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出境医 / 临床实验 / Study to Assess the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects.

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

Study Description
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

Study Design
Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual 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 : July 16, 2021
Estimated Study Completion Date : April 18, 2023
Arms and Interventions
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
Outcome Measures
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).


Eligibility Criteria
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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.

Contacts and Locations

Locations
Layout table for location information
China, Jiangsu
Novartis Investigative Site
Nanjing, Jiangsu, China
China, Jiangxi
Novartis Investigative Site
Nanchang, Jiangxi, China, 330006
China, Sichuan
Novartis Investigative Site
Chengdu, Sichuan, China, 610041
China, Tianjin
Novartis Investigative Site
Tianjin, Tianjin, China, 300020
China
Novartis Investigative Site
Tianjin, China, 300052
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Layout table for investigator information
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Tracking Information
First Submitted Date  ICMJE June 13, 2019
First Posted Date  ICMJE June 17, 2019
Last Update Posted Date April 29, 2021
Actual Study Start Date  ICMJE December 9, 2019
Estimated Primary Completion Date July 16, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 11, 2019) 		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).
Original Primary Outcome Measures  ICMJE
 (submitted: June 13, 2019) 		Hematologic response rate [ Time Frame: 6 months (Week 26) ]
Hematologic response rate is ddefined as the percentage of all subjects who meet any of the IWG criteria (International Working Group).
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 11, 2019)
  • 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).
  • 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.
  • 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.
  • 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).
  • 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).
  • 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).
  • Frequency of transfusion [ Time Frame: Baseline to Week 26 or up to 3.5 years ]
    Frequency of transfusion (platelet and RBC (Red Blood Cell))
  • Volume of transfusion [ Time Frame: Baseline to Week 26 or up to 3.5 years ]
    Volume of transfusion (platelet and RBC (Red Blood Cell))
  • Plasma concentration of eltrombopag including the trough concentrations [ Time Frame: Baseline to Week 26 ]
    Plasma concentration of eltrombopag including the trough.
  • 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).
Original Secondary Outcome Measures  ICMJE
 (submitted: June 13, 2019)
  • Hematologic response rate [ Time Frame: Week 13 and Week 52 ]
    Hematologic response rate is ddefined as the percentage of all subjects who meet any of the IWG criteria (International Working Group).
  • 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.
  • 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.
  • 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).
  • 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).
  • 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).
  • Frequency of transfusion [ Time Frame: Baseline to Week 26 or up to 3.5 years ]
    Frequency of transfusion (platelet and RBC (Red Blood Cell))
  • Volume of transfusion [ Time Frame: Baseline to Week 26 or up to 3.5 years ]
    Volume of transfusion (platelet and RBC (Red Blood Cell))
  • Plasma concentration of eltrombopag including the trough concentrations [ Time Frame: Baseline to Week 26 ]
    Plasma concentration of eltrombopag including the trough.
  • 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).
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Assess the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects.
Official Title  ICMJE 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
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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Aplastic Anemia
Intervention  ICMJE Drug: Eltrombopag 25 mg
film-coated tablets containing 25 mg of eltrombopag free acid in each tablet
Other Name: ETB115
Study Arms  ICMJE Experimental: Eltrombopag
Subjects will start eltrombopag treatment at 25 mg/day since Day 1.
Intervention: Drug: Eltrombopag 25 mg
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: June 13, 2019) 		20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE April 18, 2023
Estimated Primary Completion Date July 16, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03988608
Other Study ID Numbers  ICMJE CETB115E2202
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
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
Responsible Party Novartis ( Novartis Pharmaceuticals )
Study Sponsor  ICMJE Novartis Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
PRS Account Novartis
Verification Date April 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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