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

  • STATUS
    Recruiting
  • End date
    Nov 30, 2024
  • participants needed
    162
  • sponsor
    Baylor College of Medicine
Updated on 6 October 2021
platelet count
prednisone
thrombocytopenia
immunoglobulins
eltrombopag
igiv
intravenous immunoglobulin
immune globulin
autoimmune thrombocytopenia

Summary

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.

Description

This is a prospective, open label, randomized, two-arm, multi-center Phase 3 trial.

Patients with newly diagnosed ITP are randomized 2:1 to receive the experimental treatment, eltrombopag, or investigator's choice of 3 standard therapies. The primary objective is to determine if the proportion of patients with platelet response is significantly greater in patients treated with eltrombopag compared to those treated with standard therapies.

Details
Condition IDIOPATHIC THROMBOCYTOPENIC PURPURA, Autoimmune disease, Immune Thrombocytopenia, Idiopathic Thrombocytopenic Purpura (ITP), Thrombocytopenic, autoimmune thrombocytopenia
Treatment Eltrombopag, IVIG, Steroids, Rho(D) Immune Globulin
Clinical Study IdentifierNCT03939637
SponsorBaylor College of Medicine
Last Modified on6 October 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

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

Exclusion Criteria

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
Known immediate or delayed hypersensitivity reaction to eltrombopag or its excipient
Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study. Women of childbearing potential (have achieved menarche) must have a negative serum or urine pregnancy test and agree to use basic methods of contraception (if sexually active) or maintain abstinence for the duration of the study. Basic contraception methods include
Total abstinence (when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception
Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy, or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment
Male sterilization (at least 6 months prior to screening). The vasectomized male partner should be the sole partner for that subject
Barrier methods of contraception: Condom or Occlusive cap. For the UK: with spermicidal foam/gel/film/cream/ vaginal suppository
Use of oral, injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception. In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment
Male patients who are sexually active and do not agree to abstinence or to use a condom during intercourse while taking eltrombopag, and for 7 days after stopping treatment
History of alcohol/drug abuse
Presence of a medical condition that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data
Concurrent participation in an investigational study within 30 days prior to enrollment or within 5-half-lives of the investigational product, whichever is longer. Note: parallel enrollment in a non-therapeutic trial such as disease registry or biology study is permitted
Other Eligibility Criteria Considerations All patients and/or their parents or
legal guardians must sign a written informed consent (and assent when
applicable)
Patients and/or parents who are unable to read English at a grade 2 level will be excluded from the patient-reported outcome component of the study. They will not be excluded from all other aspects of the study
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