Trial · NCT03025698
A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia
NCT03025698, Phase 2, Completed, Novartis Pharmaceuticals
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia. Trials, record NCT03025698. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03025698
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia
- NCT number
- NCT03025698
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 51 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Aplastic Anemia
- Interventions
- Eltrombopag, hATG, CsA
- First posted
- 2017-01-19
- Last updated
- 2026-08-10
- Other registries' IDs
- eudract:2015-003166-91
Source ClinicalTrials.gov
Registered outcomes27
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| 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. |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 40315366 | DERIVED |
Source 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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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.
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.
GET https://conductscience.org/api/trials/v1/NCT03025698
GET https://conductscience.org/api/trials/v1/NCT03025698/registered?page=2