ClinicalTrials.gg
CompletedNCT03718234Updated Jun 25, 2025Results posted

Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia

A Phase 1 interventional study of Subcutaneous hydrocortisone in Congenital Adrenal Hyperplasia, Hyperplasia and Adrenal Hyperplasia, sponsored by University of Minnesota. Completed at 1 site in United States. Open to participants aged 4 Years to 18 Years. Per ClinicalTrials.gov, last updated 2025-06-25.

Sponsored by University of Minnesota · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
11
Allocation
Not applicable
Ages
4 Years to 18 Years
Sex
All
01

Study 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). 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

02

Conditions studied

  • Congenital Adrenal Hyperplasia
  • Hyperplasia
  • Adrenal Hyperplasia
  • Congenital Disorders
  • Adrenocortical Hyperfunction
  • Disorders of Sex Development
  • Urogenital Abnormalities
  • Genetic Diseases, Inborn
  • Steroid Metabolic Diseases, Inborn
  • Adrenal Gland Disease
  • Hydrocortisone

Keywords

  • adrenal gland diseases
  • cortisol succinate
  • hydrocortisone
  • hyperplasia
03

In context

Adrenal Hyperplasia, Congenital

101 studies on the registry are indexed under Adrenal Hyperplasia, Congenital; 28 are open to participants now.

This study's enrollment of 11 is below the median of 37 across 50 interventional studies indexed under Adrenal Hyperplasia, Congenital.

Browse Adrenal Hyperplasia, Congenital studies →

Lead sponsor

University of Minnesota is the lead sponsor of 1,216 studies on the registry; 197 are open to participants now.

Of its 136 completed or terminated interventional studies of FDA-regulated products, 95 (70%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
4 Years to 18 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • 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

Exclusion criteria

Exclusion Criteria:

  • Patients with non-classic CAH.
  • Patients on:

    • Dexamethasone
    • Prednisone, or
    • inhaled .
  • Patients with body surface areas under 1m2 or over 2m2
  • Non-English speaking patients
Terms in these criteria, in plain language(1)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
11 participants (actual)

Study arms

  • Experimental
    Subcutaneous Hydrocortisone via Infusion Pump

    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

    Drug: Subcutaneous hydrocortisone

Interventions

  • Pulsatile subcutaneous hydrocortisone administered in 8 doses per day via SQ infusion pump for 6 weeks

    Also known as: Hydrocortisone delivered via subcutaneous pump

06

What researchers measure

Primary outcomes

  1. Cortisol Exposure

    24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).

    Time frame: Week 1

  2. Cortisol Exposure

    The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)

    Time frame: Week 14

  3. 17-OHP Exposure

    24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).

    Time frame: Week 1

  4. 17-OHP Exposure

    The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)

    Time frame: Week 14

  5. Androstenedione (A4) Exposure

    24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).

    Time frame: Week 1

  6. Androstenedione (A4) Exposure

    The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)

    Time frame: Week 14

07

Results

Posted Jun 25, 2025

Participant flow

Participant flow — Overall Study
MilestoneSubcutaneous Hydrocortisone Via Infusion Pump
Started11
Completed8
Not completed3

Outcome measures

PrimaryCortisol Exposure

24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).

Time frame:
Week 1
Reported as:
Mean · hr*mcg/dL
Cortisol Exposure
hr*mcg/dLSubcutaneous Hydrocortisone Via Infusion Pump
Cortisol Exposure110 ± 9.96
PrimaryCortisol Exposure

The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)

Time frame:
Week 14
Reported as:
Mean · hr*mcg/dL
Cortisol Exposure
hr*mcg/dLSubcutaneous Hydrocortisone Via Infusion Pump
Cortisol Exposure118 ± 9.10
Primary17-OHP Exposure

24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).

Time frame:
Week 1
Reported as:
Mean · hr*ng/dL
17-OHP Exposure
hr*ng/dLSubcutaneous Hydrocortisone Via Infusion Pump
17-OHP Exposure46700 ± 19200
Primary17-OHP Exposure

The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)

Time frame:
Week 14
Reported as:
Mean · hr*ng/dL
17-OHP Exposure
hr*ng/dLSubcutaneous Hydrocortisone Via Infusion Pump
17-OHP Exposure26900 ± 16200
PrimaryAndrostenedione (A4) Exposure

24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).

Time frame:
Week 1
Reported as:
Mean · hr*ng/dL
Androstenedione (A4) Exposure
hr*ng/dLSubcutaneous Hydrocortisone Via Infusion Pump
Androstenedione (A4) Exposure3350 ± 2410
PrimaryAndrostenedione (A4) Exposure

The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)

Time frame:
Week 14
Reported as:
Mean · hr*ng/dL
Androstenedione (A4) Exposure
hr*ng/dLSubcutaneous Hydrocortisone Via Infusion Pump
Androstenedione (A4) Exposure1420 ± 968

Adverse events

Collected over 20 weeks. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Subcutaneous Hydrocortisone Via Infusion Pump0/11 (0%)0/11 (0%)9/11 (81.8%)
Standard Glucocorticoid Therapy0/11 (0%)0/11 (0%)9/11 (81.8%)
Most frequent other events
Showing 10 of 17
Most frequent other events
EventSubcutaneous Hydrocortisone Via Infusion PumpStandard Glucocorticoid Therapy
Stinging/Burning sensationGeneral disorders8/118/11
ItchingGeneral disorders5/115/11
ErythemaGeneral disorders4/114/11
ExcoriationGeneral disorders2/112/11
FeverGeneral disorders2/112/11
Skin rashGeneral disorders1/111/11
HivesGeneral disorders1/111/11
FatigueGeneral disorders1/111/11
HeadacheGeneral disorders1/111/11
Nasal congestionGeneral disorders1/111/11

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Subcutaneous Hydrocortisone Via Infusion Pump
<=18 years11
Between 18 and 65 years0
>=65 years0
Sex: Female, Male
Sex: Female, Male(Participants)Subcutaneous Hydrocortisone Via Infusion Pump
Female5
Male6
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Subcutaneous Hydrocortisone Via Infusion Pump
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American0
White10
More than one race1
Unknown or Not Reported0
08

Study locations

1 site
  • Prism Clinical Research
    Minneapolis, Minnesota 55455, United States
09

References and documents

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 documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: No

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from Jan 2019 to primary completion in Feb 2023, 49 months.

Results were first posted in Jun 2025, 2 years 4 months after primary completion.

How these figures are worked out, and which studies are left out of them.

Registered
Oct 11, 2018
First posted
Oct 24, 2018
StartActual
Jan 1, 2019
Primary completionActual
Feb 23, 2023
CompletionActual
Feb 23, 2023
Results first posted
Jun 25, 2025
Last update
Jun 25, 2025
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Jun 25, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03718234
Responsible party
Sponsor
First posted
Oct 24, 2018
Start date
Jan 1, 2019
Primary completion
Feb 23, 2023
Completion
Feb 23, 2023
Results posted
Jun 25, 2025
Last update
Jun 25, 2025

Study contacts

Kyriaki Sarafoglou, MD
principal investigator · University of Minnesota

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Jun 2025. You cannot join it, but the record below documents what was studied.

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Studies of the drug tested here: Hydrocortisone

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