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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 : December 13, 2018
Sponsor:
Collaborators:
Cystic Fibrosis Foundation Therapeutics
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

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.


Condition or disease Intervention/treatment Phase
Pulmonary Cystic Fibrosis Drug: Standard of care IV antibiotic(s) Phase 4

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 : Interventional  (Clinical Trial)
Estimated Enrollment : 1300 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Standardized Treatment of Pulmonary Exacerbations II (STOP2)
Study Start Date : June 2016
Estimated Primary Completion Date : February 2020
Estimated Study Completion Date : February 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
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.
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.

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.
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.

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.
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.

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.
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.




Primary Outcome Measures :
  1. 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 ]
  2. 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 ]

Secondary Outcome Measures :
  1. 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 ]
  2. 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 ]
  3. 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 ]
  4. 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 ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02781610


Contacts
Contact: Barbra Fogarty 206-884-7592 barbra.fogarty@seattlechildrens.org

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Sponsors and Collaborators
Chris Goss
Cystic Fibrosis Foundation Therapeutics
CF Therapeutics Development Network Coordinating Center
Medical University of South Carolina
University of Washington
Investigators
Principal Investigator: Chris Goss, MD University of Washington
Principal Investigator: Patrick Flume, MD Medical University of South Carolina

Responsible Party: Chris Goss, Professor of Medicine and Pediatrics, Seattle Children's Hospital
ClinicalTrials.gov Identifier: NCT02781610     History of Changes
Other Study ID Numbers: STOP2-IP-15
First Posted: May 24, 2016    Key Record Dates
Last Update Posted: December 13, 2018
Last Verified: December 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Chris Goss, Seattle Children's Hospital:
Cystic fibrosis
Pulmonary exacerbation
Antibiotic
Treatment duration
Lung infection
Cystic Fibrosis Foundation
Cystic Fibrosis Foundation National Patient Registry

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Anti-Bacterial Agents
Antibiotics, Antitubercular
Anti-Infective Agents
Antitubercular Agents