Treatment of Adrenal Insufficiency in Children

• Status: Completed
• Trial end date: 2016-07-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Diurnal Limited
• Collaborator: TAIN (Treatment of Adrenal Insufficiency in Neonates) consortium

Criteria

Inclusion Criteria:

1. Male and female children less than 6 years of age.

2. A diagnosis of adrenal insufficiency as confirmed by an inappropriately low cortisol usually with other supporting tests.

3. Receiving appropriate adrenocortical replacement therapy (hydrocortisone with/without fludrocortisone).

4. Adequately hydrated and nourished.

5. Ability of parents/carers to understand and give written Informed Consent

Exclusion Criteria:

1. Clinically evident acute adrenal insufficiency (adrenal crisis).

2. Inability of the child to take oral therapy.

3. Concomitant therapy (other than that required to treat adrenal insufficiency, Vitamin D, Fluoride, Thyroxine and growth hormone).

4. Subjects with clinical signs of acute infection or fever on Day 1.

5. Any surgical or medical condition which in the opinion of the investigator may place the subject at higher risk from his/her participation in the study.

6. Parents/carers of subjects unwilling to consent to saving and propagation of pseudonymised medical data for study reasons.

7. Subjects who are dependent on the investigator or the sponsor.