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

• Register: EUCTR
• Last refreshed on: 28 February 2019
• Main ID: EUCTR2015-000458-40-DE
• Date of registration: 21/08/2015
• Prospective Registration: Yes
• Primary sponsor: Diurnal Limited
• Public title: 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
• Scientific title: 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 - Infacort004
• Date of first enrolment: 20/10/2015
• Target sample size: 24
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-000458-40
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Germany

Contacts

• Name: Dena Digweed
• Affiliation: Diurnal

• Name: Dena Digweed
• Affiliation: Diurnal

Key inclusion & exclusion 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:
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



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

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 hydrocortisone that is provided in appropriate unit dosage (0.5mg, 1.0mg, 2.0mg and 5mg)

Therapeutic area: Diseases [C] - Hormonal diseases [C19]

Intervention(s)

Product Name: infacort
Pharmaceutical Form: Granules
CAS Number: 50-23-7
Current Sponsor code: infacort
Other descriptive name: HYDROCORTISONE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 0.5-

Product Name: infacort
Pharmaceutical Form: Granules
CAS Number: 50-23-7
Current Sponsor code: infacort
Other descriptive name: HYDROCORTISONE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1-

Product Name: infacort
Pharmaceutical Form: Granules
CAS Number: 50-23-7
Current Sponsor code: infacort
Other descriptive name: HYDROCORTISONE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 2-

Product Name: infacort
Pharmaceutical Form: Granules
CAS Number: 50-23-7
Current Sponsor code: infacort
Other descriptive name: HYDROCORTISONE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-

Primary Outcome(s)

Main Objective: To gather data on the long-term safety of Infacort® in subjects completing study Infacort 003

Primary end point(s): • Nature and occurrence of SAEs and AEs observed throughout the study

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

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

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: the end of the study (2 years);

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

Secondary ID(s)

Infacort004

Source(s) of Monetary Support

European Commission (TAIN-Project No: 281654)

Ethics review

Status: Approved
Approval date:
Contact:

Results

• Results available: Yes
• Date Posted: 14/02/2019
• Date Completed: 10/08/2018
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-000458-40/results