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Trial to Evaluate Beta-Lactam Antimicrobial Therapy of Community Acquired Pneumonia in Children

Phase 4
Completed
Conditions
Pneumonia
Interventions
Drug: Amoxicillin-clavulanate
Other: Placebo
Registration Number
NCT02891915
Lead Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Brief Summary

This is a multi-center, randomized, double-blind, placebo-controlled, superiority clinical trial will test the effectiveness of short (5-day) vs.standard (10-day) course therapy in children who are diagnosed with CAP and initially treated in outpatient clinics, urgent care facilities, and emergency departments. Primary objective is to compare the composite overall outcome (Desirability of Outcome Ranking, DOOR) among children 6-71 months of age with CAP assigned to a strategy of short course (5 days) vs standard course (10 days) outpatient beta-lactam therapy at Outcome Assessment Visit #1 (Study Day 8 +/- 2 days)

Detailed Description

This is a multi-center, randomized, double-blind, placebo-controlled, superiority clinical trial evaluating short course (5 day) vs. standard course (10 day) of oral beta-lactam antibiotic therapy (amoxicillin, amoxicillin-clavulanate, cefdinir) for treatment of CAP in children 6-71 months of age who have clinically improved prior to enrollment. The study will randomize approximately 400 enrolled subjects to one of the two study arms (approximately 200 children in each arm) in order to reach 360 subjects completing Outcome Assessment Visit 1. Subjects will be randomized (1:1) to receive either a standard course of the initially prescribed antibiotic (10 days) or a short course of the initially prescribed antibiotic (5 days) plus 5 days of matching placebo. The study will recruit potential subjects from children who are diagnosed with CAP and who are initiated on oral beta-lactam therapy by healthcare providers in EDs, outpatient clinics, and urgent care centers at the study sites. Day -5 is defined as the date on which oral beta-lactam therapy is initiated for a diagnosis of CAP. Potential subjects will be identified at any time following clinical diagnosis of pneumonia. These subjects will be assessed for eligibility and enrolled on Day -3 to -1 of their initially prescribed oral beta-lactam therapy. Subjects may also be enrolled on Day 1 (the first day of receipt of study agent) provided they have not yet received any doses of the healthcare provider-prescribed antibiotic therapy for that day. The Primary objective is to compare the composite overall outcome (Desirability of Outcome Ranking, DOOR) among children 6-71 months of age with CAP assigned to a strategy of short course (5 days) vs standard course (10 days) outpatient beta-lactam therapy at Outcome Assessment Visit #1 (Study Day 8 +/- 2 days). The Secondary objectives are: 1) To compare the composite overall outcome (DOOR) among children 6-71 months of age with CAP assigned to a strategy of short course (5 days) vs standard course (10 days) outpatient beta-lactam therapy at Outcome Assessment Visit #2 (Study Day 22 +/- 3 days); 2) To compare the resolution of symptoms (a component of DOOR) among children 6-71 months of age with CAP assigned to a strategy of short course (5 days) vs standard course (10 days) outpatient beta-lactam therapy at Outcome Assessment Visits #1 and #2; 3) To compare the clinical response (a component of DOOR) among children 6-71 months of age with CAP assigned to a strategy of short course (5 days) vs standard course (10 days) outpatient beta-lactam therapy at Outcome Assessment Visits #1 and #2; 4) To compare solicited events (a component of DOOR) among children 6-71 months of age with CAP assigned to a strategy of short course (5 days) vs standard course (10 days) outpatient beta-lactam therapy at Outcome Assessment Visits #1 and #2; 5) To compare medically attended visits to Emergency Departments (ED) or outpatient clinics, hospitalizations, surgical procedures, and receipt of non-study systemic antibiotics (components of the clinical response) among children 6-71 months of age with CAP assigned to a strategy of short course (5 days) vs standard course (10 days) outpatient beta-lactam therapy at Outcome Assessment Visits #1 and #2

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
385
Inclusion Criteria
  1. Age 6 - 71 months

  2. Provider diagnosis of CAP and prescription of antibiotic therapy with amoxicillin, amoxicillin-clavulanate, or cefdinir

    • amoxicillin or amoxicillin-clavulanate prescribed at a amoxicillin dose of 60 mg/kg/day

    -- cefdinir prescribed at a minimum dose of 10 mg/kg/day

  3. Parental report of clinical improvement

    • based on lack of either subjective or known fever temperature >/= 38.3°C in the preceding 24 hours; current respiratory rate no greater than 50 breaths/minute (<2 years of age) or breaths/minute (= / > 2 years of age); and current grade of cough < 3
  4. Ability of a parent or guardian to understand and comply with the study procedures and be available for all study visits

  5. Signed written informed consent by a parent or guardian

Exclusion Criteria
  1. Treatment with any systemic antibiotic therapy within 7 days before the diagnosis of CAP 2. Initial therapy for CAP with combination antibiotic therapy
  • amoxicillin, amoxicillin/clavulanate or cefdinir plus one or more additional oral, intravenous, or intramuscular antibiotics 3. History of anaphylaxis or severe drug allergy to amoxicillin, if prescribed amoxicillin or amoxicillin/clavulanic acid; or oral cephalosporin antibiotics (except cefaclor), if prescribed cefdinir 4. Presence of concomitant bacterial infection that requires > 5 days of antibiotic therapy 5. Radiographic findings (where applicable) of complicated pneumonia at presentation or any subsequent chest radiograph up to the time of enrollment

  • clinically significant pleural effusion, lung abscess, or pneumatocele 6. Hospitalization for pneumonia during Day -5 to -1 of antibiotic therapy for CAP

  • subjects who require serial clinical assessments, but are discharged within 24 hours will not be considered hospitalized and will not satisfy this exclusion criterion 7. Pneumonia due to S. aureus or group A streptococcus documented by positive blood culture or PCR, at the time of enrollment 8. History of pneumonia within the previous 6 months 9. History of persistent asthma within the previous 6 months or current acute asthma exacerbation

  • persistent asthma is defined as receiving daily asthma maintenance therapy such as inhaled corticosteroids, cromolyn, theophylline, or leukotriene receptor antagonists

    -- acute asthma exacerbation is defined as receiving concomitant bronchodilator therapy and systemic corticosteroids 10. Provider-diagnosis of aspiration pneumonia, bronchiolitis, or bronchitis 11. Surgery or other invasive procedures of the upper or lower airway (e.g., bronchoscopy, laryngoscopy) with general anesthesia or hospitalization </=7 days before diagnosis of CAP 12. History of an underlying chronic medical condition

  • including chronic heart disease, chronic lung disease (except asthma), congenital anomalies of the airways or lung, cystic fibrosis, chronic renal disease including nephrotic syndrome, protein-losing enteropathy of any cause, severe malnutrition, neurocognitive disorders, metabolic disorders (including phenylketonuria), or genetic disorders (note: genetic syndromes such as Down syndrome and Edwards Syndrome are excluded; however, children with genetic disorders (e.g., hemophilia) but who do not have a genetic syndrome may not satisfy this particular exclusion criterion; it is important that children with such genetic disorders do not have symptoms and/or comorbidities that would pose additional risk to them nor jeopardize the adequacy of study assessments.) 13. History of a condition that compromises the immune system

  • HIV infection, primary immunodeficiency, anatomic or functional asplenia; receipt of a hematopoietic stem cell or solid organ transplant at any time; receipt of immunosuppressive therapy including chemotherapeutic agents, biologic agents, antimetabolites or radiation therapy during the past 12 months; or daily use of systemic corticosteroids for more than 7 consecutive days during the past 14 days 14. Any other condition that in the judgment of the investigator precludes participation because it could affect the safety of the subject 15. Current enrollment in another clinical trial of an investigational agent 16. Previous enrollment in this trial

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
ShortAmoxicillin-clavulanate200 subjects will receive a short course of the initially prescribed antibiotic for 5 days plus 5 days of matching placebo
ShortAmoxicillin200 subjects will receive a short course of the initially prescribed antibiotic for 5 days plus 5 days of matching placebo
ShortPlacebo200 subjects will receive a short course of the initially prescribed antibiotic for 5 days plus 5 days of matching placebo
StandardAmoxicillin-clavulanate200 subjects will receive a standard course of the initially prescribed antibiotic( Amoxicillin, Amoxicillin-Clavulanate, Cefdinir) for 10 days
ShortCefdinir200 subjects will receive a short course of the initially prescribed antibiotic for 5 days plus 5 days of matching placebo
StandardAmoxicillin200 subjects will receive a standard course of the initially prescribed antibiotic( Amoxicillin, Amoxicillin-Clavulanate, Cefdinir) for 10 days
StandardCefdinir200 subjects will receive a standard course of the initially prescribed antibiotic( Amoxicillin, Amoxicillin-Clavulanate, Cefdinir) for 10 days
Primary Outcome Measures
NameTimeMethod
Desirability of Outcome Ranking (DOOR)Outcome Assessment Visit 1 (Study Day 8 +/- 2 days)

DOOR is a composite endpoint created using clinical outcomes from the first 5 days and at Outcome Assessment Visit #1 (OAV #1). It is based on adequate clinical response at OAV #1, solicited symptoms from first 5 days and number of days of antibiotics use for worsening pneumonia from the first 5 days of the study.

Secondary Outcome Measures
NameTimeMethod
Number of Participants Receiving Non-study Systemic Antibiotics for All Causes During Medically Attended VisitsOutcome Assessment Visit 2 (Study Day 22 +/- 3 days)

This table provides number and percentage of subjects who received non-study antibiotics for any reason from Day 1 to Day 18

Number of Participants Receiving Non-study Systemic Antibiotics for Persistent or Worsening Pneumonia During Medically Attended VisitsOutcome Assessment Visit 2 (Study Day 22 +/- 3 days)

This table provides number and percentage of subjects who received non-study antibiotics for any use from Day 1 to Day 18

Adequate Clinical Response Rates (a Component of DOOR)Outcome Assessment Visit 2 (Study Day 22 +/- 3 days)

Lack of adequate clinical response at OAV #2 is defined as the presence of a medically attended visit to an ED or outpatient clinic or hospitalization or receipt of non-study antibiotics or surgical procedures for persistent or worsening pneumonia from Day 1 to Day 18.

Number of Participants Reporting Solicited SymptomsOutcome Assessment Visit 2 (Study Day 22 +/- 3 days)

This table summarizes the number and percentage of subjects experiencing any solicited events of mild, moderate or severe severity from Day 1 to Day 18

Desirability of Outcome Ranking (DOOR)Outcome Assessment Visit 2 (Study Day 22 +/- 3 days)

DOOR is a composite endpoint created using clinical outcomes from the first 18 days and at Outcome Assessment Visit #2 (OAV #2). It is based on adequate clinical response at OAV #2, solicited symptoms from first 18 days and number of days of antibiotics use for worsening pneumonia from the first 18 days of the study.

Resolution of Symptoms (a Component of DOOR)Outcome Assessment Visit 2 (Study Day 22 +/- 3 days)

This table provides number and percentage of subjects who experienced lack of resolution of symptoms by their OAV #2.

Trial Locations

Locations (9)

University of Alabama - Children's of Alabama - Infectious Diseases/Virology

🇺🇸

Birmingham, Alabama, United States

University of Louisville School of Medicine - Norton Children's Hospital - Infectious Diseases

🇺🇸

Louisville, Kentucky, United States

Washington University School of Medicine in St. Louis - Infectious Diseases

🇺🇸

Saint Louis, Missouri, United States

Duke Human Vaccine Institute - Duke Vaccine and Trials Unit

🇺🇸

Durham, North Carolina, United States

Cincinnati Children's Hospital Medical Center - Infectious Diseases

🇺🇸

Cincinnati, Ohio, United States

Children's Hospital of Philadelphia - The Center for Pediatric Clinical Effectiveness

🇺🇸

Philadelphia, Pennsylvania, United States

Children's Hospital of Pittsburgh of UPMC - General Academic Pediatric

🇺🇸

Pittsburgh, Pennsylvania, United States

Arkansas Children's Hospital - Infectious Diseases

🇺🇸

Little Rock, Arkansas, United States

Vanderbilt University - Pediatric - Vanderbilt Vaccine Research Center

🇺🇸

Nashville, Tennessee, United States

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