Overview

Efficacy and Safety of Eltrombopag + Tacrolimus/Cyclosporin A in Chinese Refractory or Relapsed Aplastic Anemia Patients

Status:
Not yet recruiting

Trial end date:
2022-12-31

Target enrollment:
0

Participant gender:
All

Summary

This is a randomized, open-label, phase II study to compare the efficacy of eltrombopag combined with tacrolimus//Cyclosporin A 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//Cyclosporin A. 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. Tacrolimus will be given at 1mg bid with the target concentration to be 5-10 ng/ml throughout the study. Cyclosporin A will be give at the target comcentration to be 100-150ng/ml. 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).

Phase:

Phase 2

Accepts Healthy Volunteers?

Details

Lead Sponsor:

Peking Union Medical College Hospital

Treatments:

Cyclosporine
Cyclosporins
Tacrolimus

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;