Back to resultsExtension Study for Patients Entered Into Study Infacort 003
Primary Purpose
Adrenal Insufficiency
Status
Completed
Phase
Phase 3
Locations
Germany
Study Type
Interventional
Intervention
Infacort®
Sponsored by
About this trial
This is an interventional treatment trial for Adrenal Insufficiency
Eligibility Criteria
Inclusion Criteria:
Subjects successfully completing study Infacort 003, whose inclusion criteria were:
- Male and female children less than 6 years of age.
- A diagnosis of adrenal insufficiency (AI) as confirmed by an inappropriately low cortisol usually with other supporting tests.
- Receiving appropriate adrenocortical replacement therapy (hydrocortisone with/without fludrocortisone).
- 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:
- Clinically evident acute AI (adrenal crisis) (Note: the subject can be re-evaluated for eligibility once the episode is over)
- Inability of the child to take oral therapy
- 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)
- 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
- Parents/carers of subjects unwilling to consent to saving and propagation of pseudonymised medical data for study reasons
- Subjects who are in a dependent relationship with the Investigator or the Sponsor
Sites / Locations
- Charité-Universitätsmedizin Berlin, CVK
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Infacort
Arm Description
Infacort® granules
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
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).
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).
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02733367
Brief Title
Extension Study for Patients Entered Into Study Infacort 003
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
Study Type
Interventional
2. Study Status
Record Verification Date
October 2019
Overall Recruitment Status
Completed
Study Start Date
March 4, 2016 (Actual)
Primary Completion Date
August 10, 2018 (Actual)
Study Completion Date
August 10, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Diurnal Limited
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief 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.
Detailed 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Adrenal Insufficiency
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
18 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Infacort
Arm Type
Experimental
Arm Description
Infacort® granules
Intervention Type
Drug
Intervention Name(s)
Infacort®
Intervention Description
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).
Primary Outcome Measure Information:
Title
Incidence of Serious Adverse Events (SAEs) and Adverse Events (AEs)
Description
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.
Time Frame
29 months
Secondary Outcome Measure Information:
Title
Growth Velocity
Description
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.
Time Frame
29 months
Title
Cortisol Levels
Description
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).
Time Frame
29 months
Title
Number of Participants Exhibiting a Change in Tanner Development Stage
Description
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).
Time Frame
29 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Month
Maximum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Subjects successfully completing study Infacort 003, whose inclusion criteria were:
Male and female children less than 6 years of age.
A diagnosis of adrenal insufficiency (AI) as confirmed by an inappropriately low cortisol usually with other supporting tests.
Receiving appropriate adrenocortical replacement therapy (hydrocortisone with/without fludrocortisone).
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:
Clinically evident acute AI (adrenal crisis) (Note: the subject can be re-evaluated for eligibility once the episode is over)
Inability of the child to take oral therapy
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)
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
Parents/carers of subjects unwilling to consent to saving and propagation of pseudonymised medical data for study reasons
Subjects who are in a dependent relationship with the Investigator or the Sponsor
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Wiegand
Organizational Affiliation
Charite University, Berlin, Germany
Official's Role
Principal Investigator
Facility Information:
Facility Name
Charité-Universitätsmedizin Berlin, CVK
City
Berlin
ZIP/Postal Code
13353
Country
Germany
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Extension Study for Patients Entered Into Study Infacort 003
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