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

• Register: ClinicalTrials.gov
• Last refreshed on: 30 January 2023
• Main ID: NCT04403321
• Date of registration: 21/05/2020
• Prospective Registration: Yes
• Primary sponsor: Peking Union Medical College Hospital
• Public title: Efficacy and Safety of Eltrombopag + Tacrolimus in Chinese Refractory or Relapsed Aplastic Anemia Patients
• Scientific title: Efficacy and Safety of Eltrombopag + Tacrolimus in Chinese Refractory or Relapsed Aplastic Anemia Patients
• Date of first enrolment: July 1, 2020
• Target sample size: 114
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT04403321
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

China

Key inclusion & exclusion 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;



Age minimum: 14 Years
Age maximum: 85 Years
Gender: All

Health Condition(s) or Problem(s) studied

Drug Effect

Aplastic Anemia

Intervention(s)

Drug: Placebo (for Tacrolimus)

Drug: Tacrolimus

Primary Outcome(s)

ORR at 6 Months [Time Frame: Week 26]

Secondary Outcome(s)

Changes in Haemoglobin in the Absence of Red Blood Cells Transfusion [Time Frame: Week 26]

Changes in Platelet in the Absence of Platelet Transfusion [Time Frame: Week 26]

Duration of hematologic response [Time Frame: by 6 months (all patients), at 24 months (responders only)]

ORR at 3 Months [Time Frame: Week 14]

Percentage of patients with clonal evolution to myelodysplasia, PNH, acute leukemia [Time Frame: 12 months]

Secondary ID(s)

ELT-1

Source(s) of Monetary Support

Please refer to primary and secondary sponsors