Adrenal Insufficiency (AI) in children is most commonly due to Congenital Adrenal Hyperplasia (CAH) and results in cortisol deficiency with or without aldosterone deficiency and androgen excess. Current standard treatment in neonates is unsatisfactory, as unlicensed and crushed adult dosage formulations (Hydrocortisone tablets,10 mg) are used. Infacort® is a new paediatric and neonatal formulation of
Conditions
Interventions
Product Name: infacort
Pharmaceutical Form: Granules
CAS Number: 50-23-7
Current Sponsor code: infacort
Other descriptive name: HYDROCOR
Sponsors
Diurnal Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects successfully completing study Infacort 003, whose inclusion criteria were: 1. Male and female children less than 6 years of age. 2. A diagnosis of AI 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. In addition, the parents/carers must be able to understand and give written Informed Consent (according to AMG §40 (1) 3b) for this extension study. Are the trial subjects under 18? yes Number of subjects for this age range: 24 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Clinically evident acute AI (adrenal crisis) (Note: the subject can be re-evaluated for eligibility once the episode is over) 2. Inability of the child to take oral therapy 3. 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) 4. 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 5. Parents/carers of subjects unwilling to consent to saving and propagation of pseudonymised medical data for study reasons 6. Subjects who are in a dependent relationship with the Investigator or the Sponsor
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: the final Evaluation will be done after the end of the study (2 years); after each visit (3 months Intervall) the safety Information were updated ;Main Objective: To gather data on the long-term safety of Infacort® in subjects completing study Infacort 003; Secondary Objective: •Growth velocity (growth velocity standard deviation score [SDS]) •Cortisol (all subjects) and adrenal androgen levels (17 OHP, A4, testosterone) in CAH subjects only ;Primary end point(s): • Nature and occurrence of SAEs and AEs observed throughout the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Growth velocity (growth velocity standard deviation score [SDS]) • Cortisol (all subjects) and adrenal androgen levels (17 OHP, A4, testosterone) in CAH subjects only ; Timepoint(s) of evaluation of this end point: the end of the study (2 years); | — |
Countries
Germany
Contacts
Public ContactDena Digweed
Diurnal
Outcome results
None listed