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

• Register: ClinicalTrials.gov
• Last refreshed on: 20 November 2023
• Main ID: NCT03988608
• Date of registration: 13/06/2019
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharmaceuticals
• Public title: Study to Assess the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects.
• Scientific title: 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
• Date of first enrolment: December 9, 2019
• Target sample size: 20
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03988608
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

China

Contacts

• Name: Novartis Pharmaceuticals
• Affiliation: Novartis Pharmaceuticals

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Aplastic Anemia

Intervention(s)

Drug: Eltrombopag 25 mg

Primary Outcome(s)

Hematologic response rate [Time Frame: 6 months (Week 26)]

Secondary Outcome(s)

Duration of hematologic response [Time Frame: Baseline to Week 26 or up to 3.5 years]

Plasma concentration of eltrombopag including the trough concentrations [Time Frame: Baseline to Week 26]

Changes in neutrophil count [Time Frame: Baseline to Week 26 or up to 3.5 years]

Hematologic response rate [Time Frame: Week 13 and Week 52]

Frequency of transfusion [Time Frame: Baseline to Week 26 or up to 3.5 years]

Time to hematologic response [Time Frame: Baseline to Week 26 or up to 3.5 years]

Changes in platelet count [Time Frame: Baseline to Week 26 or up to 3.5 years]

Volume of transfusion [Time Frame: Baseline to Week 26 or up to 3.5 years]

Changes in hemoglobin count [Time Frame: Baseline to Week 26 or up to 3.5 years]

Rate of clonal evolution [Time Frame: Baseline to Week 26 or up to 3.5 years]

Secondary ID(s)

CETB115E2202

Source(s) of Monetary Support

Please refer to primary and secondary sponsors