SOAR, 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

Phase 1

• Conditions: First-line severe aplastic anaemia; MedDRA version: 20.0Level: PTClassification code 10002967Term: Aplastic anaemiaSystem Organ Class: 10005329 - Blood and lymphatic system disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2016-002814-29-IT
• Lead Sponsor: OVARTIS PHARMA AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-05-24
• Last Update Posted: 17 August 2021

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 > 18 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
•QTcF at screening <450 msec (male patients), 460 msec (female patients)
Are the trial subjects under 18? no
Number of subjects for this age range:
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 clonal bone marrow hematologic disorder on cytogenetics by centralized revision
3. Previous immunosuppressive therapy with cyclosporine, alemtuzumab, rabbit or horse ATG and thrombopoietin receptor agonists (TPO-R).
4. Hypersensitivity to eltrombopag or ciclosporin or their components and contraindications to ciclosporin.
5. AST or ALT > 3 x ULN.
6. Creatinine, total bilirubin and alkaline phosphatase > 3 x ULN.
7. Patients with cirrhosis of the liver.
8. Infection not adequately controlled with appropriate therapy; Patients who are infected with acquired immunodeficiency virus (HIV), hepatitis C virus or are positive for hepatitis B surface antigen (HbsAg). Enlistment is allowed in HCV-RNA negative patients.
9. Agonic state or concomitance of liver, kidney, cardiac, neurological, pulmonary, infectious or metabolic pathology of such severity as to preclude the patient's ability to give consent, adhere to study procedures, tolerate protocol therapy, or patients with a life expectancy of less than 3 months.
10. Patients with cancer who are not considered to be cured, are undergoing active chemotherapy or taking drugs with hematologic effects.
11. Administration of an experimental drug within 30 days or 5 half-lives, whichever is longer, before administration of the first dose of the investigational treatment.
12. Definition of pregnancy and contraceptive requirements: Pregnant or lactating women. Potentially fertile women or female partners of male patients, unless they use a highly effective method of contraception during the administration of treatment and for 3 months after discontinuation of the study treatment.
13. Patients who do not understand the experimental nature of the study or who are unable to provide informed consent.
14. Clinically relevant ECG changes that include cardiac arrhythmias (e.g., ventricular tachycardia), complete left branch block, high grade atrioventricular block, or inability to determine the QTcF interval at the ECG.
15. Presence of heart disease or family history of sudden idiopathic death or congenital long QT syndrome.
16. Risk factors for peak torsion including incorrect hypokalemia or hypomagnesemia, or use of concomitant drug(s) with a known risk of inducing prolonged QT interval that cannot be discontinued.
17. ECOG performance status = 2.
18. Patients under 40 years of age should be considered for bone marrow transplantation (HSCT) if HLA (human leukocyte antigen) comparison has been performed and a suitable compatible sibling donor is available and the patient is willing to undergo the transplant.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified