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

NCT03988608 | Completed Phase 2 Results

• 1 other identifier: CETB115E2202
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 5

Brief Summary

This was 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 was 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 was assessed at 3, 6 months and 1 year after starting the study treatment (Week 13, 26 and 52).

Conditions

Aplastic Anemia

Keywords

Severe Aplastic Anemia; Eltrombopag; ETB115; Chinese subject; Chinese refractory or relapsed SAA subjects; refractory severe aplastic anemia; relapsed severe aplastic anemia

Outcome Measures

Primary Outcomes (1)

• Hematologic Response Rate at 6 Months (Week 26) by Investigator

  Hematologic response rate: percentage of subjects who met any of the International Working Group criteria: Platelet count: If platelet transfusion independent at baseline (BL): Transfusion independent and increase from BL by 20×10\^9/L or more; If platelet transfusion dependent at BL: No platelet transfusion requirement for 8 weeks; Hemoglobin: If red blood cells (RBC) transfusion independent at BL: transfusion independent and increase from BL by 15 g/L or more; If RBC transfusion dependent at BL: A decrease of at least 4 units in RBC transfusions in the post-treatment 8-week period (1 unit = RBC derived from 200 mL blood) or no RBC transfusion requirement for 8 weeks; Neutrophil count: In the absence of granulocyte colony stimulating factor (G-CSF) taken within 21 days preceding the blood sample collection: Increase from BL by 0.5×10\^9/L or more, or (if \< 0.5×10\^9/L at BL) increase by 100% or more. Patients who discontinued from the trial before week 26 were treated as non-responders.

  6 months (Week 26)

Secondary Outcomes (16)

• Hematologic Response Rate by Investigator

  Week (Wk) 13, Week 52

• Change From Baseline in Platelet Count

  Baseline, Week 13, Week 26, Week 52

• Change From Baseline in Hemoglobin Levels

  Baseline, Week 13, Week 26 and Week 52

• Change From Baseline in Neutrophil Count

  Baseline, Week 13, Week 26 and Week 52

• Time to Hematologic Response by Investigator

  Baseline to Week 26

Study Arms (1)

Eltrombopag

EXPERIMENTAL

Subjects started eltrombopag treatment at 25 mg/day since Day 1.

Drug: Eltrombopag

Interventions

Eltrombopag DRUG

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

Also known as: ETB115

Eltrombopag

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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 (of such severity that it would preclude the patient's ability to consent, be compliant with study procedures, tolerate protocol therapy) bacterial, fungal, mycobacterial, parasitic or viral infection (Patient with acute bacterial infections requiring antibiotic use should delay Screening/enrollment until the course of antibiotic therapy has been completed).
• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (5)

Novartis Investigative Site

Nanjing, Jiangsu, 210000, China

Location

Novartis Investigative Site

Nanchang, Jiangxi, 330006, China

Location

Novartis Investigative Site

Chengdu, Sichuan, 610041, China

Location

Novartis Investigative Site

Tianjin, 300020, China

Location

Novartis Investigative Site

Tianjin, 300052, China

Location

Related Publications (1)

• Chang H, He G, Fu R, Li F, Han B, Li T, Liu L, Mittal H, Jin H, Zhang F. Efficacy and safety of eltrombopag in Chinese patients with refractory or relapsed severe aplastic anemia. Sci Rep. 2023 Nov 2;13(1):18955. doi: 10.1038/s41598-023-45607-0.

  PMID: 37919313 DERIVED

Related Links

• A Plain Language Trial Summary is available on www.novctrd.com

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

eltrombopag

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Results Point of Contact

• Title: Clinical Disclosure Office
• Organization: Novartis Pharmaceuticals

novartis.email@novartis.com

862-778-3000

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 13, 2019
• First Posted: June 17, 2019
• Study Start: December 9, 2019
• Primary Completion: July 16, 2021
• Study Completion: May 17, 2023
• Last Updated: December 30, 2025
• Results First Posted: February 3, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share

Locations

CN (5)