Extension Study for Patients Entered Into Study Infacort 003

Adrenal Insufficiency Clinical Trial

Official 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02733367
• Other study ID #: Infacort 004
• Status: Completed
• Phase: Phase 3
• Start date: March 4, 2016
• Est. completion date: August 10, 2018

Study information

• Verified date: October 2019
• Source: Diurnal Limited
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

A Phase 3, open-label, single-group, non-randomised, observational study of the safety and biochemical disease control of Infacort® in neonates, infants and children with adrenal insufficiency and congenital adrenal hyperplasia who had completed study Infacort 003. All subjects who had satisfactorily completed study Infacort 003 were offered the opportunity to take part in Infacort 004.

Description:

A Phase 3, open-label, single-group, non-randomised, observational study of the safety and biochemical disease control of Infacort® in neonates, infants and children with AI who had completed study Infacort 003 (EudraCT number 2014-002265-30). All subjects who had satisfactorily completed study Infacort 003 wiere offered the opportunity to participate in study Infacort 004 at or after their final visit of study Infacort 003. Subjects received the usual clinically-appropriate dose (since bioequivalence has been demonstrated with conventional hydrocortisone), as determined by the Investigator, which was administered according to usual clinical practice - generally 3 or 4 times a day. Subjects could continue to be treated in this study until they met the study withdrawal criteria, until Infacort® was commercially available locally (which has now been achieved), or until the Sponsor decided to discontinue the study.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 18
• Est. completion date: August 10, 2018
• Est. primary completion date: August 10, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Month to 6 Years

Eligibility

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 adrenal insufficiency (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 for this extension study.

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

Related Conditions & MeSH terms

• Adrenal Insufficiency

Intervention

Drug:
• Infacort®
Infacort® is a dry granule formulation of hydrocortisone stored in capsules available in different strengths (0.5, 1.0, 2.0 and 5.0 mg).

Locations

Country	Name	City	State
Germany	Charité-Universitätsmedizin Berlin, CVK	Berlin

Sponsors (1)

Lead Sponsor	Collaborator
Diurnal Limited

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Serious Adverse Events (SAEs) and Adverse Events (AEs)	The primary endpoint was the nature and occurrence of serious adverse events (SAEs) and adverse events (AEs) observed throughout the study. AEs were recorded from the time of the first intake of Infacort until the final visit.	29 months
Secondary	Growth Velocity	Growth velocity standard deviation score (SDS). Body height/length (cm) was obtained at each visit by specially trained paediatric endocrine nurses or physicians using standard calibrated auxological methods.	29 months
Secondary	Cortisol Levels	Cortisol levels measured from dried blood spots. The dried blood spots were analysed for multi-steroids, including cortisol (all subjects). Blood spot absolute laboratory values for the safety population are presented. A dried blood spot sample was collected at the initial and final visits, every month for the first 2 months of the study and thereafter every 6 months (unless required after 3 months).	29 months
Secondary	Number of Participants Exhibiting a Change in Tanner Development Stage	The Tanner Development Stage was assessed as an additional analysis in this study. All assessments (breast, genitalia, and pubic hair) were Grade 1 (pre-pubertal) at baseline, with only 1 subject (in Cohort 2) showing a change during the study. Subject 018 showed progression to Grade 2 in the pubic hair category (sparse, pigmented hair mainly on labia).	29 months