Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia

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Brief Title

Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia

Official Title

Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia

Brief Summary

      The investigators hypothesis is that eltrombopag given to patients with moderate to very
      severe aplastic anemia will result in an increase in platelet counts. The investigators
      hypothesize that in patients with moderate to very severe aplastic anemia, treatment with
      eltrombopag will lead to fewer platelet transfusions, red blood cell transfusions, and fewer
      bleeding events. The investigators hypothesize that in patients with moderate to very severe
      aplastic anemia, eltrombopag will have an acceptable toxicity rate <3%, at doses that result
      in increased platelet counts. Finally the investigators hypothesize that plasma eltrombopag
      levels in peripheral blood will correlate with improved platelet counts.
    


Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Proportion of Participants With Platelet Response

Secondary Outcome

 Platelet Count Twice Baseline.

Condition

Severe Aplastic Anemia

Intervention

Eltrombopag

Study Arms / Comparison Groups

 Eltrombopag
Description:  Single arm study. Dose Escalation.

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

13

Start Date

November 2012

Completion Date

June 2016

Primary Completion Date

June 2016

Eligibility Criteria

        Inclusion Criteria:

          -  Able to provide written informed consent and any other authorizations required by
             local law (e.g., Protected Health Information [PHI])

          -  Have severe or very severe aplastic anemia, or moderate aplastic anemia with platelet
             counts that have dropped below 20,000/μl

          -  Have moderate, severe, or very severe aplastic anemia with moderate bleeding during or
             after a surgical procedure, (including bone marrow biopsy, lumbar puncture,
             thoracentesis, paracentesis, port placement, dermal biopsy) or minimal mucocutaneous
             bleeding otherwise noted

          -  Subjects with current or previous exposure to approved medications for the treatment
             of aplastic anemia will not be excluded; these include but may not be limited to,
             anti-thymocyte globulin (ATG), cyclosporine, corticosteroids, and G-CSF.

        Exclusion Criteria:

          -  Have diagnosis of Fanconi anemia

          -  Have infection not adequately responding to appropriate therapy

          -  Have Paroxysmal Nocturnal Hemoglobinuria (PNH) clone size in neutrophils of greater
             than or equal to 50%

          -  Have known HIV positivity

          -  Have creatinine and/or blood urea nitrogen (BUN) ≥2 times the upper limit of normal

          -  Have serum bilirubin ≥ 1.5 times the upper limit of normal, or ≥4.0 times the upper
             limit of normal if the patient has been treated with ATG within three weeks of
             screening.

          -  Have AST and/or ALT ≥ 3 times the upper limit of normal

          -  Have hypersensitivity to eltrombopag or its components

          -  Have chemotherapy given less than or equal to 14 days prior to initiating the study
             medication. This does not include immunosuppressive agents and growth factor as
             described above

          -  Are female and are nursing or pregnant or are unwilling to take oral contraceptives or
             refrain from pregnancy if of childbearing potential

          -  Are unable to understand the investigational nature of the study or give informed
             consent

          -  Have a history of arterial or venous thrombosis within the last 1 year (excluding
             those due to indwelling lines)

          -  Have an ECOG performance status of 3 or greater

          -  Have had treatment with Campath within 6 months of entry into the study

          -  Have pre-existing cardiovascular disease (congestive heart failure with New York Heart
             Association [NYHA] grade III/IV), arrhythmia known to increase the risk of
             thromboembolic events (e.g. atrial fibrillation), unstable angina, or QTc > 450 msec
             (QTc 480 msec for subjects with bundle branch block), or myocardial infarction within
             the preceding 6 months) at study entry

          -  Have had other TPO-R agonists medication in the previous 4 weeks.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

George M Rodgers, M.D., , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01703169

Organization ID

HCI54443

Secondary IDs

ELT115895

Responsible Party

Sponsor

Study Sponsor

University of Utah

Collaborators

 Novartis

Study Sponsor

George M Rodgers, M.D., Principal Investigator, University of Utah


Verification Date

September 2017