A phase II, open-label, non-controlled, intra-patient dose-escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in pediatric patients with refractory, relapsed or treatment naïve severe aplastic anemia or recurrent aplastic anemia (CETB115E2201)
- Conditions
- severe aplastic anemia10002086
- Registration Number
- NL-OMON50357
- Lead Sponsor
- ovartis
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Withdrawn
- Sex
- Not specified
- Target Recruitment
- 2
* Cohort-specific criteria: see protocol page 46.
* Male and female 1 up to 18 years.
* Aplastic anemia.
* Hematopoietic stem cell transplantation not suitable or available as a
treatment option or has been refused by the subject.
* Bone marrow aspirate and biopsy at any time during the 4 weeks prior to first
dose of eltrombopag.
* Normal karyotype with FISH for chromosomes 7 and 8.
* Performance status score: Karnofsky *50 for patients 16 years of age and
older or Lansky *50 for patients below 16 years of age.
* Prior and/or active history of several diseases, see protocol page 47 for
details.
* Active infection not responding to appropriate therapy.
* Prior eltrombopag or other thrombopoietin receptor (TPO-R) agonist treatment
for at least 2 months and a lack of response.
* Impaired cardiac function. See protocol page 48 for details.
* Pregnant or nursing (lactating) women.
* Females of childbearing potential and males not using adequate contraception.
See protocol page 48 for more details.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Eltrombopag PK parameters at week 2 and 12 (incl. highest dose level at<br /><br>steady-state).</p><br>
- Secondary Outcome Measures
Name Time Method <p>(key only, see protocol page 40-41 for all objectives) Adverse events.<br /><br>Proportion of patients with an overall response at week 12, 26, 52, 78 and<br /><br>proportion of patients with a platelet response.</p><br>