Immune ThrombocytopeniaICON1NCT01971684
ICON1: Treatment Decisions and Outcomes in Pediatric Refractory ITP
An observational study of Immune Thrombocytopenia, run by Boston Children's Hospital at 29 sites in 2 countries.
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. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- Second Line ITP agents
- How many people
- 120 participants
- Where
- 29 sitesin 2 countries
- Study dates
- Aug 2013to Apr 2017
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Pediatric refractory ITP patients starting on a new second line therapy
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
29 sites in 2 countries.
29 sites in 2 countries
United States26 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Boston Children's Hospital
Boston, Massachusetts, 02115
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, 75235
Colorado Children's Hospital
Denver, Colorado, 80045
St. John Hospital & Medical Center
Detroit, Michigan, 48236
Duke University Medical Center
Durham, North Carolina, 27710
Texas Children's Hospital
Houston, Texas, 77030
James Whitcomb Riley Hospital for Children
Indianapolis, Indiana, 46202
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Mattel Children's Hospital
Los Angeles, California, 90095
St. Jude's Hospital
Memphis, Tennessee, 38105
Goryeb Children's Hospital
Morristown, New Jersey, 07960
Columbia University Medical Center
New York, New York, 10032
New York-Presbyterian University Hospital of Columbia and Cornell
New York, New York, 10065
Children's Hospital of Oakland
Oakland, California, 94609
Children's Hospital of Orange County
Orange, California, 92868
Lucile Packard Children's Hospital
Palo Alto, California, 94304
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Oregon Health and Sciences University
Portland, Oregon, 97239
Hasbro Children's Hospital
Providence, Rhode Island, 02903
UC Davis Medical Center
Sacramento, California, 95817
UCSF School of Medicine
San Francisco, California, 94143
Seattle Children's
Seattle, Washington, 98105
Canada3 sites
McMaster Children's Hospital
Hamilton, Ontario, L8S 4K1
U. de Montreal CHU St. Justine
Montreal, Quebec, H3T1C5
Children's Hospital of Eastern Ontario
Ottawa, Ontario, KIH8L1
About this study
Official title: 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.
Read the 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:
- To model factors that determine physician treatment decisions in selecting specific second line agents in pediatric ITP.
- To assess patient reported outcomes with relation to specific second line pediatric ITP therapies.
- To determine the comparative effectiveness of second line ITP treatments in terms of bleeding and platelet counts.
Secondary Objectives:
- To describe phenotypic variation among patients with refractory ITP;
- To assess side effects and complications related to specific treatments for refractory ITP;
- To describe monitoring and follow up practices among pediatric hematologists with each second line agent;
- To weight factors that physicians use when deciding to treat pediatric ITP patients with second line agents;
- To determine whether physician perception of patient quality of life correlates with patient derived quality of life measures;
- To measure the correlation between the ITP Bleeding Scale and the Bleeding Assessment Tool in refractory pediatric ITP patients.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Second Line ITP agents
- Keywords
- 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, Blood Platelet Disorders, Immune System Diseases, Hemorrhagic Disorders, Autoimmune Diseases, Therapeutic Uses
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic, Purpura, Purpura, Thrombocytopenic, Blood Coagulation Disorders, Hematologic Diseases, Hemorrhage, Skin Manifestations, Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Second Line ITP agentsDrug
Also called rituximab, 6-mercaptopurine, mycophenolate mofetil, sirolimus
The treating physicians will select the second line agent and clinical data will be collected.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Second Line ITP agents |
What is being measured
The primary outcome is the main question the study is designed to answer.
change from baseline in patient reported outcomes
Kids ITP Tool, Memorial Symptom Assessment Scale, Fatigue Scale
Measured: Enrollment, 1 and 12 months
change from baseline in bleeding assessment
ITP Bleeding Scale, Bleeding Assessment Tool
Measured: Enrollment, 1, 6, and 12 months
change from baseline in platelet count
Measured: over 1 year
Secondary outcomes (1)
side effects and complications of treatments
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2013 (estimated)
- Study startAug 2013
- Primary completionApr 2017
- Study completionApr 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 120 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Terrana ITP Research Fund
- Responsible party
- Principal investigator, Rachael Grace, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01971684
- Sponsor's protocol ID
- P00008709
- Acronym
- ICON1
- Status last verified
- May 2020
- First submitted
- 21 Oct 2013
- Last updated
- 21 May 2020
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
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 JM. Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes. Am J Hematol. 2019 Jul;94(7):741-750. doi: 10.1002/ajh.25479. Epub 2019 Apr 29.
PubMed 30945320Grace 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, Lambert MP. Physician decision making in selection of second-line treatments in immune thrombocytopenia in children. Am J Hematol. 2018 Jul;93(7):882-888. doi: 10.1002/ajh.25110. Epub 2018 May 6.
PubMed 29659042
Study at a glance
From the registry record, last updated by the sponsor on 21 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ICON1: Physician Treatment Decisions and Patient-Reported Outcomes in Pediatric Refractory Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT01971684
- Acronym / sponsor ID
- ICON1 / P00008709
- Conditions
- Immune Thrombocytopenia
- Keywords
- ITP, idiopathic thrombocytopenic purpura, bleeding score, platelet count, health-related quality of life, HRQL, Purpura, Purpura, Thrombocytopenic
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 120 participants (actual)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Collaborators
- Terrana ITP Research Fund
- Study start
- Aug 2013
- Primary completion
- Apr 2017 (actual)
- Study completion
- Apr 2017 (actual)
- First posted
- 29 Oct 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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:
- To model factors that determine physician treatment decisions in selecting specific second line agents in pediatric ITP.
- To assess patient reported outcomes with relation to specific second line pediatric ITP therapies.
- To determine the comparative effectiveness of second line ITP treatments in terms of bleeding and platelet counts.
Secondary Objectives:
- To describe phenotypic variation among patients with refractory ITP;
- To assess side effects and complications related to specific treatments for refractory ITP;
- To describe monitoring and follow up practices among pediatric hematologists with each second line agent;
- To weight factors that physicians use when deciding to treat pediatric ITP patients with second line agents;
- To determine whether physician perception of patient quality of life correlates with patient derived quality of life measures;
- To measure the correlation between the ITP Bleeding Scale and the Bleeding Assessment Tool in refractory pediatric ITP patients.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Refractory Pediatric ITP Patients | – | Drug: Second Line ITP agentsPediatric ITP patients, ages 1-18, starting a new second line ITP therapy, defined as not IVIG, steroids, anti-D, or aminocaproic acid. |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Pediatric refractory ITP patients starting on a new second line therapy
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Second Line ITP agentsAlso: rituximab, 6-mercaptopurine, mycophenolate mofetil, sirolimusThe treating physicians will select the second line agent and clinical data will be collected. | Drug | Refractory Pediatric ITP Patients |
Primary outcome
change from baseline in patient reported outcomes
Time frame: Enrollment, 1 and 12 months
Kids ITP Tool, Memorial Symptom Assessment Scale, Fatigue Scale
change from baseline in bleeding assessment
Time frame: Enrollment, 1, 6, and 12 months
ITP Bleeding Scale, Bleeding Assessment Tool
change from baseline in platelet count
Time frame: over 1 year
1 secondary outcome
side effects and complications of treatments
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 May 2020.
- Results publication: PubMed 30945320Grace 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 JM. Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes. Am J Hematol. 2019 Jul;94(7):741-750. doi: 10.1002/ajh.25479. Epub 2019 Apr 29.
- Results publication: PubMed 29659042Grace 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, Lambert MP. Physician decision making in selection of second-line treatments in immune thrombocytopenia in children. Am J Hematol. 2018 Jul;93(7):882-888. doi: 10.1002/ajh.25110. Epub 2018 May 6.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
29 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Immune Thrombocytopenia, run by Boston Children's Hospital at 29 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 May 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
change from baseline in patient reported outcomes (time frame: Enrollment, 1 and 12 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01971684, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 May 2020.