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Evaluation of Pharmacokinetics, Safety, and Tolerability of Ceftazidime-avibactam in Neonates and Infants.

Phase 2
Terminated
Conditions
Gram-negative Bacterial Infection
Registration Number
NCT04126031
Lead Sponsor
Pfizer
Brief Summary

This study will assess the pharmacokinetics, safety, and tolerability of single and multiple doses of intravenous ceftazidime-avibactam in hospitalized infants and neonates from 26 weeks gestation to 3 months of age. In Part A of the study all patients will receive a single dose of ceftazidime-avibactam. In Part B all patients will received multiple doses of ceftazidime-avibactam. Efficacy will be assessed in the infants and neonates receiving multiple doses of ceftazidime-avibactam.

Detailed Description

This is a 2-part, Phase 2a, non-randomized, open-label multicenter, multinational study of intravenous ceftazidime-avibactam in hospitalized neonates and infants with suspected or confirmed bacterial infection. In Part A of the study, patients already receiving intravenous antibacterial therapy with another antibiotic will receive a single intravenous dose of ceftazidime-avibactam followed by observation for 48 hours and a Late Follow-Up assessment 4-5 weeks later. In Part B of the study, patients with suspected or confirmed Gram-negative bacterial infections requiring intravenous antibacterial therapy will receive multiple doses of intravenous ceftazidime-avibactam for up to 14 days. At the discretion of the investigator, patients may also receive other antibiotics if the infection is suspected to include Gram-positive bacteria, multi-drug resistant Gram-negative bacteria, or anaerobic bacteria. At the discretion of the investigator, patients may be switched to oral therapy or outpatient parenteral antimicrobial therapy with an alternative antibiotic after receiving intravenous ceftazidime-avibactam for at least 48 yhours. Clinical outcomes will be assessed at the End of Intravenous (EOIV) treatment with ceftazidime-avibactam, the End-of-Therapy (EOT), the Test-of-Cure (TOC) at 7-14 days after the last study therapy and at a Late Follow-Up (LFU) visit, 28-55 days after the last dose of ceftazidime-avibactam. Safety assessments will occur throughout the study. Ceftazidime-avibactam blood levels will be assessed during the first 12 hours after the single dose of ceftazidime-avibactam in Part A and during 12 hours after at least 3 consecutive doses of ceftazidime-avibactam in Part B.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
48
Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Primary Outcome Measures
NameTimeMethod
Plasma Concentrations of Ceftazidime and Avibactam 2 Hours Post-dose: Part A2 hours post dose on Day 1
Plasma Concentrations of Ceftazidime and Avibactam 2 Hours and 30 Minutes Post-dose: Part A2 hours and 30 minutes post dose on Day 1
Plasma Concentrations of Ceftazidime and Avibactam of 7 Hours Post-dose: Part A7 hours post dose on Day 1
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs): Part BDay 1 up to maximum of Day 49

An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in participant hospitalization; life-threatening experience (immediate risk of dying) ; persistent or significant disability/incapacity; congenital anomaly.

Number of Participants Who Discontinued Treatment and Study Due to AEs: Part BDay 1 up to maximum of Day 49
Number of Participants Who Died: Part BDay 1 up to maximum of Day 49
Number of Participants With Clinically Significant Laboratory Parameters Occurred in More Than 2 Participants: Part BDay 1 up to maximum of Day 49

Number of participants in Part B with clinically significant abnormal laboratory parameters that occurred in more than 2 participants from Day 1 up to 35 days after the last dose of CAZ-AVI were reported in this outcome measure. Clinically significant labs were abnormal laboratory results which the investigator reported as being clinically significant. Only parameters with non-zero values are reported.

Secondary Outcome Measures
NameTimeMethod
Number of Participants According to Clinical Outcome At End of IV Treatment(EOIV), End of Treatment(EOT), Test of Cure(TOC) and Late Follow-Up(LFU) in Modified-ITT Analysis Population: Part BEOIV, EOT, TOC, LFU

Clinical outcome assessed based on clinical cure, improvement, failure, indeterminate. Clinical cure=resolution of acute signs, symptoms.Clinical improvement=participants switched to oral therapy;met following criteria at EOIV:afebrile for 24 hours(H);improvement in at least 1 symptom,sign.Clinical failure=received \>48H of therapy, met any of these:therapy discontinuation due to insufficient effect, AE, death. Indeterminate=data not available for evaluation(death;lost to follow up;diagnosis of CNS infection, osteomyelitis, endocarditis or necrotizing enterocolitis after enrollment). EOIV (Up to 14 days), EOT (Up to 27 days), TOC (Up to 34 days), LFU (Up to 49 days).

Number of Participants According to Microbiological Response at TOC Visit in Micro-ITT Population: Part BUp to 34 days

Microbiological response was assessed based on eradication, presumed eradication, persistence, presumed persistence, indeterminate. Eradication: source specimen demonstrated absence of the original baseline pathogen. Presumed eradication: source specimen was not available to culture and the participant was assessed as a clinical cure. Persistence: source specimen demonstrated continued presence of the original baseline pathogen. Presumed persistence: source specimen was not available to culture and the participant was assessed as a clinical failure. Indeterminate: source specimen was not available to culture and the participant's clinical outcome was assessed as indeterminate.

Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs): Part ADay 1 up to maximum of Day 35

An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly.

Number of Participants Who Died: Part ADay 1 up to maximum of Day 35
Number of Participants Who Discontinued Treatment and Study Due to AEs: Part ADay 1 up to maximum of Day 35
Plasma Concentrations of Ceftazidime and Avibactam 2 Hours, 2 Hours and 30 Minutes, 7 Hours Post Doses on Day 1: Part B2 hours, 2 hours 30 mins, and 7 hours post dose on Day 1
Number of Participants According to Clinical Outcome At End of IV Treatment(EOIV), End of Treatment(EOT), Test of Cure(TOC) and Late Follow-Up(LFU): Intent to Treat (ITT) Analysis Population: Part BEOIV, EOT, TOC, LFU

Clinical outcome assessed based on clinical cure, improvement, failure, indeterminate. Clinical cure=resolution of acute signs, symptoms.Clinical improvement=participants switched to oral therapy;met following criteria at EOIV:afebrile for 24 hours(H);improvement in at least 1 symptom,sign.Clinical failure=received \>48H of therapy, met any of these:therapy discontinuation due to insufficient effect, AE, death. Indeterminate=data not available for evaluation(death;lost to follow up;diagnosis of CNS infection, osteomyelitis, endocarditis or necrotizing enterocolitis after enrollment). EOIV (Up to 14 days), EOT (Up to 27 days), TOC (Up to 34 days), LFU (Up to 49 days).

Number of Participants According to Clinical Outcome At End of IV Treatment(EOIV), End of Treatment(EOT), Test of Cure(TOC) and Late Follow-Up(LFU) in Micro-ITT Analysis Population: Part BEOIV, EOT, TOC, LFU

Clinical outcome assessed based on clinical cure, improvement, failure, indeterminate. Clinical cure=resolution of acute signs, symptoms.Clinical improvement=participants switched to oral therapy;met following criteria at EOIV:afebrile for 24 hours(H);improvement in at least 1 symptom,sign.Clinical failure=received \>48H of therapy, met any of these:therapy discontinuation due to insufficient effect, AE, death. Indeterminate=data not available for evaluation(death;lost to follow up;diagnosis of CNS infection, osteomyelitis, endocarditis or necrotizing enterocolitis after enrollment). EOIV (Up to 14 days), EOT (Up to 27 days), TOC (Up to 34 days), LFU (Up to 49 days).

Number of Participants With Emergent Infections in Micro-ITT Analysis Population: Part BDay 1 up to maximum of Day 49

Emergent infections included superinfection and new infection. Superinfection: a culture identified pathogen other than a baseline pathogen during the course of active treatment with study therapy requiring alternative antimicrobial therapy. New infection: a culture identified pathogen other than a baseline pathogen at any time after study treatment has finished requiring alternative antimicrobial therapy.

Number of Participants With Clinically Significant Laboratory Parameters Occurred in More Than 2 Participants: Part ADay 1 up to maximum of Day 35

Number of participants in Part A with clinically significant abnormal laboratory parameters that occurred in more than 2 participants from Day 1 up to 35 days after the last dose of CAZ-AVI were reported in this outcome measure. Clinically significant labs were abnormal laboratory results which the investigator reported as being clinically significant.

Trial Locations

Locations (18)

"ATTIKON" University General Hospital

🇬🇷

Chaidari, Athens, Greece

Duke University Medical Center

🇺🇸

Durham, North Carolina, United States

Tufts Children's Hospital at Tufts Medical Center

🇺🇸

Boston, Massachusetts, United States

Hsinchu Mackay Memorial Hospital

🇨🇳

Hsinchu, Taiwan

Kasturba Medical College and Hospital

🇮🇳

Manipal, Karnataka, India

Tallinn Children's Hospital

🇪🇪

Tallinn, Estonia

Debreceni Egyetem Klinikai Központ

🇭🇺

Debrecen, Hungary

Kanizsai Dorottya Korhaz

🇭🇺

Nagykanizsa, Hungary

Ospedale Pediatrico Bambino Gesu

🇮🇹

Rome, RM, Italy

University of Utah

🇺🇸

Salt Lake City, Utah, United States

Duke University Investigational Drug Services

🇺🇸

Durham, North Carolina, United States

"Hippokration" General Hospital of Thessaloniki

🇬🇷

Thessaloniki, Greece

Athens General Children's Hospital "Panagioti and Aglaias Kyriakou"

🇬🇷

Athens, Ampelokipi, Greece

Szabolcs-Szatmár-Bereg Megyei Kórházak és Oktatókórház, Jósa András Oktatókórház

🇭🇺

Nyíregyháza, Hungary

National Taiwan University Hospital

🇨🇳

Taipei, Taiwan

Hsinchu Mackay Memorial Hospital, Department of Pharmacy

🇨🇳

Hsinchu City, R.o.c, Taiwan

Univerzitna nemocnica Martin

🇸🇰

Martin, Slovakia

Primary Children's Hospital

🇺🇸

Salt Lake City, Utah, United States

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