SOAR Trial: Eltrombopag combined with cyclosporine as first line therapy in patients with severe acquired aplastic anemia with follow-up up to 60-months.

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 50

Inclusion Criteria

1.Patient has signed the Informed Consent (ICF) prior to any screening procedures being performed.
2.Patient is male/female =6 years old at the time of informed consent and able to swallow a tablet.
3.Patient has SAA 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 <20 000/µL
•Absolute reticulocyte count <60 000/µL
4.Normal ECG defined as the following as determined via the mean of a triplicate ECG
• Resting heart rate
6-<12 years: 60-130 bpm
12-<18 years: 60-120 bpm
=18 years: 50-90 bpm
•QTcF at screening <450 msec (for male patients), 460 msec (for female patients)
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 35
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion Criteria

1.Diagnosis of Fanconi anemia.
2.Evidence of a clonal hematologic bone marrow disorder on cytogenetics. Patients with very severe neutropenia (ANC < 200 /µL) will not be excluded initially if cytogenetics are not available or pending. If a clonal disorder is identified, the patient will be excluded.
3.Prior immunosuppressive therapy with cyclosporine, alemtuzumab, rabbit or horse ATG and thrombopoietin receptor (TPO-R) agonists.
4. a. Hypersensitivity to eltrombopag or cyclopsorine or their components.
b. Contraindications to cyclosporine
5.AST or ALT >3 x ULN.
6.Serum creatinine, total bilirubin, and alkaline phosphatase >1.5 x ULN .
7.Patient with liver cirrhosis.
8.Infection not adequately controlled with appropriate therapy.
9.Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient’s ability to consent, be compliant with study procedures, tolerate protocol therapy, or that death within 30 days is likely.
10.Patients with cancer who are not considered cure, are on active chemotherapeutic treatment or who take drugs with hematological effects.
11.Administration of an investigational drug within 30 days or 5 half-lives, whichever is longer, preceding the first dose of study treatment.
12.Pregnancy statements and contraception requirements:
Pregnancy or nursing (lactating) women
Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant (or female partners of male patients), unless they are using highly effective methods of contraception during dosing and for 3 months after stopping medication.
In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment.
13.Not able to understand the investigation nature of the study or to give informed consent.
14.Clinically significant ECG abnormality including cardiac arrhythmias (e. g. ventricular tachycardia) complete left bundle branch block, high grade atrioventricular block, or inability to determine the QTcF interval on the ECG.
15.Presence of cardiac disease, or family history of idiopathic sudden death or congenital long QT syndrome.
16.Risk factors for Torsades de Pointe including uncorrected hypokalemia or hypomagnesemia, or use of concomitant medication(s) with a known risk to prolong the QT interval that cannot be discontinued or replaced by safe alternative medication per www.qtdrugs.org.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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