CompletedPhase 2

Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia

Brazil, Hong Kong, India54 participantsStarted 2017-05-11

Plain-language summary

The purpose of this study was to evaluate the efficacy and safety of eltrombopag in combination with cyclosporine alone as first-line therapy on overall hematologic response

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Patient had signed the Informed Consent (ICF) prior to any screening procedures
✓. Patient was male/female ≥18 years old at the time of informed consent and able to swallow a tablet.
✓. Patient had SAA characterized by:
✓. Bone marrow cellularity \<30% (excluding lymphocytes) and
✓. At least two of the following (peripheral blood):
✓. Normal ECG defined as the following as determined via the mean of a triplicate ECG

Exclusion criteria

✕. Diagnosis of Fanconi anemia.
✕. Evidence of a clonal hematologic bone marrow disorder on cytogenetics by central review
✕. Prior immunosuppressive therapy with cyclosporine, alemtuzumab, rabbit or horse ATG and thrombopoietin receptor (TPO-R) agonists.
✕. Hypersensitivity to eltrombopag or cyclosporine or their components.
✕. AST or ALT \>3 x ULN.
✕. Serum creatinine, total bilirubin, or alkaline phosphatase \>1.5 x ULN.
✕. Patient with liver cirrhosis.
✕. Patients who were human immune deficiency virus (HIV), hepatitis C virus or hepatitis B surface antigen (HBsAg) positive. HCV-RNA negative patients were allowed to be enrolled.

What they're measuring

Overall Hematologic Response Rate by 6 Months

Timeframe: Up to 6 months

Trial details

NCT IDNCT02998645

SponsorNovartis Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-11-03

Results submitted2023-02-24

View on ClinicalTrials.gov More Severe Aplastic Anemia trials