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Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia

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ClinicalTrials.gov Identifier: NCT03718234
Recruitment Status : Recruiting
First Posted : October 24, 2018
Last Update Posted : July 22, 2019
Sponsor:
Information provided by (Responsible Party):
University of Minnesota - Clinical and Translational Science Institute

Tracking Information
First Submitted Date  ICMJE October 11, 2018
First Posted Date  ICMJE October 24, 2018
Last Update Posted Date July 22, 2019
Actual Study Start Date  ICMJE January 1, 2019
Estimated Primary Completion Date January 1, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 14, 2018)
  • 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)
  • 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)
Original Primary Outcome Measures  ICMJE
 (submitted: October 23, 2018)
  • Duration of symptoms will be significantly shorter [ Time Frame: From date of randomization assessed up to 12 months ]
    Duration of hypocortisolemia and hypercortisolemia will be significantly shorter on SQHC pump therapy (admissions 2 and 3) than with conventional oral HC dosing (admission 1).
  • Serum concentrations will be within an acceptable range [ Time Frame: From date of randomization assessed up to 12 months ]
    Duration of time 17OHP and D4A serum concentrations are outside an acceptable range of suppression will be significantly shorter while on the SQHC pump (admissions 2 and 3) than with oral dosing (admission 1).
Change History Complete list of historical versions of study NCT03718234 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Subcutaneous Hydrocortisone Children With Congenital Adrenal Hyperplasia
Official Title  ICMJE Interval Bolus Delivery of Subcutaneous Hydrocortisone Via Infusion Pump in Children With Congenital Adrenal Hyperplasia
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
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE 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
Condition  ICMJE
  • 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
Intervention  ICMJE
  • 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
  • Drug: Standard glucocorticoid therapy
    Subjects in this arm will continue on standard oral hydrocortisone therapy
Study Arms  ICMJE
  • 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.
    Intervention: Drug: Subcutaneous hydrocortisone
  • Active Comparator: Standard glucocorticoid therapy
    Subjects in this arm will continue on standard oral hydrocortisone therapy
    Intervention: Drug: Standard glucocorticoid therapy
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 23, 2018)
8
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 1, 2021
Estimated Primary Completion Date January 1, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 4 Years to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Kyriakie Sarafoglou, MD 612.624.5409 saraf010@umn.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03718234
Other Study ID Numbers  ICMJE FD-R-6100
FD-R-6100 ( Other Grant/Funding Number: FDA Office of Orphan Products Development )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party University of Minnesota - Clinical and Translational Science Institute
Study Sponsor  ICMJE University of Minnesota - Clinical and Translational Science Institute
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Kyriaki Sarafoglou, MD University of Minnesota - Clinical and Translational Science Institute
PRS Account University of Minnesota - Clinical and Translational Science Institute
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP