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).
Phase:
Phase 3
Details
Lead Sponsor:
Diurnal Limited
Collaborator:
TAIN (Treatment of Adrenal Insufficiency in Neonates) consortium