Brief Title
Efficacy and Safety of Eltrombopag + Tacrolimus in Chinese Refractory or Relapsed Aplastic Anemia Patients
Official Title
Efficacy and Safety of Eltrombopag + Tacrolimus in Chinese Refractory or Relapsed Aplastic Anemia Patients
Brief Summary
This is a randomized, open-label, phase II study to compare the efficacy of eltrombopag
combined with tacrolimus to eltrombopag alone in Chinese subjects with refractory or relapsed
aplastic anemia. The safety would also be evaluated. Patients would be randomized to receive
eltrombopag alone or eltrombopag combined with tacrolimus. 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, or the best response was achieved. Tacrolimus will be given at 1mg bid with
the target trough concentration of 4-10 ng/mL throughout the study. The hematological
response rate and safety will be recorded and compared at 3, 6 months and 1 year after
starting the study treatment (Week 13, 26 and 52).
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
ORR at 6 Months
Secondary Outcome
ORR at 3 Months
Condition
Aplastic Anemia
Intervention
Tacrolimus
Study Arms / Comparison Groups
Eltrombopag
Description: Eltrombopag and the placebo would be applied. 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, or the best response was achieved.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
114
Start Date
July 1, 2020
Completion Date
July 31, 2022
Primary Completion Date
July 30, 2022
Eligibility Criteria
Inclusion Criteria:
1. Patient with a previous diagnosis of aplastic anemia and had no response or relapsed
following at least one treatment course in a period time of ≥ 6 months of
immunosuppression containing CsA or CsA+anti-thymocyte globulin (ATG);
2. Current diagnosis of aplastic anemia by bone marrow biopsy;
3. did not receive HSCT nor were HSCT candidates;
4. Patient has an Eastern Cooperative Oncology Group (ECOG) performance status 0-2;
5. 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.
6. Subjects are able to understand and comply with protocol requirements and instructions
and have signed and dated informed consent.
Exclusion Criteria:
1. Congenital aplastic anemia;
2. Presence of chromosomal aberration;
3. Evidence of a clonal hematologic bone marrow disorder on cytogenetics;
4. Have any concomitant malignancies and must be fully recovered from treatment for any
other malignancy and have been disease-free for 5 years;
5. AST or ALT ≥3 times the upper limit of normal;
6. Serum creatinine, total bilirubin, or alkaline phosphatase >1.5 x ULN;
7. Cardiac disorder (NYHA) functional classification Grade II/III/IV;
8. Past history of thromboembolic event (including anti-phospholipid antibody syndrome)
and current use of anticoagulants;
9. Infection not adequately responding to appropriate therapy;
10. Other known or suspected underlying primary immunodeficiency;
11. Prior treatment with eltrombopag, romiplostim, or any other TPO (thrombopoietin)
receptor agonist;
12. Pregnant or nursing (lactating) woman;
Gender
All
Ages
14 Years - 85 Years
Accepts Healthy Volunteers
No
Contacts
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Location Countries
China
Location Countries
China
Administrative Informations
NCT ID
NCT04403321
Organization ID
ELT-1
Responsible Party
Principal Investigator
Study Sponsor
Peking Union Medical College Hospital
Study Sponsor
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Verification Date
January 2023