ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2014 by Children's Hospital Boston
Sponsor:
Collaborator:
Terrana ITP Research Fund
Information provided by (Responsible Party):
Children's Hospital Boston
ClinicalTrials.gov Identifier:
NCT01971684
First received: October 21, 2013
Last updated: July 21, 2014
Last verified: July 2014
  Purpose

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.


Condition Intervention
Immune Thrombocytopenia
Drug: Second Line ITP agents

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: ICON1: Physician Treatment Decisions and Patient-Reported Outcomes in Pediatric Refractory Immune Thrombocytopenia

Resource links provided by NLM:


Further study details as provided by Children's Hospital Boston:

Primary Outcome Measures:
  • change from baseline in patient reported outcomes [ Time Frame: Enrollment, 1 and 12 months ] [ Designated as safety issue: No ]
    Kids ITP Tool, Memorial Symptom Assessment Scale, Fatigue Scale

  • change from baseline in bleeding assessment [ Time Frame: Enrollment, 1, 6, and 12 months ] [ Designated as safety issue: No ]
    ITP Bleeding Scale, Bleeding Assessment Tool

  • change from baseline in platelet count [ Time Frame: over 1 year ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • side effects and complications of treatments [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 120
Study Start Date: August 2013
Estimated Primary Completion Date: September 2016 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
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:
  • rituximab
  • 6-mercaptopurine
  • mycophenolate mofetil
  • sirolimus
  • splenectomy
  • romiplostim
  • eltrombopag

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.
  Eligibility

Ages Eligible for Study:   1 Year to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Pediatric refractory ITP patients starting on a new second line therapy

Criteria

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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01971684

Contacts
Contact: Rachael F Grace, MD, MMSc 617-919-2144 Rachael.Grace@childrens.harvard.edu

Locations
United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
Contact: Jacquelyn Jackson, MS, CCRP    501-364-1691    JacksonJacquelynF@uams.edu   
Principal Investigator: Kimo Stine, MD         
United States, California
Children's Hospital of Orange County Recruiting
Orange, California, United States, 92868
Contact: Brandy Gonzales    714-532-8459    bgonzales@CHOC.org   
Principal Investigator: Diane Nugent, MD         
Lucile Packard Children's Hospital Recruiting
Palo Alto, California, United States, 94304
Contact: Amanda Merlino    650-736-1177    amerlino@stanford.edu   
Principal Investigator: Michael Jeng, MD         
Principal Investigator: Clara Lo, MD         
Rady Children's Hospital Recruiting
San Diego, California, United States, 92123
Contact: Theresa Vella    858-966-5811    tvella@rchsd.org   
Principal Investigator: Amy Geddis, MD         
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Pauline Hess    312-227-4811    phess@luriechildrens.org   
Principal Investigator: Alexis A Thompson, MD, MPH         
United States, Indiana
James Whitcomb Riley Hospital for Children Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Shannon Maraldo    317-944-8784    saranjo@iu.edu   
Principal Investigator: James Croop, MD         
Principal Investigator: Manjusha Kumar, MD         
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Travis Brown    617-355-7203    Travis.Brown@childrens.harvard.edu   
Principal Investigator: Rachael F Grace, MD, MMSc         
United States, Michigan
St. John Hospital & Medical Center Recruiting
Detroit, Michigan, United States, 48236
Contact: Elilzabeth Mill    313-647-3214    Elizabeth.Mill@stjohn.org   
Principal Investigator: Adonis Lorenzana, MD         
United States, New Jersey
Goryeb Children's Hospital Recruiting
Morristown, New Jersey, United States, 07960
Contact: Lisa Paley    973-971-6720    Lisa.Paley@atlantichealth.org   
Principal Investigator: Michele Neier, MD         
United States, New York
New York-Presbyterian University Hospital of Columbia and Cornell Recruiting
New York, New York, United States, 10065
Contact: Micha Thompson    212-746-3978    mit2017@med.cornell.edu   
Contact: Megan Wissert    (212) 746-3978    mwissert@med.cornell.edu   
Principal Investigator: Catherine McGuinn, MD         
Principal Investigator: James Bussel, MD         
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27710
Principal Investigator: Jennifer Rothman, MD         
United States, Ohio
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
Contact: Dianna Hidalgo    614-722-3550    Dianna.Hidalgo@nationwidechildrens.org   
Contact: Terah Koch    614-722-3550    Terah.Koch@nationwidechildrens.org   
Principal Investigator: Melissa Rose, DO         
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Lisa Bevilacqua    215-590-4667    BevilacquaL@email.chop.edu   
Principal Investigator: Michele Lambert, MD         
United States, Texas
University of Texas Southwestern Medical Center at Dallas Recruiting
Dallas, Texas, United States, 75235
Contact: Anna Winborn    214-648-3322    ANNA.WINBORN@childrens.com   
Principal Investigator: George Buchanan, MD         
Canada, Ontario
McMaster Children's Hospital Recruiting
Hamilton, Ontario, Canada, L8S 4K1
Contact: Nagel Kim    905-521-2100 ext 73080    Nagel@HHSC.ca   
Principal Investigator: Vicky Breakey, MD         
Sponsors and Collaborators
Children's Hospital Boston
Terrana ITP Research Fund
  More Information

No publications provided

Responsible Party: Children's Hospital Boston
ClinicalTrials.gov Identifier: NCT01971684     History of Changes
Other Study ID Numbers: P00008709
Study First Received: October 21, 2013
Last Updated: July 21, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Children's Hospital Boston:
Blood Platelet Disorders
ITP
idiopathic thrombocytopenic purpura
bleeding score
platelet count
health-related quality of life
HRQL
Purpura
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhage
Skin Manifestations
Thrombocytopenia
Immune System Diseases
Hemorrhagic Disorders
Autoimmune Diseases
Therapeutic Uses

Additional relevant MeSH terms:
Thrombocytopenia
Blood Platelet Disorders
Hematologic Diseases
Mycophenolate mofetil
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on September 22, 2014