Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia

Phase 1

Completed

• Conditions: Congenital Disorders; Adrenal Hyperplasia; Adrenocortical Hyperfunction; Congenital Adrenal Hyperplasia; Hydrocortisone; Genetic Diseases, Inborn; Steroid Metabolic Diseases, Inborn; Adrenal Gland Disease; Hyperplasia; Disorders of Sex Development
• Interventions: Drug: Subcutaneous hydrocortisone; Drug: Standard glucocorticoid therapy

• Registration Number: NCT03718234
• Lead Sponsor: University of Minnesota

Brief 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

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2018-10-11
• Study First Submitted QC: 2018-10-23
• Study First Posted: 2018-10-24
• Study Start: 2019-01-01
• Primary Completion: 2023-02-23
• Study Completion: 2023-02-23
• Status Verified: 2023-07
• Last Update Submitted: 2023-07-19
• Last Update Posted: 2023-07-20

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 11

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Subcutaneous Hydrocortisone via Infusion Pump	Subcutaneous hydrocortisone	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.
Standard glucocorticoid therapy	Standard glucocorticoid therapy	Subjects in this arm will continue on standard oral hydrocortisone therapy

Primary Outcome Measures

Name	Time	Method
Serum concentrations will be within an acceptable range	From date of randomization assessed up to 20 weeks.	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)
Duration of hypocortisolemia and hypercortisolemia will be significantly shorter	From date of randomization assessed up to 20 weeks.	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)

Trial Locations

Locations (1)

Prism Clinical Research; 🇺🇸 Minneapolis, Minnesota, United States