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: 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, 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

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: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

11 participants in 1 patient group

Subcutaneous Hydrocortisone via Infusion Pump

Experimental group

Description:

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

Treatment:

Drug: Subcutaneous hydrocortisone