A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With … (NCT03025698) | Clinical Trial Compass
CompletedPhase 2
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
United States, Hong Kong, Portugal51 participantsStarted 2017-09-30
Plain-language summary
This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics (PK) after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric patients with previously untreated or relapsed/refractory severe aplastic anemia or recurrent aplastic anemia.
Who can participate
Age range
1 Year – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
For Cohort A patients:
* History of prior diagnosis of SAA,
* Diagnosis of relapsed/refractory SAA or recurrent AA following treatment for SAA, as per Section 5.1. Patients with recurrent AA (e.g., losing their response) are exempt from meeting the diagnostic criteria for SAA relapse at the time of study enrollment, but must have been previously diagnosed with SAA.
* Agree to concurrent eltrombopag treatment with appropriate, investigator-selected Immunosuppressive therapy (IST) with either hATG + CsA or CsA.
For Cohort B patients:
* Diagnosis of SAA at time of enrollment.
* Patients must not have been previously treated with IST, and must meet all criteria as described in Table 5-1.
* Patients must agree to treatment with hATG + CsA concurrent with eltrombopag.
All patients eligible for inclusion in this study must meet all of the following criteria:
* Age 1 to \<18 years.
* Assessments to rule out congenital/inherited bone marrow failure syndromes and other causes of immune-mediated pancytopenia, which may be treated with transplant, must be completed prior to enrollment.
* Hematopoietic stem cell transplantation (HSCT) is not suitable or available as a treatment option or has been refused by the patient. (Candidacy for HSCT will be determined as per local practices or national guidelines.)
* Bone marrow aspirate and biopsy at any time during the 4 weeks prior to first dose of eltrombopag.
* Performance status score: Karnofsky ≥50 for patients 16 y…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Eltrombopag PK parameter: AUCtau
Timeframe: at the highest dose level, i.e. 11 weeks after dose initiation
2
Eltrombopag PK parameter: Cmax
Timeframe: at the highest dose level, i.e. 11 weeks after dose initiation
3
Eltrombopag PK parameter: Ctrough
Timeframe: at the highest dose level, i.e. 11 weeks after dose initiation