation ≥94% on room air by pulse oximetry 5 . Clinically stable CF disease as assessed by the Investigator and not requiring any new class of interventional treatment within the last 3 months prior to Screening
• 1. Any change in established pulmonary treatment (including antibiotics) within 28 days prior to Screening Visit. However, inhaled beta-agonists can be included within 2 weeks prior to Screening Visit.
• 2. Clinically significant episode of hemoptysis (\>50 mL or ¼ cup or 10 teaspoons per day) within 12 weeks prior to dosing with study drug on Day 1
• 3. Lung infection with Mycobacterium abscessus associated with a more rapid decline in pulmonary status
• 4. Currently receiving treatment for active lung infection with Burkholderia cenocepacia or Burkholderia dolosa
• 5. History of solid organ or hematological transplantation
• 6. History of clinically significant cirrhosis with or without portal hypertension
• 7. History of pulmonary hypertension
• 8. History of cardiotoxicity, a history of known coronary artery disease, and/or existing cardiomyopathy
• 9. Current active fungal infection (not just a positive culture), acute blood, lung, or bladder infection, clinically significant hepatic or renal dysfunction, and/or viral infection (including human immunodeficiency virus or hepatitis virus B or C) requiring the initiation of new therapy within 30 days prior to Screening
• 10. History of allergic bronchopulmonary aspergillosis (ABPA)
• 11. Uncontrolled diabetes mellitus, as evidenced by hemoglobin A1c \>9% at Screening
• 12. Clinically significant laboratory abnormalities at Screening
• 13. Subjects with any medical condition or abnormal laboratory result that, in the opinion of the Investigator, will interfere with the safe completion of the study
• 14. Subjects who received any investigational products within 30 days (or 5 therapeutic half-lives, whichever is longer) prior to Screening
• 15. Subjects who have previously received any gene therapy agent
• 16. Subjects with known sensitivity to SP-101, doxorubicin or its excipients