Treatment of Adrenal Insufficiency in Children
Condition: Adrenal Insufficiency
MeSH terms: Adrenal Insufficiency
Study type: Interventional
Phase: Phase 3

Summary: 
      The study will be conducted in a total of 24 subjects before their 6th birthday, requiring
      replacement therapy for adrenal insufficiency due to either CAH, primary adrenal failure or
      hypopituitarism.

      The study will consist of three consecutive cohorts. Cohort 1 will include 12 subjects aged
      between 2 and < 6 years. If no safety concerns emerge, then 6 subjects aged 28 days to <2
      years will be enrolled (Cohort 2). A review of accumulated data will be undertaken and only
      if again no safety concerns emerge, then 6 neonates aged from birth to <28 days will be
      enrolled (Cohort 3).

      The decision to continue after each cohort will be based on the recommendation of an
      Independent Data Monitoring Committee (IDMC).
    

Detailed description: 
      This is a Phase 3, open label, single centre study of Infacort® in neonates, infants and
      children less than 6 years of age with adrenal insufficiency. The study will be conducted in
      a total of 24 subjects, before their 6th birthday, requiring replacement therapy for adrenal
      insufficiency due to either CAH, primary adrenal failure or hypopituitarism. Due to the rare
      occurrence of adrenal insufficiency it is expected that for the third cohort of this study
      (neonates) only subjects with CAH will be recruited.

      The study will consist of three consecutive parts. Cohort 1 will include 12 subjects aged
      between 2 and < 6 years. If no safety concerns emerge, then 6 subjects aged 28 days to <2
      years will be enrolled (Cohort 2). A review of accumulated data will be undertaken and only
      if again no safety concerns emerge, then 6 neonates aged from birth to <28 days will be
      enrolled (Cohort 3).

      The decision to continue after each cohort will be based on the recommendation of an
      Independent Data Monitoring Committee (IDMC).

      The study will consist of a screening visit (Visit 1 performed as a minimum the day before
      the intake of study drug), one treatment visit (Visit 2, Day 1), a follow-up visit (Visit 3)
      one to three days after intake of study drug (Day 2, Day 3 or Day 4) and a follow-up
      telephone call (Visit 4) 7 - 10 days after intake of study drug. Study completion evaluation
      will be performed at Visit 3. Parents/ carers will have at least 1 night to consider
      participation of their child before completing written informed consent. Children aged 3 - 6
      years will be informed about their involvement in the study in the presence of their
      parents/carers.

      All subjects will receive their standard treatment including fludrocortisone other than the
      dose of hydrocortisone that is to be substituted by Infacort®.

      Subjects who meet the eligibility criteria at screening (Visit 1) will attend for Visit 2 at
      a suitable time before the next planned dose of hydrocortisone is due. Subjects may have
      insertion of an intravenous cannula (with suitable local anaesthesia) allowing blood samples
      to be taken as well as their routine clinical samples (where required) prior to their next
      dose of hydrocortisone given as Infacort®. If a cannula is not used, direct venous sampling
      may be carried out instead. After all planned study procedures have been completed the
      subjects will go home and will return one to three days later for the follow-up assessments
      (Visit 3). A follow-up telephone call (Visit 4) 7 - 10 days after intake of study drug will
      be undertaken.
    

Intervention name(s): Infacort®
Intervention type(s): Drug
Intervention description(s): dry granule formulation of hydrocortisone
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment