Steroid Use in Pediatric Fluid and Vasoactive Infusion Dependent Shock - Pilot Study

NCT ID: NCT02044159

Last Updated: 2019-03-26

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

57 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-07-31

Study Completion Date

2016-04-30

Brief Summary

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Approximately 20,000 children per year in North America present to the hospital with severe shock. Children who develop this condition have very low blood pressures and as a result may suffer damage to their internal organs and may even die. Some children with this condition may significantly benefit from the use of steroids but steroids in such patients may also have potential side effects. Therefore it is important to study the use of steroids carefully in these children. The STRIPES research program will examine the effectiveness and safety of steroids in children. Before conducting a large, randomized controlled trial (RCT), a pilot study (STRIPES Pilot Study) will be conducted in multiple sites across Canada. The STRIPES Pilot Study will allow testing of the STRIPES study protocol in a smaller group of patients.

Detailed Description

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This study is a multi-centre pilot RCT to determine the feasibility of doing a full trial on the effect of steroids versus placebo in children with fluid and vasoactive infusion dependent shock. The principal investigator and colleagues recently published two articles on this topic which were selected as the Feature Article and Feature Review in the June edition of Pediatric Critical Care Medicine. Both of these publications strongly support the interest in this area and the accompanying editorial strongly supported the need for a well-designed RCT.

RATIONALE: Approximately 20,000 children per year present to emergency departments, pediatric wards and intensive care units in North America with fluid and vasoactive infusion dependent shock. This severe type of shock results in significant morbidity and carries a 2-10% mortality rate depending on the setting in which it occurs. This form of shock is thought to arise from dysfunction of the hypothalamic-pituitary-adrenal axis through a variety of mechanisms and has been referred to as relative adrenal insufficiency or critical illness related adrenal insufficiency. Many clinicians believe that corticosteroids improve outcomes in such patients and therefore use them when confronted with a critically ill child with fluid and vasoactive infusion dependent shock. However, the effectiveness and safety of steroid replacement therapy in pediatric shock remain to be demonstrated.

OBJECTIVES - PILOT STUDY: Before embarking on a multi-centre definitive trial to address the questions listed above, the STRIPES Pilot Study has 3 specific feasibility objectives: 1) To estimate the rate of patient recruitment and understand barriers to recruitment; 2) To assess adherence to our specific treatment protocol; and 3) To document the frequency of and understand the reasons for open label steroid use.

OBJECTIVES - FULL RCT: Following successful completion of the STRIPES Pilot Study, the next phase will be a large, multi centre RCT to answer the following questions: 1) What is the effect of hydrocortisone versus placebo on the time to discontinuation of vasoactive agents among pediatric patients with fluid and vasoactive infusion dependent shock?; and 2) In patients with fluid and vasoactive infusion dependent shock, what is the effect of hydrocortisone versus placebo on i) pediatric intensive care unit (PICU) mortality; ii) duration of mechanical ventilation; iii) new onset of organ dysfunction; iv) PICU length of stay; and v) incidence of adverse events?

RESEARCH PLAN: The STRIPES Pilot Study is designed as a pragmatic, multi-centre, double blind, RCT of intravenous hydrocortisone versus intravenous placebo in fluid and vasoactive infusion dependent shock. This study aims to enroll 72 patients from 7 pediatric centres across Canada over a one year period. Patients will be recruited from the Emergency Department and the PICU within 6 hours of being stared on a vasoactive agent. Research ethics board approval will be obtained from all participating centres and application for deferred consent will be made at 5 sites. Health Canada approval is not required as hydrocortisone is approved for use in children at the doses and for the indication for which it is being used in this study.

As part of the pragmatic design, the prescriptive component of the protocol will be limited to the administration and weaning of the study drug. The requirement for intubation, mechanical ventilation, sedation and analgesia, use of hemodynamic triggers and endpoints, red cell transfusions, antibiotics and fluid boluses will be left to the discretion of the treating physician. The Surviving Sepsis Guidelines Flowchart will be attached to the study protocol for easy reference by the treating physician but its use will not be mandated; however, the use of vasoactive infusions and other therapies will be recorded. The proposed duration of treatment will range from a minimum of 20 hours to a maximum of 7 days of study drug. Outcome data, including survival status and frequency of adverse events, will be collected daily until discharge from hospital.

Although the primary focus of this pilot study is to determine the feasibility of conducting a clinical outcome based RCT of steroids versus placebo in shock, this pilot also provides an excellent opportunity to perform some exploratory mechanistic studies. These will include 1) comparison of total and free cortisol levels of patients with shock; 2) measurement of stratification biomarkers; and 3) determination of 25 hydroxyvitamin D and 1,25 hydroxyvitamin D levels on admission. Patients with access for blood sampling and for whom consent has been obtained will have blood samples sent for these mechanistic studies.

Patients will be randomized to the hydrocortisone or placebo arm using web-based, centralized permuted block randomization, stratified by centre. All study personnel (the overall study research coordinator, research assistants, site investigators, principal investigator, co-investigators, data management personnel, and statisticians), members of the health care team (treating physicians, bedside nurses, and clinical pharmacists) and patients/families will be blinded to the study group assignment.

SIGNIFICANCE: Results of the STRIPES Pilot Study will provide essential feasibility data for planning and conducting a larger, multi-centre trial that will help to establish the role of steroids in children with fluid and vasoactive infusion dependent shock. There are special challenges to patient recruitment in critically ill pediatric populations, adherence to treatment protocols and to the limitation of open label use of steroids, all of which the STRIPES Pilot study will help to address. Success of the STRIPES Pilot Study will be based on the ability to achieve each of the three feasibility objectives listed above. If the pilot study demonstrates feasibility, no major protocol changes are needed, and no safety concerns are raised by the Data Safety Monitoring Board, then the results of the pilot study will be rolled into the full trial. However, if any of the above criteria are not met, then the protocol will be re-evaluated and the feasibility results of the pilot study published independently.

Conditions

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Shock

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Hydrocortisone

Patients randomized to the hydrocortisone arm will receive a 2 mg/kg hydrocortisone IV bolus on enrolment followed by 1 mg/kg of hydrocortisone IV q6h until the patient has not had an escalation in therapy for at least 12 hours. If the patient meets these criteria their hydrocortisone will be weaned to 1 mg/kg every 8 hours which will be continued until they are off all vasoactive infusions for 12 hours. If following the initial hydrocortisone wean, the patient requires fluid boluses and/or an increase in their vasoactive infusion(s), their hydrocortisone will be increased back to 1 mg/kg of hydrocortisone IV q6h until they meet stability criteria again. Duration of treatment will range from a minimum of 20 hours to a maximum of 7 days of study drug.

Group Type EXPERIMENTAL

Hydrocortisone

Intervention Type DRUG

Hydrocortisone will be made up as a 10 mg/ml solution so the volume of added fluid will be very small (2 to 10 mls even for the initial dose of 2 mg/kg).

Placebo

Patients randomized to the placebo arm will receive a placebo solution consisting of normal saline equivalent in volume to the appropriate dose of hydrocortisone. Hydrocortisone and placebo will be identical in appearance, volume and smell as hydrocortisone is made up in normal saline and dissolves completely with no visible precipitate. The dosing regimen will be identical to the hydrocortisone arm.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

The placebo (normal saline) will be identical in appearance, volume and smell to the active study drug (hydrocortisone).

Interventions

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Hydrocortisone

Hydrocortisone will be made up as a 10 mg/ml solution so the volume of added fluid will be very small (2 to 10 mls even for the initial dose of 2 mg/kg).

Intervention Type DRUG

Placebo

The placebo (normal saline) will be identical in appearance, volume and smell to the active study drug (hydrocortisone).

Intervention Type OTHER

Other Intervention Names

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SOLU-CORTEF Placebo (saline solution)

Eligibility Criteria

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Inclusion Criteria

* Children newborn to 17 years
* On any dose of any vasoactive infusion for between 1 to 6 hours

Exclusion Criteria

* Patients who have known or suspected hypothalamic, pituitary or adrenal disease
* Patients who are currently receiving steroids for the treatment of shock/suspected shock prior to randomization
* Patients who are expected to have treatment withdrawn
* Patients who are premature infants (\<38 weeks corrected gestational age)
* Patients who are pregnant
* Patients post cardiac surgery
* Patient who received their first dose of vasoactive infusion \>24 hours after PICU admission
* Patient who is no longer on vasoactive infusion at the time of study enrollment, and/or is expected to no longer be on vasoactive infusion at the time the first dose of study drug will be administered
* Patients for whom primary cardiogenic shock is strongly suspected
* Patients for whom spinal shock is strongly suspected
* Patients for whom hemorrhagic or hypovolemic shock is strongly suspected
* Patients who were previously enrolled in the STRIPES study
* Patients who receive a vasoactive agent for reasons not related to shock
* Physician refusal
Minimum Eligible Age

1 Day

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alberta Children's Hospital

OTHER

Sponsor Role collaborator

British Columbia Children's Hospital

OTHER

Sponsor Role collaborator

London Health Sciences Centre

OTHER

Sponsor Role collaborator

St. Justine's Hospital

OTHER

Sponsor Role collaborator

IWK Health Centre

OTHER

Sponsor Role collaborator

Montreal Children's Hospital of the MUHC

OTHER

Sponsor Role collaborator

Children's Hospital of Eastern Ontario

OTHER

Sponsor Role lead

Responsible Party

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Kusum Menon

Senior Investigator, CHEO Research Institute; Associate Professor, University of Ottawa; Physician, Division of Critical Care

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Kusum Menon, MD, MSc

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Eastern Ontario

Karen Choong, MB, MSc

Role: STUDY_CHAIR

McMaster Children's Hospital

James D McNally, MD, PhD

Role: STUDY_CHAIR

Children's Hospital of Eastern Ontario

Lauralyn McIntyre, MD, MSc

Role: STUDY_CHAIR

The Ottawa Hospital

Margaret Lawson, MD, MSc

Role: STUDY_CHAIR

Children's Hospital of Eastern Ontario

Hector Wong, MD

Role: STUDY_CHAIR

Children's Hospital Medical Center, Cincinnati

Tim Ramsay, MSc, PhD

Role: STUDY_CHAIR

Ottawa Hospital Research Institute

Locations

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Alberta Children's Hospital

Calgary, Alberta, Canada

Site Status

British Columbia Children's Hospital

Vancouver, British Columbia, Canada

Site Status

IWK Health Centre

Halifax, Nova Scotia, Canada

Site Status

McMaster Children's Hospital

Hamilton, Ontario, Canada

Site Status

Children's Hospital of Eastern Ontario

Ottawa, Ontario, Canada

Site Status

Montreal Children's Hospital of the MUHC

Montreal, Quebec, Canada

Site Status

Hospital St. Justine

Montreal, Quebec, Canada

Site Status

Countries

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Canada

References

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Menon K, McNally JD, Choong K, Ward RE, Lawson ML, Ramsay T, Wong HR. A survey of stated physician practices and beliefs on the use of steroids in pediatric fluid and/or vasoactive infusion-dependent shock. Pediatr Crit Care Med. 2013 Jun;14(5):462-6. doi: 10.1097/PCC.0b013e31828a7287.

Reference Type BACKGROUND
PMID: 23628832 (View on PubMed)

Menon K, McNally D, Choong K, Sampson M. A systematic review and meta-analysis on the effect of steroids in pediatric shock. Pediatr Crit Care Med. 2013 Jun;14(5):474-80. doi: 10.1097/PCC.0b013e31828a8125.

Reference Type BACKGROUND
PMID: 23867428 (View on PubMed)

Odetola FO, Gebremariam A, Freed GL. Patient and hospital correlates of clinical outcomes and resource utilization in severe pediatric sepsis. Pediatrics. 2007 Mar;119(3):487-94. doi: 10.1542/peds.2006-2353.

Reference Type BACKGROUND
PMID: 17332201 (View on PubMed)

Kissoon N, Carcillo JA, Espinosa V, Argent A, Devictor D, Madden M, Singhi S, van der Voort E, Latour J; Global Sepsis Initiative Vanguard Center Contributors. World Federation of Pediatric Intensive Care and Critical Care Societies: Global Sepsis Initiative. Pediatr Crit Care Med. 2011 Sep;12(5):494-503. doi: 10.1097/PCC.0b013e318207096c.

Reference Type BACKGROUND
PMID: 21897156 (View on PubMed)

Dellinger RP, Levy MM, Rhodes A, Annane D, Gerlach H, Opal SM, Sevransky JE, Sprung CL, Douglas IS, Jaeschke R, Osborn TM, Nunnally ME, Townsend SR, Reinhart K, Kleinpell RM, Angus DC, Deutschman CS, Machado FR, Rubenfeld GD, Webb S, Beale RJ, Vincent JL, Moreno R; Surviving Sepsis Campaign Guidelines Committee including The Pediatric Subgroup. Surviving Sepsis Campaign: international guidelines for management of severe sepsis and septic shock, 2012. Intensive Care Med. 2013 Feb;39(2):165-228. doi: 10.1007/s00134-012-2769-8. Epub 2013 Jan 30.

Reference Type BACKGROUND
PMID: 23361625 (View on PubMed)

Menon K, Ward RE, Lawson ML, Gaboury I, Hutchison JS, Hebert PC; Canadian Critical Care Trials Group. A prospective multicenter study of adrenal function in critically ill children. Am J Respir Crit Care Med. 2010 Jul 15;182(2):246-51. doi: 10.1164/rccm.200911-1738OC. Epub 2010 Mar 18.

Reference Type BACKGROUND
PMID: 20299532 (View on PubMed)

Zimmerman JJ, Barker RM, Jack R. Initial observations regarding free cortisol quantification logistics among critically ill children. Intensive Care Med. 2010 Nov;36(11):1914-22. doi: 10.1007/s00134-010-2007-1. Epub 2010 Aug 19.

Reference Type BACKGROUND
PMID: 20721535 (View on PubMed)

Poomthavorn P, Lertbunrian R, Preutthipan A, Sriphrapradang A, Khlairit P, Mahachoklertwattana P. Serum free cortisol index, free cortisol, and total cortisol in critically ill children. Intensive Care Med. 2009 Jul;35(7):1281-5. doi: 10.1007/s00134-009-1480-x. Epub 2009 Apr 8.

Reference Type BACKGROUND
PMID: 19352620 (View on PubMed)

Hamrahian AH, Oseni TS, Arafah BM. Measurements of serum free cortisol in critically ill patients. N Engl J Med. 2004 Apr 15;350(16):1629-38. doi: 10.1056/NEJMoa020266.

Reference Type BACKGROUND
PMID: 15084695 (View on PubMed)

Pollack MM, Patel KM, Ruttimann UE. PRISM III: an updated Pediatric Risk of Mortality score. Crit Care Med. 1996 May;24(5):743-52. doi: 10.1097/00003246-199605000-00004.

Reference Type BACKGROUND
PMID: 8706448 (View on PubMed)

Zimmerman JJ, Donaldson A, Barker RM, Meert KL, Harrison R, Carcillo JA, Anand KJ, Newth CJ, Berger J, Willson DF, Jack R, Nicholson C, Dean JM; Eunice Kennedy Shriver National Institute of Child Health and Human Development Collaborative Pediatric Critical Care Research Network. Real-time free cortisol quantification among critically ill children. Pediatr Crit Care Med. 2011 Sep;12(5):525-31. doi: 10.1097/PCC.0b013e3181fe4474.

Reference Type BACKGROUND
PMID: 21057361 (View on PubMed)

Boonen E, Vervenne H, Meersseman P, Andrew R, Mortier L, Declercq PE, Vanwijngaerden YM, Spriet I, Wouters PJ, Vander Perre S, Langouche L, Vanhorebeek I, Walker BR, Van den Berghe G. Reduced cortisol metabolism during critical illness. N Engl J Med. 2013 Apr 18;368(16):1477-88. doi: 10.1056/NEJMoa1214969. Epub 2013 Mar 19.

Reference Type BACKGROUND
PMID: 23506003 (View on PubMed)

Watson RS, Carcillo JA, Linde-Zwirble WT, Clermont G, Lidicker J, Angus DC. The epidemiology of severe sepsis in children in the United States. Am J Respir Crit Care Med. 2003 Mar 1;167(5):695-701. doi: 10.1164/rccm.200207-682OC. Epub 2002 Nov 14.

Reference Type BACKGROUND
PMID: 12433670 (View on PubMed)

Wong HR, Cvijanovich NZ, Allen GL, Thomas NJ, Freishtat RJ, Anas N, Meyer K, Checchia PA, Lin R, Shanley TP, Bigham MT, Wheeler DS, Doughty LA, Tegtmeyer K, Poynter SE, Kaplan JM, Chima RS, Stalets E, Basu RK, Varisco BM, Barr FE. Validation of a gene expression-based subclassification strategy for pediatric septic shock. Crit Care Med. 2011 Nov;39(11):2511-7. doi: 10.1097/CCM.0b013e3182257675.

Reference Type BACKGROUND
PMID: 21705885 (View on PubMed)

McNally JD, Doherty DR, Lawson ML, Al-Dirbashi OY, Chakraborty P, Ramsay T, Menon K. The relationship between vitamin D status and adrenal insufficiency in critically ill children. J Clin Endocrinol Metab. 2013 May;98(5):E877-81. doi: 10.1210/jc.2013-1126. Epub 2013 Apr 1.

Reference Type BACKGROUND
PMID: 23547046 (View on PubMed)

O'Hearn K, McNally D, Choong K, Acharya A, Wong HR, Lawson M, Ramsay T, McIntyre L, Gilfoyle E, Tucci M, Wensley D, Gottesman R, Morrison G, Menon K; Canadian Critical Care Trials Group. Steroids in fluid and/or vasoactive infusion dependent pediatric shock: study protocol for a randomized controlled trial. Trials. 2016 May 6;17(1):238. doi: 10.1186/s13063-016-1365-6.

Reference Type DERIVED
PMID: 27153945 (View on PubMed)

Other Identifiers

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STRIPES-Pilot

Identifier Type: -

Identifier Source: org_study_id

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