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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03988608
Recruitment Status : Recruiting
First Posted : June 17, 2019
Last Update Posted : September 28, 2020
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
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 or disease Intervention/treatment Phase
Aplastic Anemia Drug: Eltrombopag 25 mg Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : December 9, 2019
Estimated Primary Completion Date : August 31, 2021
Estimated Study Completion Date : July 24, 2023

Resource links provided by the National Library of Medicine

Drug Information available for: Eltrombopag

Arm Intervention/treatment
Experimental: Eltrombopag
Subjects will start eltrombopag treatment at 25 mg/day since Day 1.
Drug: Eltrombopag 25 mg
film-coated tablets containing 25 mg of eltrombopag free acid in each tablet
Other Name: ETB115




Primary Outcome Measures :
  1. Hematologic response rate [ Time Frame: 6 months (Week 26) ]
    Hematologic response rate is defined as the percentage of all subjects who meet any of the IWG criteria (International Working Group).


Secondary Outcome Measures :
  1. Hematologic response rate [ Time Frame: Week 13 and Week 52 ]
    Hematologic response rate is defined as the percentage of all subjects who meet any of the IWG criteria (International Working Group).

  2. Changes in platelet count [ Time Frame: Baseline to Week 26 or up to 3.5 years ]
    Changes in platelet count in the absence of platelet transfusion.

  3. Changes in hemoglobin count [ Time Frame: Baseline to Week 26 or up to 3.5 years ]
    Changes in hemoglobin in the absence of RBC (Red Blood Cell) transfusion.

  4. Changes in neutrophil count [ Time Frame: Baseline to Week 26 or up to 3.5 years ]
    Changes in neutrophil count in the absence of G-CSF (Granulocyte Colony Stimulating Factor).

  5. Time to hematologic response [ Time Frame: Baseline to Week 26 or up to 3.5 years ]
    Time to hematologic response (any response according to the response criteria for the primary endpoint).

  6. Duration of hematologic response [ Time Frame: Baseline to Week 26 or up to 3.5 years ]
    Duration of hematologic response (any response according to the response criteria for the primary endpoint).

  7. Frequency of transfusion [ Time Frame: Baseline to Week 26 or up to 3.5 years ]
    Frequency of transfusion (platelet and RBC (Red Blood Cell))

  8. Volume of transfusion [ Time Frame: Baseline to Week 26 or up to 3.5 years ]
    Volume of transfusion (platelet and RBC (Red Blood Cell))

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

  10. Rate of clonal evolution [ Time Frame: Baseline to Week 26 or up to 3.5 years ]
    Rate of clonal evolution including clonal evolution to PNH (Paroxysmal Nocturnal Hemoglobinuria), evolution to AML (Acute Myeloid Leukemia) or MDS (Myelodysplastic Syndromes).



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03988608


Contacts
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Contact: Novartis Pharmaceuticals +41613241111 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals

Locations
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China, Jiangsu
Novartis Investigative Site Recruiting
Nanjing, Jiangsu, China
China, Jiangxi
Novartis Investigative Site Recruiting
Nanchang, Jiangxi, China, 330006
China, Jilin
Novartis Investigative Site Recruiting
Chang Chun, Jilin, China, 130021
China, Sichuan
Novartis Investigative Site Recruiting
Chengdu, Sichuan, China, 610041
China, Tianjin
Novartis Investigative Site Recruiting
Tianjin, Tianjin, China, 300020
China
Novartis Investigative Site Recruiting
Beijing, China, 100730
Novartis Investigative Site Recruiting
Tianjin, China, 300052
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03988608    
Other Study ID Numbers: CETB115E2202
First Posted: June 17, 2019    Key Record Dates
Last Update Posted: September 28, 2020
Last Verified: September 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data is currently available according to the process described on www.clinicalstudydatarequest.com

URL: https://www.clinicalstudydatarequest.com

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Severe Aplastic Anemia
Eltrombopag
ETB115
Chinese subject
Chinese refractory or relapsed SAA subjects
refractory severe aplastic anemia
relapsed severe aplastic anemia
Additional relevant MeSH terms:
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Anemia
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases