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Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia

NCT03718234 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 11

Last updated 2025-06-25

Study results available
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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

Interventions

DRUG

Subcutaneous hydrocortisone

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

Sponsors & Collaborators

Principal Investigators

  • Kyriaki Sarafoglou, MD · University of Minnesota

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-01-01
Primary Completion
2023-02-23
Completion
2023-02-23
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03718234 on ClinicalTrials.gov