CompletedPhase 2

Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin/Cyclosporine A in Naive Aplastic Anemia (AA) Subjects

Japan10 participantsStarted 2015-05-12

Plain-language summary

This was an open label, non-randomized, phase II study of eltrombopag in combination with rabbit ATG/CsA in subjects with moderate or more severe AA who did not received prior ATG/ALG-based immunosuppressive therapy. The objective was to assess additive effects of eltorombopag on overall response rate (ORR) at 6 months (Week 26) of treatment with ATG/CsA. Subjects were assessed at least weekly for safety during the period from the start of ATG/CsA to 4 weeks after the start of administration of eltrombopag. After that, subjects had visits every 2 weeks until Week 26. Subjects in whom the treatment was assessed as effective at Week 26 could continued treatment with eltrombopag after 6 months when clinically indicated at the discretion of the investigator. There were five follow-up visits: at discontinuation of the treatment of eltrombopag, and Weeks 1, 2, 3, 4 and 26 after treatment discontinuation. As this study was the first Japanese phase II study in which this product was administered in combination with ATG/CsA to subjects with naive moderate or more severe AA, the subject number of this study was determined to be 10 based on the feasibility survey.

Who can participate

Age range18 Years – 75 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: * Japanese subjects aged \>=18 and \<71 years at the time of informed consent. Note: subjects aged \>=71 and \<75 may be eligible when clinically indicated at the discretion of the investigator by mutual agreement with Novartis medical advisor. * Diagnosed with moderate or more severe AA according to the diagnostic criteria of AA. The severity classification is: Stage I - Mild - Other than the stages below; Stage II - Moderate - At least two of the following conditions are met: Reticulocyte \<60,000/microliter, Neutrophil \<1,000/microliter, Platelet \<50,000/microliter; Stage III - Moderately severe - At least two of the following conditions are met and regular red blood cell transfusion (a need for transfusion of \>=2 units per month) is required: Reticulocyte \<60,000/microliter, Neutrophil \<1,000/microliter, Platelet \<50,000/microliter; Stage IV - Severe - At least two of the following conditions are met: Reticulocyte \<20,000/microliter, Neutrophil \<500/microliter, Platelet \<20,000/microliter; Stage V - Very severe - At least one of the following conditions is met in addition to neutrophil \<200/microliter: Reticulocyte \<20,000/microliter, Platelet \<20,000/microliter. * Subjects who are considered an indication for the treatment with rabbit ATG and CsA. * Adequate baseline organ function defined by the following criteria: Alanine aminotransferase (ALT), aspartate aminotransferase (AST)\<=3 × local upper limit of normal (ULN) Creatinine, total b…

What they're measuring

ORR at 6 Months: Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR) at Week 26

Timeframe: Week 26

Trial details

NCT IDNCT02404025

SponsorNovartis Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2016-07-05

Results submitted2018-08-21