Cystic fibrosis MedDRA version: 19.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Flucloxacillin 250mg/5ml Sugar-Free Powder for Oral Solution
Product Name: Flucloxacillin
Product Code: na
Pharmaceutical Form: Powder for oral solution
INN or Proposed INN: Flucloxacillin
Sponsors
Alder Hey Children's NHS Foundation Trust
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. A confirmed diagnosis of cystic fibrosis through one of the following three routes: - Two CF-causing mutations are identified. OR - One or no CF- causing mutations identified and a sweat chloride test result greater than 59 mmol/L. OR - Two CFTR mutations (not known CF-causing mutations) and a sweat chloride test result greater than 29 mmol/L. 2. Age 70 days or less. 3. Consent for inclusion on the national UK CF Registry. 4. Consent for inclusion in the CF START trial. Are the trial subjects under 18? yes Number of subjects for this age range: 480 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 0 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 1. An inconclusive diagnosis after newborn screening (NBS).* 2. A condition (non-CF) that, in the opinion of the recruiting investigator will impact on the long-term management and outcome of a participant with CF.** 3. Previous growth of PsA from respiratory culture. 4. Infants with a history of hypersensitivity to ß-lactam antibiotics (e.g. penicillins) or excipients 5. Infants with a history of flucloxacillin associated jaundice/hepatic dysfunction. *Infants with an inconclusive diagnosis after NBS (termed ‘CF Screen Positive Inconclusive Diagnosis (CFSPID)’) should not receive standard CF care and should not be recruited into CF START (Munck et al 2015). The two situations that result in a diagnosis of CFSPID after NBS are; • Two CFTR mutations recognised, one or both of which are not characterised as CF-causing and the sweat chloride is less than 30 mmol/L • The sweat chloride is repeatedly between 30-59 mmol/L and only one or no CFTR mutations are recognised **Significant non-CF conditions might include chromosomal abnormality (for example, Down syndrome), cerebral palsy, chronic lung disease (oxygen requirement) following pre-term birth and other significant congenital anomalies (for example, severe cardiac disease, tracheo-oesophageal fistula, diaphragmatic hernia).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate that infants on anti-staphylococcal antibiotic prophylaxis (“Prevent and Treat”) are not predisposed to earlier airway infection with Pseudomonas aeruginosa.;Secondary Objective: - To determine if anti-staphylococcal antibiotic prophylaxis (ASAP) improves respiratory function in pre-school children with CF - To determine the need for extra antibiotic treatment - To determine the number and type of respiratory culture taken during the trial period - To determine the number and proportion of respiratory cultures positive for Staphylococcus aureus (SA) - To determine the number and proportion of respiratory cultures positive for Pseudomonas aeruginosa (PsA) - To determine the number and proportion of respiratory cultures positive for other significant CF pathogens - To determine if there is evidence of chronic airway infection - To determine the frequency of hospital admission - To identify any adverse events - To determine nutritional status - To determine the costs to the NHS ;Primary end point(s): Age at first growth of Pseudomonas aeruginosa from respiratory culture collected as part of routine care;Timepoint(s) of evaluation of this end point: All routine clinical encounters to trial completion (age, 48 months) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): To determine the need for extra antibiotic treatment To determine the number and type of respiratory culture taken during the trial period To determine the number and proportion of respiratory cultures positive for Staphylococcus aureus (SA) To determine the number and proportion of respiratory cultures positive for Pseudomonas aeruginosa (PsA) To determine the number and proportion of respiratory cultures positive for other significant CF pathogens To determine if there is evidence of chronic airway infection To determine the frequency of hospital admission To identify any adverse events To determine nutritional status To determine the costs to the NHS ;Timepoint(s) of evaluation of this end point: All secondary endpoints detailed above will be recorded at all encounters to trial completion (age, 48 months). Apart from determining if ASAP improves respiratory function in pre-school children with CF which will be evaluated at trial visits between the age of 40-48 months. As well as determining nutritional status which will be evaluated at encounters between 40-48 months. | — |
Countries
United Kingdom
Contacts
Public ContactAbigail Bennett
MC- CTU
Outcome results
None listed