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Intravenous and Oral Fosfomycin in Hospitalised Neonates With Clinical Sepsis

Phase 2
Completed
Conditions
Neonatal SEPSIS
Interventions
Drug: Standard of Care + Fosfomycin
Procedure: PK
Procedure: Analysis of Bacterial Isolates
Registration Number
NCT03453177
Lead Sponsor
Drugs for Neglected Diseases
Brief Summary

Neonatal sepsis has a high risk of morbidity and mortality. The current WHO and national guidelines recommend antibiotics to which resistance is reported in neonatal populations, although the available data is limited. Research on alternative empirical regimens for neonatal sepsis which are affordable, safe and cost-effective, with a step-down oral option, is needed. AMR is an issue of global public health concern and is one of the WHO's global health priority areas. Understanding the benefits, risks, MIC capacity and PK of fosfomycin will influence global policy on the case management of neonates with sepsis in Kenya and international settings.

Detailed Description

Antimicrobial resistance (AMR) has become a major issue in global health. Despite progress in the reduction of under 5 mortality rates in recent decades, the proportion of neonatal deaths occurring within this age group has increased, with almost one quarter of all neonatal deaths occurring due to serious bacterial infection. Common bacteria causing neonatal sepsis are now exhibiting widespread resistance to several classes of antibiotics. There is an urgent need to discover new, effective treatments and re-evaluate existing therapeutic agents to treat infections potentially caused by multi-drug resistant (MDR) pathogens. Gram-negative bacteria (GNB) predominate as the cause of neonatal sepsis, and are increasingly associated with high rates of resistance to the currently recommended WHO empirical therapy regimen of ampicillin/penicillin and gentamicin. There is therefore a need to develop an updated empiric regimen with improved efficacy in the context of increasing MDR sepsis in neonates. New antimicrobials under development will be expensive once licensed, and there are currently virtually no planned trials to assess their efficacy in neonates in low- and middle-income countries (LMICs).

One potential strategy is utilising an existing off-patent (and therefore affordable) antibiotic available in intravenous and oral formulations - fosfomycin. Fosfomycin has a wide spectrum of activity against Gram-positive and Gram-negative bacteria causing neonatal sepsis. It is mainly used for resistant urinary tract infections in adults, but has licenced neonatal and paediatric doses in Europe (though dosing regimens vary between countries). Both oral and IV formulations are available. A large clinical trial to assess the efficacy of a fosfomycin plus an aminoglycoside combination (compared to the current WHO recommended ampicillin and gentamicin) is anticipated, including sites in Kenya. The ultimate aim is for fosfomycin to be included in the WHO Essential Medicines List for children (EMLc) and be available for use in developing countries, where rates of resistance to ampicillin and gentamicin have been estimated at over 40%. The first steps before this trial are to clarify the pharmacokinetics (PK) and safety profile of fosfomycin in neonates, as well as generating further information regarding local patterns of bacterial susceptibility to fosfomycin. The aim of this study is to fulfil both these steps. Fosfomycin (IV and oral) PK will be investigated among 60 babies admitted to hospital and being treated for presumed sepsis; administered alongside the standard antibiotics. Another 60 babies receiving standard treatment only (without PK sampling) will be monitored in the same way to compare adverse events. In the laboratory at CGMR-C, previously archived bacterial isolates will be tested for their sensitivity to fosfomycin.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
120
Inclusion Criteria
  • Age 0 to 28 days inclusive
  • Weight >1500g
  • Born (an estimated) >34 weeks gestation (calculated as per the Ballard Maturational Assessment)
  • Admitted to hospital and eligible to receive IV antibiotics, according to national guidelines
Exclusion Criteria
  • Baseline sodium level >= 150mmol/L
  • Baseline creatinine >= 150 micromol/L
  • Presenting with severe (grade 3) Hypoxic Ischaemic Encephalopathy (HIE), defined as per Sarnat and Sarnat as a stuporous, flaccid infant (with or without seizure activity) with suppressed brainstem and autonomic functions and absent reflexes
  • Requiring cardiopulmonary resuscitation on admission
  • Jaundice requiring exchange transfusion
  • Admitted as a transfer after an overnight inpatient stay at another hospital
  • Known allergy or contraindication to fosfomycin
  • A specific clinical indication for another class of antibiotic (other than the nationally recommended standard-of-care)
  • More than 4 hours after initiating ampicillin plus gentamicin (one dose), which allows for administration of these first-line antibiotics not to be delayed by study procedures
  • Concurrent participation in another clinical trial
  • Attending clinician's judgement that the child is so severely ill that adequate communication about the study with the parent or legal guardian is not possible.
  • Not planning to remain resident in the County for the next 28 days.
  • Lack of consent

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Standard of Care plus FosfomycinStandard of Care + FosfomycinFosfomycin will initially be administered IV for at least 48 hours together with standard care (ampicillin + gentamicin). Then, once babies are tolerating oral feeds and clinically improved, fosfomycin will be changed to oral administration to complete a total of 7 days of fosfomycin (or until the baby is discharged).
Standard of Care plus FosfomycinPKFosfomycin will initially be administered IV for at least 48 hours together with standard care (ampicillin + gentamicin). Then, once babies are tolerating oral feeds and clinically improved, fosfomycin will be changed to oral administration to complete a total of 7 days of fosfomycin (or until the baby is discharged).
Standard of Care plus FosfomycinAnalysis of Bacterial IsolatesFosfomycin will initially be administered IV for at least 48 hours together with standard care (ampicillin + gentamicin). Then, once babies are tolerating oral feeds and clinically improved, fosfomycin will be changed to oral administration to complete a total of 7 days of fosfomycin (or until the baby is discharged).
Primary Outcome Measures
NameTimeMethod
Pharmacokinetic disposition and absorption parameters of IV and oralParticipants will be followed for the duration of enrolment, an expected average of 7 days

Fosfomycin Oral Bioavailability (F)

Pharmacokinetic disposition and absorption parameters of IV and oral fosfomycin in neonates with clinical sepsisParticipants will be followed for the duration of enrolment, an expected average of 7 days

Fosfomycin Clearance (CL)

Secondary Outcome Measures
NameTimeMethod
Difference between the groups in mean 48-hour plasma sodium concentrations48 hours

Biochemistry will be checked at 48 hours for participants in both groups

Difference between the groups in mean 7-day plasma sodium concentrations7 days

Biochemistry will be checked at 7 days for participants in both groups

Difference between groups in the rate of adverse events (any grade) to 28 days after enrolment in the studyfrom patient randomization to visit D28

Neonates will be reviewed every day by study clinicians, working together with the hospital team. All adverse events will be documented and reported in both arms.

Trial Locations

Locations (1)

KEMRI / Wellcome Trust Research Programme

🇰🇪

Kilifi, Kenya

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