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 States51 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 range1 Year – 18 Years
SexALL
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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…
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