SOAR Trial Interventional phase II single-arm study to assess efficacy and safety of Eltrombopag combined with cyclosporine as first line therapy in adult patients with severe acquired aplastic anemia.<br>

Phase 2

Withdrawn

Conditions: acquired severe aplastic anemia
10002086

Registration Number: NL-OMON50032

Lead Sponsor: ovartis

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Withdrawn

Sex: Not specified

Target Recruitment: 2

Inclusion Criteria

• Male and female >= 18 years of age.
• Severe aplastic anemia characterized by a. Bone marrow cellularity <30%
(excluding lymphocytes) and b. At least two of the following (peripheral blood):
- Absolute neutrophil count <500/ µL
- Platelet count <20000/ µL
- Absolute reticulocyte count <60000/ µL
• Normal ECG (see protocol page 40 for details).

Exclusion Criteria

• Fanconi anemia.
• Evidence of a clonal hematologic bone marrow disorder. See protocol page 40
for details.
• Prior immunosuppressive therapy with cyclosporine, alemtuzumab, r- or h-ATG
and TPO-R agonists.
• Liver cirrhosis.
• Infection not adequately controlled with appropriate therapy.
• Moribund or concurrent hepatic, renal, cardiac, neurologic, pulmonary,
infectious, cancer or metabolic disease of such severity that it would
interfere with a proper completion of study visits or treatment.
• Pregnant or nursing (lactating) women.
• Females and males not using adequate contraception. See protocol page 45 for
more details.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Overall hematologic response (neutrophil, platelet, hemoglobin) by 6 months.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Overall hematologic response (neutrophil, platelet, hemoglobin) by 3 and 12<br /><br>months, duration of hematologic response, disease relapse rate, clonal<br /><br>evolution, number of transfusions, time without transfusions, overall survival,<br /><br>symptoms and result of quality of life questionnaires, adverse events,<br /><br>pharmacokinetic parameters.</p><br>