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Completed

NCT Number: NCT03718234

Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia

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

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Key information

Conditions

Congenital Adrenal Hyperplasia Adrenal Gland Disease Adrenal Gland Diseases Adrenal Hyperplasia Adrenal Hyperplasia, Congenital Adrenocortical Hyperfunction Adrenogenital Syndrome Congenital Abnormalities Congenital Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disorders of Sex Development Endocrine System Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Gonadal Disorders Hydrocortisone Hyperplasia Male Urogenital Diseases Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Steroid Metabolic Diseases, Inborn Steroid Metabolism, Inborn Errors Urogenital Abnormalities Urogenital Diseases

Age range

4 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Prism Clinical Research

Minneapolis, Minnesota, 55455, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Subcutaneous hydrocortisone

Drug

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

Other names: Hydrocortisone delivered via subcutaneous pump

Primary outcomes

  1. Cortisol Exposure

    Time frame: Week 1

    24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).

  2. Cortisol Exposure

    Time frame: Week 14

    The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)

  3. 17-OHP Exposure

    Time frame: Week 1

    24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).

  4. 17-OHP Exposure

    Time frame: Week 14

    The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)

  5. Androstenedione (A4) Exposure

    Time frame: Week 1

    24-hour area under the curve (AUC) following oral hydrocortisone dosing (admission 1).

  6. Androstenedione (A4) Exposure

    Time frame: Week 14

    The 24-hour cortisol area-under-the curve (AUC) while on the interval bolus delivery (pulsatile) of SQHC (admission 3)

Sponsors and collaborators

Lead sponsor

University of Minnesota

Other

Registry information

Official study title

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

Important dates

Study start
2019
Primary completion
2023
Study completion
2023
First posted
Oct 24, 2018
Registry last updated
Jun 25, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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