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Standardized Treatment of Pulmonary Exacerbations II (STOP2)

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ClinicalTrials.gov Identifier: NCT02781610
Recruitment Status : Recruiting
First Posted : May 24, 2016
Last Update Posted : June 5, 2019
Sponsor:
Collaborators:
Cystic Fibrosis Foundation
CF Therapeutics Development Network Coordinating Center
Medical University of South Carolina
University of Washington
Information provided by (Responsible Party):
Chris Goss, Seattle Children's Hospital

Tracking Information
First Submitted Date  ICMJE May 12, 2016
First Posted Date  ICMJE May 24, 2016
Last Update Posted Date June 5, 2019
Study Start Date  ICMJE June 2016
Estimated Primary Completion Date February 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 23, 2016)
  • Absolute change in FEV1 % predicted from Visit 1 to Visit 3 between ERR-10 day and ERR-14 Day [ Time Frame: Start of IV antibiotic treatment to14 days after the end of IV antibiotic treatment ]
  • Absolute change in FEV1 % predicted from Visit 1 to Visit 3 between NERR-14 day and NERR-21 [ Time Frame: Start of IV antibiotic treatment to14 days after the end of IV antibiotic treatment ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02781610 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 23, 2016)
  • Change in CRISS from Visit 1 to Visit 3 between ERR-10 day and ERR-14 day [ Time Frame: Start of IV antibiotic treatment to14 days after the end of IV antibiotic treatment ]
  • Change in CRISS from Visit 1 to Visit 3 between NERR-14 day and NERR-21 day [ Time Frame: Start of IV antibiotic treatment to14 days after the end of IV antibiotic treatment ]
  • Change in weight from Visit 1 to Visit 3 between ERR-10 day and ERR-14 day [ Time Frame: Start of IV antibiotic treatment to14 days after the end of IV antibiotic treatment ]
  • Change in weight from Visit 1 to Visit 3 between NERR-14 day and NERR-21 day [ Time Frame: Start of IV antibiotic treatment to14 days after the end of IV antibiotic treatment ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Standardized Treatment of Pulmonary Exacerbations II
Official Title  ICMJE Standardized Treatment of Pulmonary Exacerbations II (STOP2)
Brief 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.

Detailed Description

The study will assess the non-inferiority of 10 days versus 14 days treatment duration among patients who have an early robust improvement (ERR subjects) and the superiority of 21 days versus 14 days treatment duration among the subjects who do not meet the definition of ERR (non-ERR; NERR).

Subjects will undergo pulmonary function testing (spirometry) and complete a respiratory symptom score [Chronic Respiratory Infection Symptom Score (CRISS)] at initiation of IV treatment (Baseline/ Visit 1) and at Day 7-10 (Visit 2). At Visit 2, subjects will be allocated to groups ERR or NERR based on their initial clinical response as determined by the change in forced expiratory volume in 1 second (FEV1; percent of predicted) and CRISS from Baseline and then randomized to an IV treatment duration (nested within group).

ERR subjects [≥8% predicted improvement in FEV1 from Visit 1 to Visit 2 and CRISS reduction of ≥11 points from Visit 1 to Visit 2] will be randomized 1:1 to either 10 days or 14 days total IV antibiotic treatment duration. Remaining (NERR) subjects will be randomized 1:1 to receive either 14 or 21 days total IV antibiotic treatment duration. All subjects will be evaluated again at Visit 3, 14 days following scheduled completion of IV antibiotic treatment.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Pulmonary Cystic Fibrosis
Intervention  ICMJE Drug: Standard of care IV antibiotic(s)
IV antibiotics will be selected by the treating physician following standard of care. Duration of treatment is the assigned intervention.
Study Arms  ICMJE
  • ERR-10
    ERR treatment duration - 10 Day Standard of care IV antibiotic(s) will be selected by the treating physician. Duration of treatment is the assigned intervention.
    Intervention: Drug: Standard of care IV antibiotic(s)
  • ERR-14
    ERR treatment duration - 14 Day Standard of care IV antibiotic(s) will be selected by the treating physician. Duration of treatment is the assigned intervention.
    Intervention: Drug: Standard of care IV antibiotic(s)
  • NERR-14
    NERR treatment duration - 14 Day Standard of care IV antibiotic(s) will be selected by the treating physician. Duration of treatment is the assigned intervention.
    Intervention: Drug: Standard of care IV antibiotic(s)
  • NERR-21
    NERR treatment duration - 21 Day Standard of care IV antibiotic(s) will be selected by the treating physician. Duration of treatment is the assigned intervention.
    Intervention: Drug: Standard of care IV antibiotic(s)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 12, 2018)
1300
Original Estimated Enrollment  ICMJE
 (submitted: May 23, 2016)
880
Estimated Study Completion Date  ICMJE February 2020
Estimated Primary Completion Date February 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Barbra Fogarty 206-884-7592 barbra.fogarty@seattlechildrens.org
Listed Location Countries  ICMJE Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02781610
Other Study ID Numbers  ICMJE STOP2-IP-15
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Chris Goss, Seattle Children's Hospital
Study Sponsor  ICMJE Chris Goss
Collaborators  ICMJE
  • Cystic Fibrosis Foundation
  • CF Therapeutics Development Network Coordinating Center
  • Medical University of South Carolina
  • University of Washington
Investigators  ICMJE
Principal Investigator: Chris Goss, MD University of Washington
Principal Investigator: Patrick Flume, MD Medical University of South Carolina
PRS Account Seattle Children's Hospital
Verification Date June 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP