ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP

Immune Thrombocytopenia Clinical Trial

— ICON1  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01971684
• Other study ID #: P00008709
• Status: Completed
• Start date: August 2013
• Est. completion date: April 2017

Study information

• Verified date: May 2020
• Source: Boston Children’s Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 120
• Est. completion date: April 2017
• Est. primary completion date: April 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• Second Line ITP agents
The treating physicians will select the second line agent and clinical data will be collected.

Locations

Country	Name	City	State
Canada	McMaster Children's Hospital	Hamilton	Ontario
Canada	U. de Montreal CHU St. Justine	Montreal	Quebec
Canada	Children's Hospital of Eastern Ontario	Ottawa	Ontario
United States	Children's Healthcare of Atlanta	Atlanta	Georgia
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Cincinnati Children's Hospital	Cincinnati	Ohio
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	University of Texas Southwestern Medical Center at Dallas	Dallas	Texas
United States	Colorado Children's Hospital	Denver	Colorado
United States	St. John Hospital & Medical Center	Detroit	Michigan
United States	Duke University Medical Center	Durham	North Carolina
United States	Texas Children's Hospital	Houston	Texas
United States	James Whitcomb Riley Hospital for Children	Indianapolis	Indiana
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	Mattel Children's Hospital	Los Angeles	California
United States	St. Jude's Hospital	Memphis	Tennessee
United States	Goryeb Children's Hospital	Morristown	New Jersey
United States	Columbia University Medical Center	New York	New York
United States	New York-Presbyterian University Hospital of Columbia and Cornell	New York	New York
United States	Children's Hospital of Oakland	Oakland	California
United States	Children's Hospital of Orange County	Orange	California
United States	Lucile Packard Children's Hospital	Palo Alto	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Oregon Health and Sciences University	Portland	Oregon
United States	Hasbro Children's Hospital	Providence	Rhode Island
United States	UC Davis Medical Center	Sacramento	California
United States	UCSF School of Medicine	San Francisco	California
United States	Seattle Children's	Seattle	Washington

Sponsors (2)

Lead Sponsor	Collaborator
Boston Children’s Hospital	Terrana ITP Research Fund

Countries where clinical trial is conducted

United States,  Canada, 

References & Publications (2)

Grace RF, Despotovic JM, Bennett CM, Bussel JB, Neier M, Neunert C, Crary SE, Pastore YD, Klaassen RJ, Rothman JA, Hege K, Breakey VR, Rose MJ, Shimano KA, Buchanan GR, Geddis A, Haley KM, Lorenzana A, Thompson A, Jeng M, Neufeld EJ, Brown T, Forbes PW, L — View Citation

Grace RF, Shimano KA, Bhat R, Neunert C, Bussel JB, Klaassen RJ, Lambert MP, Rothman JA, Breakey VR, Hege K, Bennett CM, Rose MJ, Haley KM, Buchanan GR, Geddis A, Lorenzana A, Jeng M, Pastore YD, Crary SE, Neier M, Neufeld EJ, Neu N, Forbes PW, Despotovic — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	change from baseline in patient reported outcomes	Kids ITP Tool, Memorial Symptom Assessment Scale, Fatigue Scale	Enrollment, 1 and 12 months
Primary	change from baseline in bleeding assessment	ITP Bleeding Scale, Bleeding Assessment Tool	Enrollment, 1, 6, and 12 months
Primary	change from baseline in platelet count		over 1 year
Secondary	side effects and complications of treatments		1 year