Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia

• Status: Recruiting
• Trial end date: 2022-07-01
• Target enrollment: 0
• Participant gender: All

Summary

This is an open-label, non-randomized crossover design feasibility trial comparing oral hydrocortisone treatment with interval bolus delivery (pulsatile) of subcutaneous hydrocortisone via infusion pump in children with congenital adrenal hyperplasia. Eight children, ages 4-18 yrs, will have 24-hr pharmacokinetic and pharmacodynamic profiles of cortisol, 17-hydroxyprogesterone and androstenedione concentrations while on oral hydrocortisone therapy (admission 1), during an initial trial of the subcutaneous hydrocortisone pump (admission 2), and after 6 weeks of subcutaneous hydrocortisone pump treatment (admission 3). An integrated pharmacokinetic and pharmacodynamic model will be used to determine cortisol, 17-hydroxyprogesterone and androstenedione parameters to compare the duration of time subjects have these concentrations outside acceptable ranges. Funding Source

• FDA OOPD

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Minnesota; University of Minnesota - Clinical and Translational Science Institute
• Treatments: Cortisol succinate; Glucocorticoids; Hydrocortisone; Hydrocortisone 17-butyrate 21-propionate; Hydrocortisone acetate; Hydrocortisone hemisuccinate

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