Adrenal InsufficiencyPhase 3NCT02733367
Extension Study for Patients Entered Into Study Infacort 003
A Phase 3 study testing Infacort® in people with Adrenal Insufficiency, run by Neurocrine UK Limited at 1 site in Germany.
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. Read more about this study
- Who is it for?
- Ages 1 month to 6 years
- Conditions the study is for
- Adrenal Insufficiency
- What is being tested?
- Infacort®
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteGermany
- Study dates
- 4 Mar 2016to 10 Aug 2018
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
Read the 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.
- Conditions
- Adrenal Insufficiency
- Being tested
- Infacort®
- Medical subject headings
- Adrenal Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Infacort®Drug
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).
| Study group | Role | What they receive |
|---|---|---|
| Infacort | Experimental | Infacort® granules Receives: Drug: Infacort® |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 29 months
Secondary outcomes (3)
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.
Measured: 29 months
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).
Measured: 29 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).
Measured: 29 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2016 (estimated)
- Study start4 Mar 2016
- Primary completion10 Aug 2018
- Study completion10 Aug 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Adrenal InsufficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurocrine UK Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Wiegand, Principal investigator, Charite University, Berlin, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02733367
- Sponsor's protocol ID
- Infacort 004
- Status last verified
- Sep 2026
- First submitted
- 16 Mar 2016
- Last updated
- 2 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02733367
- Acronym / sponsor ID
- Infacort 004
- Conditions
- Adrenal Insufficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Neurocrine UK LimitedIndustry (pharma, biotech, devices)
- Study start
- 4 Mar 2016 (actual)
- Primary completion
- 10 Aug 2018 (actual)
- Study completion
- 10 Aug 2018 (actual)
- First posted
- 11 Apr 2016 (estimated)
- Results
- Posted 13 Sep 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Infacort | Experimental | Drug: Infacort®Infacort® granules |
Who may qualify
- Ages
- 1 month to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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). | Drug | Infacort |
Primary outcome
Incidence of Serious Adverse Events (SAEs) and Adverse Events (AEs)
Time frame: 29 months
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.
3 secondary outcomes
Growth Velocity
Time frame: 29 months
Cortisol Levels
Time frame: 29 months
Number of Participants Exhibiting a Change in Tanner Development Stage
Time frame: 29 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Adrenal Insufficiency
RecruitingObservational study
Evaluation of Children With Endocrine and Metabolic-Related Conditions
Adrenal Insufficiency, Growth Disorder
Recruiting
Clinical Study on Circadian Genes Dysregulation in Patients With Glucocorticoid Disorders
Cushing Syndrome, Adrenal Insufficiency
RecruitingObservational study
Prevalence of Adrenal Insufficiency and Associated Factors in Rheumatoid Arthritis Patients Using Low-dose Corticosteroids.
Reumatoid Arthritis, Corticosteroids Adverse Reaction
Recruiting
Home Exercise for Individuals With Neurodegenerative Disease
Neurodegenerative Diseases, Leukodystrophy
Active, not recruitingObservational study
Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Wiegand principal investigator
Charite University, Berlin, Germany
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Infacort® in people with Adrenal Insufficiency, run by Neurocrine UK Limited at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 Sep 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Serious Adverse Events (SAEs) and Adverse Events (AEs) (time frame: 29 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2026.