A Study of Ceftobiprole Compared With Standard Antibiotic Treatment in Children With Staphylococcus Aureus Bacteremia (SAB) Infection, Including Right-sided Heart Infection

A Multicenter, Randomized, Investigator-blind, Active-controlled Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Ceftobiprole Versus Standard-of-care Treatment in Pediatric Patients From Birth to Less Than 18 Years With Staphylococcus Aureus Bacteremia, Including Right-sided Infective Endocarditis

This clinical trial will evaluate the safety, and effectiveness of ceftobiprole compared to standard-of-care antibiotic treatment in children from birth to less than 18 years of age with Staphylococcus aureus bloodstream infection, including right-sided infective endocarditis (IE). The trial will also evaluate the pharmacokinetics of ceftobiprole.

Study Overview

• Status: Not yet recruiting
• Conditions: Endocarditis, Bacterial; Staphylococcus Aureus Bacteremia
• Intervention / Treatment: Drug: Ceftobiprole medocaril sodium; Drug: Standard of Care (SOC)

• Study Type: Interventional
• Enrollment (Estimated): 60
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: ISTX Clinical Trials; Phone Number: 617-715-3600; Email: Clinicaltrials@istx.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 days to 17 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Key Inclusion Criteria:

• Pediatric age from birth to < 18 years old, with body weight ≥ 3.7 kg at the time of consent. Participants in the birth to < 2-year-old cohort should have a gestational age ≥ 37 weeks and postnatal age ≥ 3 days

• Parent(s) or legal guardian(s) have provided documented informed consent, and participant has provided assent as per local standards.

• SAB:

  1. Based on at least one blood culture obtained within 48 hours prior to randomization that is positive for S. aureus
  2. Persistent SAB

Key Exclusion Criteria:

• Receipt of systemic antibacterial treatment for S. aureus infections exceeding 48 hours within the 7 days prior to randomization.
• Infection with S. aureus known or suspected to be non-susceptible to ceftobiprole or available SOC comparator antibiotic options
• Known concomitant bloodstream or non-bloodstream infections with Gram-negative bacteria at screening
• Left-sided IE (known or suspected to be present at the time of randomization)
• Contraindication to the administration of study treatments, including known clinically-relevant hypersensitivity to beta-lactam antibacterials
• High probability of death within 7 days due to the underlying SAB or SAB-associated disease, or high probability of death within 28 days from an unrelated underlying disease
• An absolute neutrophil count of < 0.5 x 10^9/L
• Septic shock or hypotension unresponsive to IV fluids with or without vasopressors for ≥ 4 hours
• SAB in participants with other foreign body material that cannot be removed within 7 days after randomization
• Suspected or confirmed metastatic central nervous system S. aureus infection
• Immunocompromising conditions
• Receipt of systemic steroids (prednisolone ≥ 2 mg/kg per day, or equivalent) or immunosuppressive drugs within 2 weeks prior to screening
• Any severe, acute or chronic medical or psychiatric condition, recent neurological event, or laboratory abnormality that may increase the risk associated with study participation or administration of study treatments, or may interfere with the interpretation of study results, and which, in the judgment of the Investigator, would make the participant inappropriate for entry into this study
• eGFR of < 30 mL/min/1.73 m² for participants < 2 years old and < 15 mL/min/1.73 m² for participants ≥ 2 years old, or, for any age, requirement/anticipated requirement for plasmapheresis, peritoneal dialysis, hemodialysis, venovenous dialysis, continuous renal-replacement therapy (CRRT), or other forms of renal filtration.
• History of seizures other than well-documented febrile seizures of childhood
• Alanine transaminase (ALT) or aspartate transaminase (AST) levels > 5 × the upper limit of normal (ULN), OR total bilirubin > 2 × the ULN (except isolated unconjugated hyperbilirubinemia consistent with Gilbert's syndrome), OR severe hepatic disease classified as Child-Pugh class C

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ceftobiprole; Participants randomized to this arm will receive intravenous (IV) ceftobiprole.	Drug: Ceftobiprole medocaril sodium; Ceftobiprole dosing is based on age/body weight and renal function and given at 7.5 mg/kg, 10 mg/kg, or 15 mg/kg (equivalent to 10 mg/kg, 13.3 mg/kg, or 20 mg/kg respectively of ceftobiprole medocaril sodium) q8h, q12h or q24h up to a maximum of 500 mg (equivalent to 667 mg of ceftobiprole medocaril sodium), with all doses infused over 2 hours.
Active Comparator: Standard-of-Care (SOC); Participants randomized to this arm will receive intravenous (IV) standard-of-care (SOC) antibiotic treatment selected according to local guidelines or site-specific standard practice.	Drug: Standard of Care (SOC); Dosage is per local product label based on the participant's age/body weight, and, if applicable, renal function.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Percentage of Participants with Treatment-Emergent Adverse Events (TEAEs)	Day 1 through last follow-up (LFU) [up to 11 weeks]
Percentage of participants with serious adverse events (SAEs)	Baseline through LFU [up to 11 weeks]
Percentage of participants with treatment-related adverse events (AEs)	Day 1 through LFU [up to 11 weeks]
Percentage of participants with adverse events of special interest (AESIs)	Baseline through LFU [up to 11 weeks]
Percentage of participants with adverse events (AEs) leading to discontinuation of study-drug treatment	Baseline through LFU [up to 11 weeks]
Percentage of participants with treatment-emergent abnormal hematology laboratory test results	Baseline through the end-of-treatment (EOT) visit [up to approximately 6 weeks]
Percentage of participants with treatment-emergent abnormal blood chemistry laboratory test results	Baseline through the EOT visit [up to approximately 6 weeks]
Percentage of participants with treatment-emergent abnormal urinalysis test results	Baseline through the EOT visit [up to approximately 6 weeks]
Change from baseline in body temperature	Baseline through LFU visit [up to 11 weeks]
Change from baseline in systolic blood pressure	Baseline through LFU visit [up to 11 weeks]
Change from baseline in diastolic blood pressure	Baseline through LFU visit [up to 11 weeks]
Change from baseline in respiratory rate	Baseline through LFU visit [up to 11 weeks]
Change from baseline in heart rate	Baseline through LFU visit [up to 11 weeks]

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of All-Cause Mortality (ACM)	Defined as death from any cause including death due to SAB	Through 28 days after EOT [up to approximately 10 weeks]
Percentage of new metastatic foci or other complications of SAB	Complications are considered new between Day 8 and TOC	Day 8 through test-of-cure (TOC) [up to approximately 8 Weeks]
Percentage of participants with Clinical Success at the TOC Visit	Success is defined as either cure or improvement	TOC visit [7-14 days after EOT]
Percentage of participants with Microbiological Success at the TOC Visit	Microbiological success is defined as eradication or presumed eradication.	TOC visit (7-14 days after EOT)
Percentage of participants with Overall Success at the TOC Visit	Overall success is defined as the achievement of both clinical success and microbiological success	TOC visit (7-14 days after EOT)
Ceftobiprole Plasma Concentration		Day 3

Collaborators and Investigators

• Sponsor: ISTx, LLC
• Collaborators: Innoviva Specialty Therapeutics

Study record dates

Study Major Dates

• Study Start (Estimated): October 30, 2026
• Primary Completion (Estimated): October 31, 2028
• Study Completion (Estimated): October 31, 2028

Study Registration Dates

• First Submitted: October 19, 2026
• First Submitted That Met QC Criteria: November 4, 2026
• First Posted (Actual): November 10, 2026

Study Record Updates

• Last Update Posted (Actual): November 10, 2026
• Last Update Submitted That Met QC Criteria: November 4, 2026
• Last Verified: November 1, 2026

More Information

Terms related to this study

• Keywords: Pediatric; Phase 3; Ceftobiprole; Staphylococcus aureus bacteremia; Right-sided infective endocarditis; Standard-of-care
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Heart Diseases; Infections; Bacterial Infections; Bacterial Infections and Mycoses; Cardiovascular Infections; Endocarditis; Endocarditis, Bacterial; Health Services Administration; Health Care Quality, Access, and Evaluation; Quality of Health Care; Quality Indicators, Health Care; Standard of Care; ceftobiprole medocaril
• Other Study ID Numbers: CSBAL5788-2025-002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No