Congenital Adrenal HyperplasiaPhase 1NCT03718234
Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia
A Phase 1 study testing Subcutaneous hydrocortisone in people with Congenital Adrenal Hyperplasia and Hyperplasia, run by University of Minnesota at 1 site in United States.
Trial summary
This is an open-label, non-randomized, single arm proof-of concept feasibility trial comparing standard oral hydrocortisone (HC) treatment with a six-week intervention of interval bolus delivery (pulsatile) of subcutaneous (SQ) HC via infusion pump in children with congenital adrenal hyperplasia (CAH). Read more about this study
- Who is it for?
- Ages 4 to 18 years
- Conditions the study is for
- Congenital Adrenal Hyperplasia, Hyperplasia, Adrenal Hyperplasia
- What is being tested?
- Subcutaneous hydrocortisone
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Jan 2019to 23 Feb 2023
- Phase
- Phase 1
- 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 4 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Children 4 - 18 years of age.
- Classic congenital adrenal hyperplasia (CAH) as confirmed by hormonal and molecular testing.
- Patients who have been on the same HC dosing regimen for 1 month
Who cannot join
- Patients with non-classic CAH.
- Patients on:
- Dexamethasone
- Prednisone, or
- inhaled steroids.
- Patients with body surface areas under 1m2 or over 2m2
- Non-English speaking patients
Trial locations and contacts
1 site in 1 country.
United States1 site
Prism Clinical Research
Minneapolis, Minnesota, 55455
About this study
Official title: Interval Bolus Delivery of Subcutaneous Hydrocortisone Via Infusion Pump in Children With Congenital Adrenal Hyperplasia
This is an open-label, non-randomized, single arm proof-of concept feasibility trial comparing standard oral hydrocortisone (HC) treatment with a six-week intervention of interval bolus delivery (pulsatile) of subcutaneous (SQ) HC via infusion pump in children with congenital adrenal hyperplasia (CAH). Eight children, ages 4-18 yrs, will have 24-hr pharmacokinetic and pharmacodynamic (PKPD) profiles of cortisol, 17- hydroxyprogesterone (17OHP) and androstenedione (A4) concentrations while on standard oral hydrocortisone therapy (admission 1, week 1); during a safety check using a subcutaneous hydrocortisone pump (admission 2, week 7); and after 6 weeks of SQHC pump treatment (admission 3, week 14). A pharmacokinetic and pharmacodynamic analysis will be used to compare cortisol, 17OHP and A4 exposures between standard oral HC therapy (week 1) with SQHC pump treatment (week 14). Funding Source - FDA OOPD
- Conditions
- Congenital Adrenal Hyperplasia, Hyperplasia, Adrenal Hyperplasia, Congenital Disorders, Adrenocortical Hyperfunction, Disorders of Sex Development
- Being tested
- Subcutaneous hydrocortisone
- Keywords
- adrenal gland diseases, cortisol succinate, hydrocortisone, hyperplasia
- Medical subject headings
- Adrenal Hyperplasia, Congenital, Hyperplasia, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Adrenocortical Hyperfunction, Disorders of Sex Development, Urogenital Abnormalities, Genetic Diseases, Inborn, Steroid Metabolism, Inborn Errors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Subcutaneous hydrocortisoneDrug
Also called Hydrocortisone delivered via subcutaneous pump
Pulsatile subcutaneous hydrocortisone administered in 8 doses per day via SQ infusion pump for 6 weeks
| Study group | Role | What they receive |
|---|---|---|
| Subcutaneous Hydrocortisone via Infusion Pump | Experimental | This is a single arm study. Upon enrollment each participant was admitted for a 24-hr PKPD profile of cortisol, 17OHP and A4 concentrations while on standard oral hydrocortisone therapy (admission 1, week 1). Participant continued on stable oral HC for six weeks. On admission 2, week 7, participant was admitted SQHC pump placement and a safety check was performed to verify absorption of HC was adequate. This consisted of an identical 24-hr PKPD design as used in admission 1. The participants returned to their standard oral HC dosing for 2 weeks while the PKPD profiles from the SQHC pump were being evaluated. The SQHC pump was placed at week 9 and then sent home on the pump. .After 6 weeks of SQHC pump treatment (admission 3, week 14) the participants were admitted and the same 24-hour PKPD design as admissions 1 and 2 profiles was conducted. The subjects were then discharged on their standard oral HC therapy and were followed for an additional 6 weeks Receives: Drug: Subcutaneous hydrocortisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cortisol Exposure
24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).
Measured: Week 1
Cortisol Exposure
The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)
Measured: Week 14
17-OHP Exposure
24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).
Measured: Week 1
17-OHP Exposure
The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)
Measured: Week 14
Androstenedione (A4) Exposure
24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).
Measured: Week 1
Androstenedione (A4) Exposure
The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)
Measured: Week 14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2018
- Study start1 Jan 2019
- Primary completion23 Feb 2023
- Study completion23 Feb 2023
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Congenital Adrenal HyperplasiaStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is a a single arm non-randomized crossover study design.
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Kyriaki Sarafoglou, MD, Principal investigator, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03718234
- Sponsor's protocol ID
- PEDS-2018-26475
- Other IDs
- FD-R-6100
- Status last verified
- Jun 2025
- First submitted
- 11 Oct 2018
- Last updated
- 25 Jun 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Ramirez Alcantara J, Halper A. Adrenal insufficiency updates in children. Curr Opin Endocrinol Diabetes Obes. 2021 Feb 1;28(1):75-81. doi: 10.1097/MED.0000000000000591.
PubMed 33278125
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Interval Bolus Delivery of Subcutaneous Hydrocortisone Via Infusion Pump in Children With Congenital Adrenal Hyperplasia
- ClinicalTrials.gov ID
- NCT03718234
- Acronym / sponsor ID
- PEDS-2018-26475
- Conditions
- Congenital Adrenal Hyperplasia, Hyperplasia, Adrenal Hyperplasia, Congenital Disorders, Adrenocortical Hyperfunction, Disorders of Sex Development
- Keywords
- adrenal gland diseases, cortisol succinate, hydrocortisone, hyperplasia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- University of MinnesotaUniversity, hospital or other
- Study start
- 1 Jan 2019 (actual)
- Primary completion
- 23 Feb 2023 (actual)
- Study completion
- 23 Feb 2023 (actual)
- First posted
- 24 Oct 2018 (actual)
- Results
- Posted 25 Jun 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open-label, non-randomized, single arm proof-of concept feasibility trial comparing standard oral hydrocortisone (HC) treatment with a six-week intervention of interval bolus delivery (pulsatile) of subcutaneous (SQ) HC via infusion pump in children with congenital adrenal hyperplasia (CAH). Eight children, ages 4-18 yrs, will have 24-hr pharmacokinetic and pharmacodynamic (PKPD) profiles of cortisol, 17- hydroxyprogesterone (17OHP) and androstenedione (A4) concentrations while on standard oral hydrocortisone therapy (admission 1, week 1); during a safety check using a subcutaneous hydrocortisone pump (admission 2, week 7); and after 6 weeks of SQHC pump treatment (admission 3, week 14). A pharmacokinetic and pharmacodynamic analysis will be used to compare cortisol, 17OHP and A4 exposures between standard oral HC therapy (week 1) with SQHC pump treatment (week 14). Funding Source - FDA OOPD
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is a a single arm non-randomized crossover study design.
| Arm | Type | Receives |
|---|---|---|
| Subcutaneous Hydrocortisone via Infusion Pump | Experimental | Drug: Subcutaneous hydrocortisoneThis is a single arm study. Upon enrollment each participant was admitted for a 24-hr PKPD profile of cortisol, 17OHP and A4 concentrations while on standard oral hydrocortisone therapy (admission 1, week 1). Participant continued on stable oral HC for six weeks. On admission 2, week 7, participant was admitted SQHC pump placement and a safety check was performed to verify absorption of HC was adequate. This consisted of an identical 24-hr PKPD design as used in admission 1. The participants returned to their standard oral HC dosing for 2 weeks while the PKPD profiles from the SQHC pump were being evaluated. The SQHC pump was placed at week 9 and then sent home on the pump. .After 6 weeks of SQHC pump treatment (admission 3, week 14) the participants were admitted and the same 24-hour PKPD design as admissions 1 and 2 profiles was conducted. The subjects were then discharged on their standard oral HC therapy and were followed for an additional 6 weeks |
Who may qualify
- Ages
- 4 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Children 4 - 18 years of age.
- Classic congenital adrenal hyperplasia (CAH) as confirmed by hormonal and molecular testing.
- Patients who have been on the same HC dosing regimen for 1 month
Key exclusion criteria (7 listed)
- Patients with non-classic CAH.
- Patients on:
- Patients with body surface areas under 1m2 or over 2m2
- Non-English speaking patients
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 |
|---|---|---|
| Subcutaneous hydrocortisoneAlso: Hydrocortisone delivered via subcutaneous pumpPulsatile subcutaneous hydrocortisone administered in 8 doses per day via SQ infusion pump for 6 weeks | Drug | Subcutaneous Hydrocortisone via Infusion Pump |
Primary outcome
Cortisol Exposure
Time frame: Week 1
24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).
Cortisol Exposure
Time frame: Week 14
The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)
17-OHP Exposure
Time frame: Week 1
24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).
17-OHP Exposure
Time frame: Week 14
The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)
Androstenedione (A4) Exposure
Time frame: Week 1
24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).
Androstenedione (A4) Exposure
Time frame: Week 14
The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33278125Ramirez Alcantara J, Halper A. Adrenal insufficiency updates in children. Curr Opin Endocrinol Diabetes Obes. 2021 Feb 1;28(1):75-81. doi: 10.1097/MED.0000000000000591.
- Individual participant dataSharing planned: No
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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.
Kyriaki Sarafoglou, MD principal investigator
University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Subcutaneous hydrocortisone in people with Congenital Adrenal Hyperplasia and Hyperplasia, run by University of Minnesota at 1 site in United States.
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 3 inclusion and 7 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 25 Jun 2025. 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 United States. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 25 Jun 2025). 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?
Cortisol Exposure (time frame: Week 1). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03718234, which also lists 1 linked publication.
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 25 Jun 2025.