A Phase 3 open-label study of Infacort® in neonates, infants and children less than 6 years of age with adrenal insufficiency.

• Register: EUCTR
• Last refreshed on: 3 July 2017
• Main ID: EUCTR2014-002265-30-DE
• Date of registration: 23/12/2014
• Prospective Registration: Yes
• Primary sponsor: Diurnal Limited
• Public title: A Phase 3 open-label study of Infacort® in neonates, infants and children less than 6 years of age with adrenal insufficiency.
• Scientific title: A Phase 3 open-label study of Infacort® in neonates, infants and children less than 6 years of age with adrenal insufficiency.
• Date of first enrolment: 19/02/2015
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-002265-30
• 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:
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 (according to AMG §40 (1) 3b).

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 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.



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
INN or Proposed INN: infacort
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
INN or Proposed INN: infacort
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
INN or Proposed INN: infacort
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
INN or Proposed INN: infacort
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 demonstrate significant absorption of hydrocortisone from the Infacort® preparation.

Primary end point(s): • • 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.

Secondary Objective: In children with adrenal insufficiency receiving a single dose of Infacort® to assess:

• The safety of Infacort®.
• The tolerability of Infacort® in the target population.
• The pharmacokinetics (PK) of Infacort® in the target population using a population PK approach.
• Palatability.

Timepoint(s) of evaluation of this end point: 6 h after IMP administration

Secondary Outcome(s)

Secondary end point(s): Secondary endpoints:
• Serum cortisol concentration up to 6 hours after intake of study drug as determined by the central laboratory.

• Palatability of the investigational product.

• PK parameters estimated in the population PK analysis.

Safety endpoints:
Safety endpoints will be any (serious or non-serious) adverse events and vital signs observed throughout the study. Events (clinical or laboratory) related to a lack of absorption of hydrocortisone from Infacort® will be considered as lack of efficacy and not as adverse events.

Timepoint(s) of evaluation of this end point: up to 6 h after IMP Administration

safety endpoint will be determined 6-8 h after IMP administration

Secondary ID(s)

Infacort003

Source(s) of Monetary Support

European Commission (TAIN-Project No: 281654)