ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2015 by Children's Hospital Boston
Sponsor:
Collaborator:
Terrana ITP Research Fund
Information provided by (Responsible Party):
Rachael Grace, Children's Hospital Boston
ClinicalTrials.gov Identifier:
NCT01971684
First received: October 21, 2013
Last updated: February 2, 2015
Last verified: February 2015
  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

  Show 22 Study Locations
Sponsors and Collaborators
Children's Hospital Boston
Terrana ITP Research Fund
  More Information

No publications provided

Responsible Party: Rachael Grace, Principal Investigator, PKD Natural History Study, Children's Hospital Boston
ClinicalTrials.gov Identifier: NCT01971684     History of Changes
Other Study ID Numbers: P00008709
Study First Received: October 21, 2013
Last Updated: February 2, 2015
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:
Blood Platelet Disorders
Purpura, Thrombocytopenic, Idiopathic
Thrombocytopenia
Autoimmune Diseases
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhage
Hemorrhagic Disorders
Immune System Diseases
Pathologic Processes
Purpura
Purpura, Thrombocytopenic
Signs and Symptoms
Skin Manifestations
Thrombotic Microangiopathies
Mycophenolate mofetil
Enzyme Inhibitors
Immunologic Factors
Immunosuppressive Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on April 23, 2015