Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia

NCT ID: NCT03718234

Last Updated: 2025-06-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 11 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-01-01
• Study Completion Date: 2023-02-23

Brief Summary

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

Conditions

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Subcutaneous Hydrocortisone via Infusion Pump

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

Group Type: EXPERIMENTAL

Subcutaneous hydrocortisone

Intervention Type: DRUG

Pulsatile subcutaneous hydrocortisone administered in 8 doses per day via SQ infusion pump for 6 weeks

Interventions

Subcutaneous hydrocortisone

Pulsatile subcutaneous hydrocortisone administered in 8 doses per day via SQ infusion pump for 6 weeks

Intervention Type: DRUG

Other Intervention Names

Hydrocortisone delivered via subcutaneous pump

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

• Minimum Eligible Age: 4 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Minnesota

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Kyriaki Sarafoglou, MD

Role: PRINCIPAL_INVESTIGATOR

University of Minnesota

Locations

Prism Clinical Research

Minneapolis, Minnesota, United States

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 33278125 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

FD-R-6100

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

PEDS-2018-26475

Identifier Type: -

Identifier Source: org_study_id