Clinical trial

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

Name

PEDS-2018-26475

Description

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

Trial arms

Trial start

2019-01-01

Estimated PCD

2023-02-23

Trial end

2023-02-23

Status

Completed

Phase

Early phase I

Treatment

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.

Arms:

Subcutaneous Hydrocortisone via Infusion Pump

Other names:

Hydrocortisone delivered via subcutaneous pump

Standard glucocorticoid therapy

Subjects in this arm will continue on standard oral hydrocortisone therapy

Arms:

Standard glucocorticoid therapy

Size

Primary endpoint

Duration of hypocortisolemia and hypercortisolemia will be significantly shorter

From date of randomization assessed up to 20 weeks.

Serum concentrations will be within an acceptable range

From date of randomization assessed up to 20 weeks.

Eligibility 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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'This is a non-randomized crossover study design.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 11, 'type': 'ACTUAL'}}

Updated at

2023-07-20

1 organization

2 products

12 indications

Organization

University of Minnesota

Product

Hydrocortisone

Indication

Congenital Adrenal Hyperplasia

Indication