Clinical Trial: Treatment of Adrenal Insufficiency in Children

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase 3 Open-label Study of Infacort® in Neonates, Infants and Children Less Than 6 Years of Age With Adrenal Insufficiency

Brief 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 Summary:

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 fludrocortison
Sponsor: Diurnal Limited

Current Primary Outcome: Serum Cortisol Concentration up to 240 Minutes [ Time Frame: 240 minutes ]

The primary endpoint will be the maximum levels of serum cortisol concentration up to 240 minutes after intake of study drug as determined by the central laboratory.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Serum Cortisol Concentration up to 6 Hours [ Time Frame: 240 minutes ]
    Serum cortisol concentration 240 minutes after intake of study drug as determined by the central laboratory
  • Subject Assessment of Taste of the Product [ Time Frame: 1 minute ]

    Palatability of the investigational product as determined by parent/carer responses to the following questions:

    Question 1: My child found swallowing easy. Question 2: My child showed a positive reaction after Infacort was given. Question 3: I would be happy to give my child Infacort in the future. Question 4: Overall, I would prefer Infacort for my child over the usual hydrocortisone medication.

  • Incidence of Serious Adverse Events (SAEs) and Adverse Events (AE) [ Time Frame: 7-10 days ]

    SAEs and AEs reported over the study period.

    N.B., Data will not be entered in this section as this is described within the Adverse Events section.



Original Secondary Outcome:

  • Serum Cortisol Concentration up to 6 Hours [ Time Frame: 6 hours ]
    Serum cortisol concentration up to 6 hours after intake of study drug as determined by the central laboratory
  • Subject Assessment of Taste of the Product [ Time Frame: 1 minute ]
    Palatability of the investigational product
  • Incidence of Serious Adverse Events (SAEs) and Adverse Events (AE) [ Time Frame: 7-10 days ]
    SAEs and AEs reported over the study period


Information By: Diurnal Limited

Dates:
Date Received: March 16, 2016
Date Started: March 2015
Date Completion:
Last Updated: April 11, 2017
Last Verified: April 2017