Phase 1
N=11
Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia
Congenital Adrenal Hyperplasia · Hyperplasia · Adrenal Hyperplasia · Congenital Disorders · Adrenocortical Hyperfunction
Bottom Line
View on ClinicalTrials.gov: NCT03718234 ↗Enrolled (actual)
11
Serious AEs
0.0%
Results posted
Jun 2025
Primary outcome: Primary: Cortisol Exposure — 110 hr*mcg/dL
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- Subcutaneous hydrocortisone (Drug)
- Age
- Pediatric, Adult · 4+ yrs
- Sex
- All
- Sponsor
- University of Minnesota
- Primary completion
- Feb 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Cortisol Exposure |
118 | — |
| PRIMARY Cortisol Exposure |
118 | — |
| PRIMARY 17-OHP Exposure |
26900 | — |
| PRIMARY 17-OHP Exposure |
26900 | — |
| PRIMARY Androstenedione (A4) Exposure |
1420 | — |
| PRIMARY Androstenedione (A4) Exposure |
1420 | — |
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
Eligibility Criteria
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
- 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
Data sourced from ClinicalTrials.gov (NCT03718234). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.