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Phase 3 N=18 Treatment

Extension Study for Patients Entered Into Study Infacort 003

Adrenal Insufficiency

Enrolled (actual)
18
Serious AEs
16.7%
Results posted
Sep 2019
Primary outcome: Primary: Incidence of Serious Adverse Events (SAEs) and Adverse Events (AEs) — 193; 9; 0; 42 adverse events

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
Infacort® (Drug)
Age
Pediatric · 0+ yrs
Sex
All
Sponsor
Neurocrine UK Limited
Primary completion
Aug 2018

Outcome Measures

OutcomeResultp-value
PRIMARY
Incidence of Serious Adverse Events (SAEs) and Adverse Events (AEs)
193; 9; 0; 42; 151
SECONDARY
Growth Velocity
0.6343; 0.9214
SECONDARY
Cortisol Levels
43.329; 22.022
SECONDARY
Number of Participants Exhibiting a Change in Tanner Development Stage
1

Summary

A Phase 3, open-label, single-group, non-randomised, observational study of the safety and biochemical disease control of Infacort® in neonates, infants and children with adrenal insufficiency and congenital adrenal hyperplasia who had completed study Infacort 003. All subjects who had satisfactorily completed study Infacort 003 were offered the opportunity to take part in Infacort 004.

Eligibility Criteria

Inclusion Criteria

Subjects successfully completing study Infacort 003, whose inclusion criteria were:

  • Male and female children less than 6 years of age.
  • A diagnosis of adrenal insufficiency (AI) as confirmed by an inappropriately low cortisol usually with other supporting tests.
  • Receiving appropriate adrenocortical replacement therapy (hydrocortisone with/without fludrocortisone).
  • Adequately hydrated and nourished. In addition, the parents/carers must be able to understand and give written Informed Consent for this extension study.

Exclusion Criteria

  • Clinically evident acute AI (adrenal crisis) (Note: the subject can be re-evaluated for eligibility once the episode is over)
  • Inability of the child to take oral therapy
  • Subjects with clinical signs of acute infection or fever on inclusion (Note: the subject can be re-evaluated for eligibility once the episode is over)
  • Any surgical or medical condition that in the opinion of the Investigator may place the subject at higher risk from his/her participation in the study
  • Parents/carers of subjects unwilling to consent to saving and propagation of pseudonymised medical data for study reasons
  • Subjects who are in a dependent relationship with the Investigator or the Sponsor
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02733367). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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