Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children

Completed · Phase 3

Conditions studied: Immune Thrombocytopenia

In brief

This is an investigator initiated, multicenter, open label, randomized phase 3 study for subjects with newly diagnosed ITP from ages 1 to less than 18 years old.

Key facts

Study ID

NCT03939637

Run by

Baylor College of Medicine

People needed

122

Starts

2019-05-02

Expected to finish

2025-02-26

Last updated by the study team

2025-07-29

Who can join

Age: 1 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age: 1- <18 years
• Newly diagnosed ITP (<3 months from diagnosis (first abnormal platelet count), per international working group definition17)
• Platelets <30 x 10\^9/L at screening
• Requires pharmacologic treatment from the perspective of the treating clinician.
• Need to treat is at the discretion of the investigator, but there should be clinical equipoise about the use of eltrombopag vs standard treatment options (patients should not, in the opinion of the investigator, require concomitant therapy at time of enrollment).
• Treatment options include one of three standard therapies, (IVIg, steroids, or Anti-D). For example, if patient has previously shown no response to IVIg or steroids and is Rh-negative, patient would not be eligible for study.
• Patient population includes both:
• Upfront treatment: Patient within 10 days of ITP diagnosis who has not received previous treatment OR
• Treatment failure: Patients who have failed standard management (observation or treatment with one or more first-line agents)
• Failure of observation: no platelet recovery (>30 x 10\^9/L) with observation >10 days from diagnosis, with need to treat
• Poor response to first-line agent (platelets remain <30 x10\^9/L)
• Initial response to first-line agent, but response wanes and platelets fall below 30 x10\^9/L
• Family willing and able to return for required lab studies

You may not qualify if…

• Severe bleeding: Buchanan Overall Grade 4 or 5 bleeding, or severe bleeding requiring emergent treatment at the discretion of the provider. (e.g., intracranial hemorrhage, pulmonary hemorrhage, bleeding with ongoing need for pRBC transfusion)
• Prior treatment with TPO-RA (eltrombopag or romiplostim)
• Known secondary ITP (due to lupus, CVID, ALPS)
• Known HIV (or history of HIV positivity) or Hepatitis C (screening not required if no clinical suspicion)
• Evans Syndrome: positive direct Coombs with evidence of active hemolysis (elevated lactate dehydrogenase (LDH) or reticulocyte count not attributable to recent treatment or bleeding)
• Any Malignancy
• History of stem cell transplant or solid organ transplant
• aspartate aminotransferase (AST) or ALT >2 x upper limit of normal (ULN)
• Total bilirubin >1.5 × ULN
• Subjects with liver cirrhosis (as determined by the investigator)
• Creatinine >2.5 × ULN
• Known active or uncontrolled infections not responding to appropriate therapy
• On anticoagulation or anti-platelet agents
• Known thrombophilic risk factors. Exception: Subjects for whom the potential benefits of participating in the study outweigh the potential risks of thromboembolic events, as determined by the investigator.
• Baseline ophthalmic problems that may potentiate cataract development
• Impaired cardiac function, such as:
• Known prolonged QTc, with corrected QTc >450 msec
• Other clinically significant cardio-vascular disease (e.g., uncontrolled hypertension, history of labile hypertension),
• History of known structural abnormalities (e.g. cardiomyopathy).
• History or current diagnosis of cardiac disease indicating significant risk of safety for patients participating in the study such as uncontrolled or significant cardiac disease, including any of the following:
• Recent myocardial infarction (within last 6 months),
• Uncontrolled congestive heart failure,
• Unstable angina (within last 6 months),
• Clinically significant (symptomatic) cardiac arrhythmias (e.g., sustained ventricular tachycardia, and clinically significant second or third degree AV block without a pacemaker.)
• Long QT syndrome, family history of idiopathic sudden death, congenital long QT syndrome or additional risk factors for cardiac repolarization abnormality, as determined by the investigator.

Where it is running

• Children's of Alabama — Birmingham, Alabama, United States
• Phoenix CHildren's Hospital — Phoenix, Arizona, United States
• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• Children's Hospital of Orange County — Orange, California, United States
• UCSF Benioff Children's Hospital — San Francisco, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Children's National Medical Center — Washington D.C., District of Columbia, United States
• University of Florida College of Medicine — Gainesville, Florida, United States
• Alfac Cancer and Blood Disorder Center: Scottish Rite — Atlanta, Georgia, United States
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• Riley Hospital for Children-Indiana University — Indianapolis, Indiana, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Children's Hospital and Clinics of Minnesota — Minneapolis, Minnesota, United States
• University of Nebraska Medical Center — Omaha, Nebraska, United States
• Hackensack University Medical Center — Hackensack, New Jersey, United States
• Columbia University Irving Medical Center — New York, New York, United States
• Weill Cornell Medical College — New York, New York, United States
• Levine Cancer Institute — Charlotte, North Carolina, United States
• Duke University Medical Center — Durham, North Carolina, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Randall Children's Hospital — Portland, Oregon, United States
• Oregon Health and Science University — Portland, Oregon, United States
• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Hasbro Children's Hospital — Providence, Rhode Island, United States
• St. Jude Children's Hospital — Memphis, Tennessee, United States

Full record on ClinicalTrials.gov

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