Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia

University of Minnesota (UMN)

Status and phase: Completed; Phase 1

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

Treatments: Drug: Standard glucocorticoid therapy; Drug: Subcutaneous hydrocortisone

Study type: Interventional

Funder types: Other

Identifiers

NCT03718234

PEDS-2018-26475

FD-R-6100 (Other Grant/Funding Number)

Details and patient eligibility

About

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

Enrollment: 11 patients

Sex: All

Ages: 4 to 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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11 participants in 2 patient groups

Subcutaneous Hydrocortisone via Infusion Pump

Experimental group

Description:

Patients will receive a subcutaneous injection of hydrocortisone (HC). Each patient's total daily dose (TDD) of oral tablet hydrocortisone to determine the doses to be delivered of the study drug. The 24-hr schedule and percentage of the TDD of HC will be as follows: approximately 60% of the TDD of HC will be delivered in 3 equal pulses at 0300, 0600 and 0900. Another 35% will be delivered in 3 equal pulses at 1200, 1500 and 1800 and the remaining 5% at 2100 and 2400.

Treatment:

Drug: Subcutaneous hydrocortisone

Standard glucocorticoid therapy

Active Comparator group

Description:

Subjects in this arm will continue on standard oral hydrocortisone therapy

Treatment:

Drug: Standard glucocorticoid therapy