Study to Assess the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects.

A Non-randomized, Open-label, Multi-center, Phase II Study to Assess the Safety and Efficacy of Eltrombopag in Chinese Subjects With Refractory or Relapsed Severe Aplastic Anemia

ClinicalTrials.gov Identifier: NCT03988608

Novartis Reference Number: CETB115E2202

Last Update: Sep 28, 2020

All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

This is a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Chinese subjects with refractory or relapsed severe aplastic anemia (SAA). Treatment with eltrombopag will be started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count up to 150 mg/day. The hematological response rate will be assessed at 3, 6 months and 1 year after starting the study treatment (Week 13, 26 and 52).

Condition

Aplastic Anemia

Phase

Phase 2

Overall status

Recruiting

Enrollment count

20 participants

Start date

Dec 09, 2019

Completion date

Jul 24, 2023

Gender

All

Age(s)

18 Years and older (Adult, Older Adult)

Interventions

Drug

Eltrombopag 25 mg

film-coated tablets containing 25 mg of eltrombopag free acid in each tablet

Eligibility Criteria

Inclusion Criteria:

Chinese patients aged greater than or equal to 18 years old.
Patient with a previous diagnosis of severe aplastic anemia and had insufficient response following at least one treatment course in the period time of > 6 months of immunosuppression with a regimen containing anti-thymocyte globulin (ATG), anti-lymphocyte globulin (ALG), and/or cyclophosphamide, or alemtuzumab.
Platelet count ≤ 30 × 10^9/L at screening.
Patient must not currently have the option of stem cell transplantation.
Patient has an Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
Patient with QTcF (Fridericia's QT correction formula) at screening <450 msec, or <480 msec with bundle branch block, as determined via the mean of a triplicate ECG and assessed at site.

Exclusion Criteria:

Treatment with ATG/ALG, cyclophosphamide or alemtuzumab in the past 6 months.
Congenital aplastic anemia
AST or ALT ≥3 times the upper limit of normal.
Creatinine, total bilirubin, and alkaline phosphatase (ALP) ≥ 1.5× local ULN (total bilirubin ≥ 2.5 × local ULN with Gilbert's Syndrome).
Paroxysmal nocturnal hemoglobinuria (PNH) granulocyte clone size determined by flow cytometry ≥ 50%.
Presence of chromosomal aberration (-7/7q- detected by fluorescence in situ hybridization (FISH), or other aberrations detected by G-band staining).
Evidence of a clonal hematologic bone marrow disorder on cytogenetics.
Past medical history of thromboembolism within 6 months or current use of anticoagulants.
Have any concomitant malignancies and must be fully recovered from treatment for any other malignancy and have been disease-free for 5 years.
Patient with clinically significant.
Patient with known hepatocellular disease
Presences of hepatitis B surface antigen (HBsAg), positive hepatitis C antibody test result at screening.
Cardiac disorder (NYHA) functional classification Grade II/III/IV
Past medical history of immediate or delayed hypersensitivity to compounds chemically similar to eltrombopag or their excipients.
Treatment with another investigational product within 30 days.
Prior treatment with eltrombopag, romiplostim, or any other TPO (thrombopoietin) receptor agonist.
Positive result for HIV (Human Immunodeficiency Virus) antibody test.
Pregnant or nursing (lactating) woman.
Woman of child-bearing potential.

Other protocol-defined inclusion/exclusion criteria may apply.