Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia

Congenital Disorders Clinical Trial

Official title:

Interval Bolus Delivery of Subcutaneous Hydrocortisone Via Infusion Pump in Children With Congenital Adrenal Hyperplasia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03718234
• Other study ID #: PEDS-2018-26475
• Secondary ID: FD-R-6100
• Status: Completed
• Phase: Phase 1
• Start date: January 1, 2019
• Est. completion date: February 23, 2023

Study information

• Verified date: July 2023
• Source: University of Minnesota
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 11
• Est. completion date: February 23, 2023
• Est. primary completion date: February 23, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

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

Intervention

Drug:
• Subcutaneous hydrocortisone
Patients will be monitored and receive an interval bolus SQHC dosing regimen that more closely mimics cortisol, 17OHP and D4A circadian and ultradian rhythms than conventional oral HC dosing.
• Standard glucocorticoid therapy
Subjects in this arm will continue on standard oral hydrocortisone therapy

Locations

Country	Name	City	State
United States	Prism Clinical Research	Minneapolis	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
University of Minnesota

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Duration of hypocortisolemia and hypercortisolemia will be significantly shorter	Duration of hypocortisolemia and hypercortisolemia will be significantly shorter on the interval bolus delivery (pulsatile) of subcutaneous hydrocortisone (admissions 2 and 3) than with conventional oral hydrocortisone dosing (admission 1)	From date of randomization assessed up to 20 weeks.
Primary	Serum concentrations will be within an acceptable range	Duration of time 17-hydroxyprogesterone and androstenedione serum concentrations are outside an acceptable range of suppression will be significantly shorter while on on the interval bolus delivery (pulsatile) of subcutaneous hydrocortisone (admissions 2 and 3) than with oral hydrocortisone dosing (admission 1)	From date of randomization assessed up to 20 weeks.