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Clinical trial information and results are updated daily from ClinicalTrials.gov. The latest data update was conducted on 01/28/2021.

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

Clinicaltrials.gov identifier NCT03939637

Recruitment Status Recruiting

First Posted May 7, 2019

Last update posted July 21, 2020

Study Description

Brief 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.

  • Condition or Disease:Immune Thrombocytopenia
  • Intervention/Treatment: Drug: Eltrombopag
    Drug: Steroids
    Drug: IVIG
    Drug: Rho(D) Immune Globulin
  • Phase: Phase 3
Detailed 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.

Study Design
  • Study Type: Interventional
  • Estimated Enrollment: 162 participants
  • Allocation: Randomized
  • Intervention Model: Parallel Assignment
  • Masking: None (Open Label) ()
  • Primary Purpose: Treatment
  • Official Title: A Phase III Study of Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia in Children
  • Actual Study Start Date: May 2019
  • Estimated Primary Completion Date: May 2022
  • Estimated Study Completion Date: November 2024
Arms and interventions
Arm Intervention/treatment
Experimental: Eltrombopag
Patients randomized to eltrombopag will be treated for 12 weeks, with the possibility to continue therapy for up to 1 year depending on response.
Drug: Eltrombopag
Starting dose for eltrombopag will be based on manufacturer recommendations, and drug will be titrated to effect per guidelines. Children 1 to 5 years: Initial: 25 mg once daily Children ≥6 years and Adolescents: Initial: 50 mg once daily (25 mg once daily for patients of East-Asian ethnicity [e.g., Chinese, Japanese, Korean, Taiwanese]) Dose should be titrated based on platelet response. Maximum dose: 75 mg once daily.
Active Comparator: Standard first-line therapy
Subjects randomized to the standard therapy arm will receive one of three treatments at the discretion of the treating physician. Patients who previously failed standard management prior to study entry must be treated with a different agent than their original failed agent. e.g. Patient who failed steroids could receive either IVIg or anti-D if randomized to the standard treatment arm. Standard therapy will be administered as commercially available drug. Investigator may choose amongst the following: IVIg: IVIG 1 g/kg x1 (no steroids for pre-medication or adjunctive therapy) Steroids: Prednisone/Prednisolone 4 mg/kg/day (Max 120 mg/day) x 4 days Rho(D) Immune Globulin: Anti-D globulin 75 mcg/kg x1 (no steroids for pre-medication or adjunctive therapy)
Drug: Steroids
Prednisone/Prednisolone 4mg/kg/day (Max 120 mg/day) x 4 day

Drug: IVIG
IVIG 1 g/kg x1 (no steroids for pre-medication or adjunctive therapy)

Drug: Rho(D) Immune Globulin
Anti-D globulin 75 mcg/kg x1 (no steroids for pre-medication or adjunctive therapy)
Outcome Measures
  • Primary Outcome Measures: 1. Proportion of patients with a platelet response [ Time Frame: 12 weeks ]
    To determine if the proportion of patients with a platelet response is significantly greater in patients with newly diagnosed ITP treated with eltrombopag than those treated with standard first-line treatments
  • Secondary Outcome Measures: 1. Blood Iron values [ Time Frame: 1 year ]
    Serum iron, total iron binding capacity (TIBC), transferrin saturation, ferritin, mean corpuscular volume (MCV), and hemoglobin at 12 weeks, 6 months, and 1 year after study enrollment
  • 2. Bleeding Score [ Time Frame: 1 year ]
    Poor bleeding score (binary) at 1, 2, 3, 4 weeks, 12 weeks, and 1 year after study enrollment defined as World Health Organization (WHO) Bleeding Scale ≥ 2 or Modified Buchanan Scale ≥ 3
  • 3. Number of rescue therapies [ Time Frame: 12 weeks ]
    Cumulative number of rescue therapies required during the first 12 weeks of treatment
  • 4. Platelet response [ Time Frame: 12 weeks ]
    Platelet response (binary), defined as ≥ 6 of 8 weeks with platelets >50 x10^9/L during weeks 5-12 of therapy, but patient required a rescue treatment during weeks 1-2 of study
  • 5. Need for treatment [ Time Frame: 6 months ]
    No further need for treatment (binary) after 12 weeks or 6 months of study
  • 6. Treatment response [ Time Frame: 1 year ]
    Treatment response (binary endpoints) at 1 year defined as: CR is defined as platelet count >/= 150 x 10^9/L Primary Remission at 1 year is defined as CR at 1 year with no second-line agents required and >/= 3 months after discontinuing most recent platelet active medication Disease resolution at 1 year is defined as complete response (CR) at 1 year >/= 3 months after discontinuing most recent platelet active medication. May have received a second-line therapy, excluding rituximab or splenectomy. Disease stability at 1 year is defined as platelets >/= 50 x 10^9/L but <150 x 10^9/L >/= 3 months after discontinuing most recent platelet active medication.
  • 7. Number of 2nd line therapies [ Time Frame: 52 weeks ]
    Number of 2nd-line therapies in weeks 13-52
  • 8. Regulatory T-Cells [ Time Frame: 1 year ]
    Absolute change in percentage of CD4+25+Foxp3+ regulatory T cells from baseline at 12 weeks and 1 year
  • 9. KIT Scores [ Time Frame: 1 year ]
    Change in parent proxy-reported Kids ITP tool (KIT) overall scores from baseline at 1 week, 4 weeks, 12 weeks, and 1 year after study enrollment
  • 10. Hockenberry Fatigue Scale-Parent [ Time Frame: 1 year ]
    Total scale intensity ratings (continuous) from the Hockenberry Fatigue Scale-Parent (FS-P) at 1 week, 4 weeks, 12 weeks, and 1 year
  • 11. Safety evaluations [ Time Frame: 1 year ]
    Safety evaluations as defined by: Abnormal liver function tests (LFTs): ALT ≥ 3 x upper limit of normal (ULN) in patients with normal baseline ALT ≥ 3 x baseline or ≥ 5 x ULN (whichever is lower) in patients with abnormal baseline ALT ≥ 3 x ULN AND bilirubin ≥ 1.5 x ULN (>35% direct) Incidence of adverse events Incidence of serious adverse events
  • Other Outcome Measures: 1. Time to response [ Time Frame: 1 year ]
    Time to response (platelets >30x10^9/L, and at least 2-fold increase in the baseline count and absence of bleeding) (IWG definition)
  • 2. Platelet-specific endpoints [ Time Frame: 1 year ]
    Treatment response (platelets >30x10^9/L, and at least 2-fold increase in the baseline count and absence of bleeding) (IWG definition) at 12 weeks
  • 3. Time to platelet count [ Time Frame: 1 year ]
    Time to platelet count >100x10^9/L and absence of bleeding (IWG definition)
  • 4. Treatment response [ Time Frame: 1 year ]
    Treatment response (platelet count >100x10^9/L and absence of bleeding) (IWG definition) at 12 weeks
  • 5. Loss of treatment response [ Time Frame: 1 year ]
    Loss of treatment response (platelet count below 30x10^9/L, or less than 2-fold increase in the baseline count or bleeding) (IWG definition) at any time during the study period after achieving response during the first 12 weeks
  • 6. Extreme thrombocytosis [ Time Frame: 1 year ]
    Extreme thrombocytosis (platelets >1 x10^12/L)
  • 7. Patient-reported outcomes endpoints [ Time Frame: 1 year ]
    Change in child self-reported and parent impact KIT scores from baseline at 1 week, 4 weeks, 12 weeks, and 1 year after study enrollment
  • 8. Change in Hockenberry fatigue [ Time Frame: 1 year ]
    Change in Hockenberry fatigue (FS-C, FS-A, FS-P) scores from baseline at 1 week, 4 weeks, 12 weeks, and 1 year after study enrollment
  • 9. Global Change Scale scores [ Time Frame: 1 year ]
    Global Change Scale scores at 1 week, 4 weeks, 12 weeks, and 1 year after study enrollment
  • 10. Number of Hospitalizations [ Time Frame: 1 year ]
    Number of hospitalizations
Eligibility Criteria
  • Ages Eligible for Study: 1 to 18 Year (Child, Adult)
  • Sexes Eligible for Study: All
  • Accepts Healthy Volunteers: No
Criteria

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) with observation
>10 days from diagnosis, with need to treat

- Poor response to first-line agent (platelets remain 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

Contacts and Locations
Contacts

Contact: Jenny Despotovic, DO 832-822-4302 jmdespot@txch.org

Locations
Show 11 Study Locations
Sponsors and Collaborators

Baylor College of Medicine

Boston Children's Hospital

University of California, San Francisco

Investigators

Principal Investigator: Jenny Despotovic, DO Baylor College of Medicine/Texas Children's Hospital

More Information
  • Responsible Party: Baylor College of Medicine
  • ClinicalTrials.gov Identifier: NCT03939637 History of Changes
  • Other Study ID Numbers: H-42131 ICON 3, CETB115JUS33T, ICON 3
  • First Posted: May 7, 2019 Key Record Dates
  • Last Update Posted: July 21, 2020
  • Last Verified: July 2020
  • Studies a U.S. FDA-regulated Drug Product: Yes
  • Studies a U.S. FDA-regulated Device Product: No
  • Keywords provided by Baylor College of Medicine: Immune Thrombocytopenia
    Newly Diagnosed Immune Thrombocytopenia
    Newly Diagnosed ITP
    ITP
  • Additional relevant MeSH terms: Thrombocytopenia Purpura, Thrombocytopenic, Idiopathic