Registered outcomes12

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

1 to 12 of 12

List	Outcome	Time frame	Description
Primary	Participants That Responded to Eltrombopag	6 months (24 weeks +/- 14 days)	Participants that responded to Eltrombopag as defined by: Response to treatment will be defined by one or more of the following: * Erythroid response for subjects with a pretreatment hemoglobin less than 9 G/dL will be defined as an increase in hemoglobin by \>1.5 G/dL from enrollment baseline, and/or * A reduction in the units of PRBC transfusions by at least 50% during the eight consecutive weeks prior to response assessment - compared with the pretreatment transfusion number in the previous 8 weeks.
Primary	Time (Weeks) to Response	6 months (24 weeks +/- 14 days)	Time-to-response in weeks will also be measured according to the time from Eltrombopag initiation to the first time the patient met criteria for response. Response to treatment will be defined by one or more of the following: * Erythroid response for subjects with a pretreatment hemoglobin less than 9 G/dL will be defined as an increase in hemoglobin by \>1.5 G/dL from enrollment baseline, and/or * a reduction in the units of PRBC transfusions by at least 50% during the eight consecutive weeks prior to response assessment - compared with the pretreatment transfusion number in the previous 8 weeks.
Primary	Number of Adverse Events	6 months (24 weeks +/- 14 days)	Number of Adverse Events Toxicity profile as measured by using the Common Terminology Criteria for Adverse Events (CTCAE version 5.0) for grade 2 and above. According to https://ctep.cancer.gov/, CTCAE is a descriptive terminology which can be utilized for Adverse Event (AE) reporting. A grading (severity) scale is provided for each AE term. CTCAE grades are defined as: Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age appropriate instrumental activities of daily living. Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activities of daily living. Grade 4 Life-threatening consequences; urgent intervention indicated.
Secondary	Number of Participants That Responded to Eltrombopag	3 Months	Number of Participants That Responded to Eltrombopag. As determined by the response to treatment, which will be assessed based on one or more of the following criteria: Erythroid response for subjects with a pretreatment hemoglobin less than 9 G/dL will be defined as an increase in hemoglobin by \>1.5 G/dL from enrollment baseline, and/or A reduction in the units of PRBC transfusions by at least 50% during the eight consecutive weeks prior to response assessment - compared with the pretreatment transfusion number in the previous 8 weeks.
Secondary	Number of Participants With Robust Response to Eltrombopag	3 Months, 6 Months, Up to 27 Months	Number of Participants with Robust Response to Eltrombopag regardless when it is achieved. Best response is defined as robust response or not robust response. Robust response to treatment will be defined both: * Stable hemoglobin (two consecutive measurements at least 2 weeks apart) greater than 10 g/dL, and * Transfusion independence for the previous 8 weeks
Secondary	Median Change in Platelet Count	Baseline, 3 months	Median Change in Platelet Count as measured by serial CBC assessments
Secondary	Median Change Absolute Neutrophil Count	Baseline, Month 3	Median Change Absolute Neutrophil Count as measured by serial CBC assessments
Secondary	Number of Participants That Experienced Relapse	6 months up to 27 months	Number of Participants that experienced Relapse during extension phase. Relapse is defined as no longer meeting response criteria. Response criteria is defined as: Response to treatment will be defined by one or more of the following: * Erythroid response for subjects with a pretreatment hemoglobin less than 9 G/dL will be defined as an increase in hemoglobin by \>1.5 G/dL from enrollment baseline, and/or * A reduction in the units of PRBC transfusions by at least 50% during the eight consecutive weeks prior to response assessment - compared with the pretreatment transfusion number in the previous 8 weeks.
Secondary	Number of Participants That Experienced Clonal Evolution	6 Months and Up to 27 months	Number of participants that experienced clonal evolution according to cytogenetic, mutational or flow cytometric markers
Secondary	Number of Adverse Events During the Extension Phase	6 months Up to 27 months	Number of Adverse Events Toxicity profile as measured by using the Common Terminology Criteria for Adverse Events (CTCAE version 5.0) for grade 2 and above. According to https://ctep.cancer.gov/, CTCAE is a descriptive terminology which can be utilized for Adverse Event (AE) reporting. A grading (severity) scale is provided for each AE term. CTCAE grades are defined as: Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age appropriate instrumental activities of daily living. Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activities of daily living. Grade 4 Life-threatening consequences; urgent intervention indicated.
Secondary	Neurodevelopment in Pediatric Patients	At baseline and Month 6	Neurodevelopment in pediatric patients as measured by validated neurodevelopmental measurements
Secondary	Number of Participants With Iron Overload	Month 6	Number of participants with Iron overload. Iron overload as measured by serial ferritin levels, T2\* MRI measurements and bone marrow iron staining

Source ClinicalTrials.govtrials-2026-11-12-0.5.0

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 list from software.

Twenty rows a page, as JSON. No key.

GET https://conductscience.org/api/trials/v1/NCT04269889/registered

API reference For AI assistants