Trial · NCT03025698
Registered outcomes27
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 27
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Eltrombopag PK Parameters: AUCtau, AUClast | at least 11 weeks after dose initiation or later when patients are taking the highest dose, up to Week 78 | AUC tau: Area under the curve calculated to the end of the dosing interval ( tau) (mass\*time/volume) AUC last: Area under the curve calculated to the last quantifiable concentration point (Tlast) (mass\*time/volume) |
| Primary | Eltrombopag PK Parameter: Cmax | at least 11 weeks after dose initiation or later when patients are taking the highest dose, up to Week 78 | Cmax is the observed maximum plasma concentration following administration (mass/volume) |
| Primary | Eltrombopag PK Parameter: Ctrough at the Highest Dose Level | at least 11 weeks after dose initiation or later when patients are taking the highest dose, up to Week 78 | Ctrough is the pre-dose plasma concentration (mass/volume). |
| Secondary | Percentage of Participants With an Overall Response Rate and Percentage of Participants With a Platelet Response Rate Based on Per Pediatric Committee of European Medicines Agency (PDCO) | Week 12, Week 26, Week 52, Week 78 | Overall response rate (ORR) is defined as the percentage of participants who have achieved a complete response (CR) or partial response (PR) or No response (NR) by the Investigator. CR criteria: Platelet (PLT) and red blood cell (RBC) transfusion independence, Normal age-adjusted Hgb, PLT \>100 × 10\^9/L and absolute neutrophil count (ANC) \>1.5 × 10\^9/L. PR: PLT and RBC Transfusion independence and at least 2 of the following criteria: Reticulocytes \>30 × 10\^9/L, PLT \>30 × 10\^9/L, ANC \>1.5 x 10\^9L. PLT transfusion independence is defined as a period for at least 28 days without PLT transfusion. Platelet response rate (PRR): Platelet response rate is comprised of CR + PR based on the following criteria: CR: PLT \>100 × 10\^9/L; PR: PLT \>30 × 10\^9/L; NR: PLT transfusion within 4 weeks or PLT \<= 30 × 10\^9/L. |
| Secondary | Hematologic Counts (Platelets (Blood), Neutrophils (Blood)) | Week 12, Week 26, Week 52, Week 78, Week 130, Week 182, Week 234 | Individual Platelets (PLT) and neutrophil counts were summarized for all participants. |
| Secondary | Hematologic Counts (Hemoglobin (Blood)) | Week 12, Week 26, Week 52, Week 78, Week 130, Week 182, Week 234 | Individual hemoglobin (Hgb) counts were summarized for all participants. |
| Secondary | Number of Red Blood Cells (RBC) Transfusions | From date of first dose to approx. 4.5 years | Number of transfusions during the treatment period refers to total number of RBC transfusions participants have received while on treatment. |
| Secondary | Frequency of Red Blood Cell (RBC) Transfusions | From date of first dose to approx. 4.5 years | Frequency of transfusions during the treatment period refers to number of RBC transfusions during the treatment period divided by number of months of treatment duration. |
| Secondary | Number of Platelet (PLT) Transfusions | From date of first dose to approx. 4.5 years | Number of transfusions during the treatment period refers to total number of PLT transfusions participants have received while on treatment. |
| Secondary | Frequency of Platelet (PLT) Transfusions | From date of first dose to approx. 4.5 years | Frequency of transfusions during the treatment period refers to number of PLT transfusions during the treatment period divided by number of months of treatment duration. |
| Secondary | Total Duration of Red Blood Cell (RBC) Transfusion Independence During the Treatment Period | From date of first dose to approx. 4.5 years | RBC transfusion independence is defined as a period of time of at least 56 days without RBC transfusion. Duration of RBC transfusion independence is defined as a period of time of at least 56 days without RBC transfusion. First transfusion duration was calculated as the date of the day before the first transfusion after baseline minus the date of first exposure eltrombopag + 1. |
| Secondary | Total Duration of Platelet (PLT) Transfusion Independence During the Treatment Period | From date of first dose to approx. 4.5 years | Platelet transfusion independence during the treatment period is defined as the duration from the first day of the 28-day period without PLT transfusion until the occurrence of a PLT transfusion after the period free from any PLT transfusion. Duration of PLT transfusion independence is defined as a period of time of at least 28 days without platelet transfusion. First transfusion duration was calculated as the date of the day before the first transfusion after baseline minus the date of first exposure eltrombopag + 1. |
| Secondary | Maximum Duration of Red Blood Cell (RBC) Transfusion Independence | From date of first dose to approx. 4.5 years | RBC transfusion independence is defined as a period of time of at least 56 days without RBC transfusion. Maximum duration of RBC transfusion independence is defined as the maximum duration among the durations of RBC transfusion independence. First transfusion duration was calculated as the date of first transfusion after baseline minus the date of first exposure eltrombopag + 1. |
| Secondary | Maximum Duration of Platelet (PLT) Transfusion Independence | From date of first dose to approx. 4.5 years | Maximum duration of PLT transfusion independence is defined as the maximum duration among the durations of PLT transfusion independence. First transfusion duration was calculated as the date of first transfusion after baseline minus the date of first exposure eltrombopag + 1. |
| Secondary | Overall Bone Marrow Cellularity | Screening, Week 26, Week 52, Week 78, Week 130, Week 182, Week 234 | Percentage of cells in bone marrow biopsy - a comprehensive diagnostic evaluation to distinguish between the various bone marrow disorders. |
| Secondary | Bone Marrow Morphology | Screening, Week, 26, Week 52, Week 78, Week 130, Week 182, Week 234 | Percentage of morphology (erythropoiesis, granulopoiesis, megakaryopoiesis, CD34+ (blast cells) cells in bone marrow aspirate - a comprehensive diagnostic evaluation to distinguish between the various bone marrow disorders. |
| Secondary | Bone Marrow Cytogenetics | Screening, Week 12, Week 26, Week 52, Week 78, Week 130, Week 182, Week 234 | Number of bone marrow cytogenetics (chromosomal structure) by kryotyping and Fluorescence in situ hybridization (FISH). This is a comprehensive diagnostic evaluation to distinguish between the various bone marrow disorders. |
| Secondary | Acceptability and Palatability for Both Tablets and Powder Formulation for Oral Solution (PfOS) | any day from Day 1 of drug initiation up to Week 78 | Standardized (total) summary score, ranged from 0-100, (where 0 means worst and 100 means the best), was derived from all items from the questionnaire based on a scoring matrix. The questionnaire was completed by parents and caregivers of patients under 12 years of age (ObsRO) and a questionnaire completed by patients 12 years and older (PRO). |
| Secondary | Clonal Evolution to Paroxysmal Nocturnal Hemoglobinuria (PNH) | Baseline, Week (W) 26 Day (D) 1, W52D1, W78D1, W130D1, W182D1, W234D1 | Percentage of participants with clonal evolution to Paroxysmal Nocturnal Hemoglobinuria (PNH). |
| Secondary | Exposure (AUCtau) - Response Relationship of Eltrombopag and Best Overall Response Rate by Age Groups | at least 11 weeks after dose initiation or later when patients are taking the highest dose, up to Week 78 | Pharmacokinetic parameter (AUCtau) of eltrombopag at the highest dose in relationship to best overall response rate in regard to complete response (CR), partial response (PR) and no response (NR). AUC tau: Area under the curve calculated to the end of the dosing interval (tau) (mass\*time/volume) |
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.
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