Open-label, long-term follow-up of safety and biochemical disease control of Infacort® in neonates, infants and children with congenital adrenal hyperplasia and adrenal insufficiency previously enrolled in the Infacort 003 study
- Registration Number
- EUCTR2015-000458-40-DE
- Lead Sponsor
- Diurnal Limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 18
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
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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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;<br> Secondary Objective: •Growth velocity (growth velocity standard deviation score [SDS]) <br> <br> •Cortisol (all subjects) and adrenal androgen levels (17 OHP, A4, testosterone) in CAH subjects only<br> ;Primary end point(s): •Nature and occurrence of SAEs and AEs observed throughout the study
- Secondary Outcome Measures
Name Time Method <br> Secondary end point(s): •Growth velocity (growth velocity standard deviation score [SDS]) <br> •Cortisol (all subjects) and adrenal androgen levels (17 OHP, A4, testosterone) in CAH subjects only <br> ;<br> Timepoint(s) of evaluation of this end point: the end of the study (2 years); <br> <br> <br>