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Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia

Primary Purpose

Congenital Adrenal Hyperplasia, Hyperplasia, Adrenal Hyperplasia

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Subcutaneous hydrocortisone
Standard glucocorticoid therapy
Sponsored by
University of Minnesota
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Congenital Adrenal Hyperplasia focused on measuring adrenal gland diseases, cortisol succinate, hydrocortisone, hyperplasia

Eligibility Criteria

4 Years - 18 Years (Child, Adult)All SexesDoes not accept 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

Sites / Locations

  • Prism Clinical Research

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

Subcutaneous Hydrocortisone via Infusion Pump

Standard glucocorticoid therapy

Arm 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.

Subjects in this arm will continue on standard oral hydrocortisone therapy

Outcomes

Primary Outcome Measures

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)
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)

Secondary Outcome Measures

Full Information

First Posted
October 11, 2018
Last Updated
July 19, 2023
Sponsor
University of Minnesota
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1. Study Identification

Unique Protocol Identification Number
NCT03718234
Brief Title
Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia
Official Title
Interval Bolus Delivery of Subcutaneous Hydrocortisone Via Infusion Pump in Children With Congenital Adrenal Hyperplasia
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Completed
Study Start Date
January 1, 2019 (Actual)
Primary Completion Date
February 23, 2023 (Actual)
Study Completion Date
February 23, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Minnesota

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Adrenal Hyperplasia, Hyperplasia, Adrenal Hyperplasia, Congenital Disorders, Adrenocortical Hyperfunction, Disorders of Sex Development, Urogenital Abnormalities, Genetic Diseases, Inborn, Steroid Metabolic Diseases, Inborn, Adrenal Gland Disease, Hydrocortisone
Keywords
adrenal gland diseases, cortisol succinate, hydrocortisone, hyperplasia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Model Description
This is a non-randomized crossover study design.
Masking
None (Open Label)
Allocation
Randomized
Enrollment
11 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Subcutaneous Hydrocortisone via Infusion Pump
Arm Type
Experimental
Arm 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.
Arm Title
Standard glucocorticoid therapy
Arm Type
Active Comparator
Arm Description
Subjects in this arm will continue on standard oral hydrocortisone therapy
Intervention Type
Drug
Intervention Name(s)
Subcutaneous hydrocortisone
Other Intervention Name(s)
Hydrocortisone delivered via subcutaneous pump
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Standard glucocorticoid therapy
Intervention Description
Subjects in this arm will continue on standard oral hydrocortisone therapy
Primary Outcome Measure Information:
Title
Duration of hypocortisolemia and hypercortisolemia will be significantly shorter
Description
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)
Time Frame
From date of randomization assessed up to 20 weeks.
Title
Serum concentrations will be within an acceptable range
Description
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)
Time Frame
From date of randomization assessed up to 20 weeks.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kyriaki Sarafoglou, MD
Organizational Affiliation
University of Minnesota
Official's Role
Principal Investigator
Facility Information:
Facility Name
Prism Clinical Research
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
33278125
Citation
Ramirez Alcantara J, Halper A. Adrenal insufficiency updates in children. Curr Opin Endocrinol Diabetes Obes. 2021 Feb 1;28(1):75-81. doi: 10.1097/MED.0000000000000591.
Results Reference
derived

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Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia

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