Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03718234
Recruitment Status : Recruiting
First Posted : October 24, 2018
Last Update Posted : March 20, 2019
Sponsor:
Information provided by (Responsible Party):
University of Minnesota - Clinical and Translational Science Institute

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

Condition or disease Intervention/treatment Phase
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 Drug: Subcutaneous hydrocortisone Drug: Standard glucocorticoid therapy Phase 1 Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 8 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Intervention Model Description: This is a non-randomized crossover study design.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Interval Bolus Delivery of Subcutaneous Hydrocortisone Via Infusion Pump in Children With Congenital Adrenal Hyperplasia
Actual Study Start Date : January 1, 2019
Estimated Primary Completion Date : January 1, 2020
Estimated Study Completion Date : January 1, 2021


Arm Intervention/treatment
Experimental: Subcutaneous Hydrocortisone via Infusion Pump
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.
Drug: 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.
Other Name: Hydrocortisone delivered via subcutaneous pump

Active Comparator: Standard glucocorticoid therapy
Subjects in this arm will continue on standard oral hydrocortisone therapy
Drug: Standard glucocorticoid therapy
Subjects in this arm will continue on standard oral hydrocortisone therapy




Primary Outcome Measures :
  1. Duration of hypocortisolemia and hypercortisolemia will be significantly shorter [ Time Frame: From date of randomization assessed up to 20 weeks. ]
    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)

  2. Serum concentrations will be within an acceptable range [ Time Frame: From date of randomization assessed up to 20 weeks. ]
    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)



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   4 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03718234


Contacts
Layout table for location contacts
Contact: Kyriakie Sarafoglou, MD 612.624.5409 saraf010@umn.edu

Locations
Layout table for location information
United States, Minnesota
Prism Clinical Research Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Kyriaki Sarafoglou, MD         
Sponsors and Collaborators
University of Minnesota - Clinical and Translational Science Institute
Investigators
Layout table for investigator information
Principal Investigator: Kyriaki Sarafoglou, MD University of Minnesota - Clinical and Translational Science Institute

Layout table for additonal information
Responsible Party: University of Minnesota - Clinical and Translational Science Institute
ClinicalTrials.gov Identifier: NCT03718234     History of Changes
Other Study ID Numbers: FD-R-6100
FD-R-6100 ( Other Grant/Funding Number: FDA Office of Orphan Products Development )
First Posted: October 24, 2018    Key Record Dates
Last Update Posted: March 20, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by University of Minnesota - Clinical and Translational Science Institute:
adrenal gland diseases
cortisol succinate
hydrocortisone
hyperplasia

Additional relevant MeSH terms:
Layout table for MeSH terms
Disease
Hyperplasia
Metabolic Diseases
Genetic Diseases, Inborn
Adrenal Hyperplasia, Congenital
Adrenogenital Syndrome
Adrenocortical Hyperfunction
Disorders of Sex Development
Adrenal Gland Diseases
Urogenital Abnormalities
Steroid Metabolism, Inborn Errors
Pathologic Processes
Congenital Abnormalities
Metabolism, Inborn Errors
Endocrine System Diseases
Gonadal Disorders
Hydrocortisone
Hydrocortisone 17-butyrate 21-propionate
Hydrocortisone acetate
Hydrocortisone hemisuccinate
Glucocorticoids
Epinephrine
Racepinephrine
Epinephryl borate
Anti-Inflammatory Agents
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Adrenergic alpha-Agonists
Adrenergic Agonists