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

Exclusion Criteria16

• 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.
• 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
• Lung infection with Mycobacterium abscessus associated with a more rapid decline in pulmonary status
• Currently receiving treatment for active lung infection with Burkholderia cenocepacia or Burkholderia dolosa
• History of solid organ or hematological transplantation
• History of clinically significant cirrhosis with or without portal hypertension
• History of pulmonary hypertension
• History of cardiotoxicity, a history of known coronary artery disease, and/or existing cardiomyopathy
• 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
• History of allergic bronchopulmonary aspergillosis (ABPA)
• Uncontrolled diabetes mellitus, as evidenced by hemoglobin A1c >9% at Screening
• Clinically significant laboratory abnormalities at Screening
• 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
• Subjects who received any investigational products within 30 days (or 5 therapeutic half-lives, whichever is longer) prior to Screening
• Subjects who have previously received any gene therapy agent
• Subjects with known sensitivity to SP-101, doxorubicin or its excipients