Open label multicenter study of Eltrombopag for the treatment of Immune ThrombocytoPenia (ITP) secondary to Chronic Lymphoproliferative Disorders (LPDs)

INTERVENTION:

Trade Name: REVOLADE*28CPR RIV 25MG Pharmaceutical Form: Coated tablet INN or Proposed

INN:

ELTROMBOPAG OLAMINE CAS Number: 496775‐62‐3 Concentration unit: mg milligram(s) Concentration type: up to Concentration number: 150‐

CONDITION:

Immune ThrombocytoPenia (ITP) secondary to Chronic Lymphoproliferative Disorders (LPDs) ; MedDRA version: 15.0 Level: PT Classification code 10050245 Term: Autoimmune thrombocytopenia System Organ Class: 10005329 ‐ Blood and lymphatic system disorders Therapeutic area: Diseases [C] ‐ Blood and lymphatic diseases [C15]

PRIMARY OUTCOME:

Main Objective: Proportion of responders to eltrombopag as defined by changes in the platelet count, in platelet transfusion requirements and/or in the bleeding symptoms during the 6 months of treatment. Primary end point(s): Proportion of responders to eltrombopag as defined by changes in the platelet count, in platelet transfusion requirements and/or in the bleeding symptoms during the 6 months of treatment. Secondary Objective: Assessment of the safety profile of eltrombopag in patients with LPD using the CTCAE criteria. Timepoint(s) of evaluation of this end point: during the 6 months of treatment.

SECONDARY OUTCOME:

Secondary end point(s): Assessment of the safety profile of eltrombopag in patients with LPD using the CTCAE criteria. Timepoint(s) of evaluation of this end point: for the duration of the study

INCLUSION CRITERIA:

1) Diagnosis of any of the following B‐cell chronic LPD, as defined by WHO 2008 classification: small lymphocytic lymphoma/chronic lymphocytic leukemia, follicular lymphoma, marginal zone lymphoma, mantle cell lymphoma, lymphoplasmacytic lymphoma, hairy cell leukemia, Hodgkin’s lymphoma. 2) Occurrence of ITP diagnosed on the basis of predefined criteria. 3) Not likely to necessitate any cytotoxic treatment for the following 6 months, according to clinical stage and performance status. 4) Platelet count less than 30,000/µL; patients with platelet count between 30 and 50,000/µL only in case of bleeding signs or symptoms. 5) Age greater than or equal to 18 years. 6) Absence of a personal or family (up to first degree relatives) history of venous or arterial thromboembolism. 7) ECOG performance status =2. 8) Adequate liver and renal function. 9) Absence of active Hepatitis B (HBsAg+ or HBV‐DNA+), Hepatitis C (HCV‐Ab+), or HIV infection. 9) Pr