Trial · NCT03939637

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

NCT03939637, Phase 3, Completed, Baylor College of Medicine; results first posted 2025-07-29

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children. Trials, record NCT03939637. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03939637

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children
NCT number: NCT03939637
Status: Completed
Phases: Phase 3
Enrollment: 122 participants, actual
Lead sponsor: Baylor College of Medicine
Conditions: Immune Thrombocytopenia
Interventions: Eltrombopag, Steroids, IVIG, Rho(D) Immune Globulin
First posted: 2019-05-07
Last updated: 2025-07-29
Results first posted: 2025-07-29

Source ClinicalTrials.gov

Registered outcomes22

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Proportion of Patients With a Platelet Response	12 weeks	To determine if the percentage 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	Bleeding Score	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
Secondary	Cumulative Number of Rescue Therapies Required	12 weeks	The percentage of patients who received rescue therapy in the experimental (eltrombopag) arm vs the comparator (standard therapy) arm during the first 12 weeks of treatment
Secondary	Platelet Response Among Patients Requiring Rescue Therapy During Weeks 1-2 of Study	12 weeks	Platelet response (binary), defined as ≥ 3 of 4 weeks with platelets \>50 x109/L during weeks 6-12 of therapy, but patient required a rescue treatment during weeks 1-2 of study.
Secondary	Need for Treatment	6 months	No further need for treatment (binary) after 12 weeks or 6 months of study
Secondary	Treatment Response	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.
Secondary	Number of 2nd Line Therapies	52 weeks	Number of 2nd-line therapies in weeks 13-52
Secondary	Regulatory T-Cells	1 year	Absolute change in percentage of CD4+25+Foxp3+ regulatory T cells from baseline at 12 weeks and 1 year
Secondary	KIT Scores	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
Secondary	Hockenberry Fatigue Scale-Parent	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

1 to 10 of 22Source ClinicalTrials.gov

See all 22 registered outcomes

Outcomes22

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Proportion of Patients With a Platelet Response	12 weeks	This study included 118 pediatric patients (median age 8y, 49% male), 78 randomized to eltrombopag and 40 to standard therapy.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Bleeding Score	1 year
SECONDARY	Cumulative Number of Rescue Therapies Required	12 weeks	Patients who received \>/= 1 dose of protocol-directed therapy were evaluable for secondary objectives.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Platelet Response Among Patients Requiring Rescue Therapy During Weeks 1-2 of Study	12 weeks	patient required a rescue treatment during weeks 1-2 of study.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Need for Treatment	6 months
SECONDARY	Treatment Response	1 year
SECONDARY	Number of 2nd Line Therapies	52 weeks
SECONDARY	Regulatory T-Cells	1 year
SECONDARY	KIT Scores	1 year
SECONDARY	Hockenberry Fatigue Scale-Parent	1 year

1 to 10 of 22Source ClinicalTrials.gov

See all 22 outcomes

Results by group6

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Units
Proportion of Patients With a Platelet Response	Experimental: Eltrombopag	52	Participants
Proportion of Patients With a Platelet Response	Comparator: Standard Therapy	14	Participants
Cumulative Number of Rescue Therapies Required	Experimental: Eltrombopag	13	Participants
Cumulative Number of Rescue Therapies Required	Comparator: Standard Therapy	15	Participants
Platelet Response Among Patients Requiring Rescue Therapy During Weeks 1-2 of Study	Experimental: Eltrombopag	4	Participants
Platelet Response Among Patients Requiring Rescue Therapy During Weeks 1-2 of Study	Comparator: Standard Therapy	0	Participants

Source ClinicalTrials.gov

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Experimental: Eltrombopag	81
Overall Study	STARTED	Comparator: Standard Therapy	41
Overall Study	COMPLETED	Experimental: Eltrombopag	78
Overall Study	COMPLETED	Comparator: Standard Therapy	40
Overall Study	NOT COMPLETED	Experimental: Eltrombopag	3
Overall Study	NOT COMPLETED	Comparator: Standard Therapy	1

Source ClinicalTrials.gov

Adverse events28

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Anemia	Blood and lymphatic system disorders	Experimental: Eltrombopag	1	78	1
other	Anemia	Blood and lymphatic system disorders	Comparator: Standard Therapy	0	39	0
other	Epistaxis	Ear and labyrinth disorders	Experimental: Eltrombopag	1	78	1
other	Epistaxis	Ear and labyrinth disorders	Comparator: Standard Therapy	0	39	0
other	Fall	Injury, poisoning and procedural complications	Experimental: Eltrombopag	1	78	1
other	Fall	Injury, poisoning and procedural complications	Comparator: Standard Therapy	0	39	0
other	Headache	Nervous system disorders	Experimental: Eltrombopag	3	78	3
other	Headache	Nervous system disorders	Comparator: Standard Therapy	3	39	3
other	Suicidal ideation	Psychiatric disorders	Experimental: Eltrombopag	1	78	1
other	Suicidal ideation	Psychiatric disorders	Comparator: Standard Therapy	0	39	0

1 to 10 of 28Source ClinicalTrials.gov

See all 28 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Experimental: Eltrombopag	0	78
Total, all-cause mortality	Comparator: Standard Therapy	0	39
Total, other adverse events	Experimental: Eltrombopag	8	78
Total, other adverse events	Comparator: Standard Therapy	3	39
Total, serious adverse events	Experimental: Eltrombopag	6	78
Total, serious adverse events	Comparator: Standard Therapy	3	39

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
34452956	DERIVED	PubMed
41123939	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons5

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Experimental: Eltrombopag vs Comparator: Standard Therapy: Cumulative Number of Rescue Therapies Required	Trial comparisons
Experimental: Eltrombopag vs Comparator: Standard Therapy: Proportion of Patients With a Platelet Response	Trial comparisons
Experimental: Eltrombopag vs Comparator: Standard Therapy: Platelet Response Among Patients Requiring Rescue Therapy During Weeks 1-2 of Study	Trial comparisons
Experimental: Eltrombopag vs Comparator: Standard Therapy: deaths from any cause	Trial comparisons
Experimental: Eltrombopag vs Comparator: Standard Therapy: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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