Standardized Treatment of Pulmonary Exacerbations II
Condition: Pulmonary Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: Standard of care IV antibiotic(s)
Intervention description: IV antibiotics will be selected by the treating physician following standard of care. Duration of treatment is the assigned intervention.
Intervention MeSH terms: Anti-Bacterial Agents
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 982.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Cystic fibrosis; Pulmonary exacerbation; Antibiotic; Treatment duration; Lung infection; Cystic Fibrosis Foundation; Cystic Fibrosis Foundation National Patient Registry

Summary:

      Cystic fibrosis (CF), a life-shortening genetic disease, is marked by acute episodes during
      which symptoms of lung infection increase and lung function decreases. These pulmonary
      exacerbations are treated with varying antibiotics for varying time periods based on needs
      determined by individual patients, their families, and the health care providers. Cystic
      fibrosis pulmonary guidelines for the treatment of pulmonary exacerbation published by the
      Cystic Fibrosis Foundation (CFF) in 2009 provided recommendations for treatment and also
      identified key questions for which additional studies were needed.

      A strong desire among clinicians to reduce treatment durations (and reduce cost,
      inconvenience, and potential toxicities) is in conflict with belief that patients not
      responding robustly to treatment might benefit from extending treatment.

      This randomized, controlled, open-label study is designed to evaluate the efficacy and safety
      of differing durations of IV treatment, given in the hospital or at home for a pulmonary
      exacerbation in adult patients with CF.
    

Eligibility criteria:

        Inclusion Criteria:

        Key Inclusion Criteria:

          -  Male or female ≥18 years of age at Visit 1

          -  Documentation of a CF diagnosis

          -  Enrolled in the Cystic Fibrosis Foundation National Patient Registry (CFFNPR) prior to
             Visit 1 (US sites only)

          -  At the time of Visit 1, there is a plan to initiate IV antibiotics for a pulmonary
             exacerbation

          -  Performed spirometry at Visit 1 and Visit 2 and willing to perform spirometry at Visit
             3

          -  Completed the CRISS questionnaire at Visit 1 and Visit 2 and willing to complete the
             Cystic Fibrosis Respiratory Symptoms Diary (CFRSD) questionnaire at Visit 3

          -  Willing to adhere to a specific treatment duration determined by initial response to
             treatment and subsequent randomization

          -  Willing to return for follow up Visit 3

          -  Written informed consent obtained from the subject or subject's legal representative

        Exclusion Criteria:

        Key Exclusion Criteria

          -  Previous randomization in this study

          -  Treatment with IV antibiotics in the 6 weeks prior to Visit 1

          -  Admission to the intensive care unit for current pulmonary exacerbation in the two
             weeks prior to Visit 2, unless admission was due to a desensitization protocol

          -  Pneumothorax in the two weeks prior to Visit 2

          -  Primary diagnosis for current hospitalization is unrelated to worsening lower
             respiratory symptoms (e.g., pulmonary clean out, distal intestinal obstruction
             syndrome (DIOS), sinusitis)

          -  Massive hemoptysis defined as > 250 cc in a 24 hour period or 100 cc/day over 4
             consecutive days occurring in the two weeks prior to Visit 2

          -  Current pulmonary exacerbation thought to be due to allergic bronchopulmonary
             aspergillosis (ABPA)

          -  At Visit 1, receiving ongoing treatment with a duration of more than 2 weeks with
             prednisone equivalent to >10mg/day

          -  History of solid organ transplantation

          -  Receiving antimicrobial therapy to treat non-tuberculous mycobacterium (e.g., M.
             abscessus, M. avium complex) in the two weeks prior to Visit 2