ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP (ICON1)

May 19, 2020 updated by: Rachael Grace, Boston Children's Hospital

ICON1: Physician Treatment Decisions and Patient-Reported Outcomes in Pediatric Refractory Immune Thrombocytopenia

The purpose of this study is to understand physician treatment decisions in selecting specific second line treatments in pediatric ITP and to determine the effectiveness of different second line ITP treatments. Eligible patients are those ages 1-18 years who are starting on a new second line treatment for ITP, defined as any treatment other than IVIG, steroids, anti-D globulin, or aminocaproic acid. Enrolled patients remain on the study for approximately one year.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

The purpose of this observational study is to model factors that determine physician treatment decisions in selecting specific second line agents in pediatric ITP and to determine the comparative effectiveness of second line ITP treatments by bleeding measures, platelet counts, and patient reported outcome measures. This prospective observational, longitudinal, multicenter cohort study will aim to collect routine clinical care data, quality of life information from patients, and decision making data from clinicians at enrollment and at regular clinical intervals for at least one year. The primary and secondary objectives are as follows:

Primary Objectives:

  1. To model factors that determine physician treatment decisions in selecting specific second line agents in pediatric ITP.
  2. To assess patient reported outcomes with relation to specific second line pediatric ITP therapies.
  3. To determine the comparative effectiveness of second line ITP treatments in terms of bleeding and platelet counts.

Secondary Objectives:

  1. To describe phenotypic variation among patients with refractory ITP;
  2. To assess side effects and complications related to specific treatments for refractory ITP;
  3. To describe monitoring and follow up practices among pediatric hematologists with each second line agent;
  4. To weight factors that physicians use when deciding to treat pediatric ITP patients with second line agents;
  5. To determine whether physician perception of patient quality of life correlates with patient derived quality of life measures;
  6. To measure the correlation between the ITP Bleeding Scale and the Bleeding Assessment Tool in refractory pediatric ITP patients.

Study Type

Observational

Enrollment (Actual)

120

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Hamilton, Ontario, Canada, L8S 4K1
        • McMaster Children's Hospital
      • Ottawa, Ontario, Canada, KIH8L1
        • Children's Hospital of Eastern Ontario
    • Quebec
      • Montreal, Quebec, Canada, H3T1C5
        • U. de Montreal CHU St. Justine
  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital
    • California
      • Los Angeles, California, United States, 90095
        • Mattel Children's Hospital
      • Oakland, California, United States, 94609
        • Children's Hospital of Oakland
      • Orange, California, United States, 92868
        • Children's Hospital of Orange County
      • Palo Alto, California, United States, 94304
        • Lucile Packard Children's Hospital
      • Sacramento, California, United States, 95817
        • UC Davis Medical Center
      • San Francisco, California, United States, 94143
        • UCSF School of Medicine
    • Colorado
      • Denver, Colorado, United States, 80045
        • Colorado Children's Hospital
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Children's Healthcare of Atlanta
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • James Whitcomb Riley Hospital For Children
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Michigan
      • Detroit, Michigan, United States, 48236
        • St. John Hospital & Medical Center
    • New Jersey
      • Morristown, New Jersey, United States, 07960
        • Goryeb Children's Hospital
    • New York
      • New York, New York, United States, 10032
        • Columbia University Medical Center
      • New York, New York, United States, 10065
        • New York-Presbyterian University Hospital of Columbia and Cornell
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Sciences University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
    • Rhode Island
      • Providence, Rhode Island, United States, 02903
        • Hasbro Children's Hospital
    • Tennessee
      • Memphis, Tennessee, United States, 38105
        • St. Jude's Hospital
    • Texas
      • Dallas, Texas, United States, 75235
        • University of Texas Southwestern Medical Center at Dallas
      • Houston, Texas, United States, 77030
        • Texas Children's Hospital
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 18 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Pediatric refractory ITP patients starting on a new second line therapy

Description

Inclusion Criteria:

  • Immune Thrombocytopenia or Evans Syndrome
  • Ages > 12 months to <18 years
  • Starting a new second line therapy as defined as any therapy except IVIG, steroids, anti-D globulin, or aminocaproic acid
  • Starting a single agent/monotherapy

Exclusion Criteria:

  • Evans Syndrome with a history of or current evidence of autoimmune hemolytic anemia
  • Unwillingness to be followed for 1 year
  • Physician providing care is unwilling to participate
  • Patient is starting multiple second line agents simultaneously

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Refractory Pediatric ITP Patients
Pediatric ITP patients, ages 1-18, starting a new second line ITP therapy, defined as not IVIG, steroids, anti-D, or aminocaproic acid.
Drug: Second Line ITP agents
The treating physicians will select the second line agent and clinical data will be collected.
Other Names:
  • romiplostim
  • sirolimus
  • 6-mercaptopurine
  • mycophenolate mofetil
  • rituximab
  • splenectomy
  • eltrombopag

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
change from baseline in patient reported outcomes
Time Frame: Enrollment, 1 and 12 months
Kids ITP Tool, Memorial Symptom Assessment Scale, Fatigue Scale
Enrollment, 1 and 12 months
change from baseline in bleeding assessment
Time Frame: Enrollment, 1, 6, and 12 months
ITP Bleeding Scale, Bleeding Assessment Tool
Enrollment, 1, 6, and 12 months
change from baseline in platelet count
Time Frame: over 1 year
over 1 year

Secondary Outcome Measures

Outcome Measure
Time Frame
side effects and complications of treatments
Time Frame: 1 year
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Boston Children's Hospital

Collaborators

Terrana ITP Research Fund

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2013

Primary Completion (ACTUAL)

April 1, 2017

Study Completion (ACTUAL)

April 1, 2017

Study Registration Dates

First Submitted

October 21, 2013

First Submitted That Met QC Criteria

October 24, 2013

First Posted (ESTIMATE)

October 29, 2013

Study Record Updates

Last Update Posted (ACTUAL)

May 21, 2020

Last Update Submitted That Met QC Criteria

May 19, 2020

Last Verified

May 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • P00008709

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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