Inherited thrombocytopenias MedDRA version: 20.0 Level: HLT Classification code 10043555 Term: Thrombocytopenias System Organ Class: 100000004851
Conditions
Interventions
Trade Name: Revolade
Product Name: Eltrombopag
Pharmaceutical Form: Film-coated tablet
Sponsors
CHU Toulouse
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Related to patients: Symptomatic inherited thrombocytopenia (IT) with family history and/or molecular identification with an average platelet count in the previous year below the safety level required for the procedure. Written informed consent of the patient or relatives. Related to the procedure: scheduled (=4 weeks) invasive procedure with anticipated risk and limited possibility of mechanical control of bleeding, which would have systematically required prophylactic and therapeutic PC transfusions, according to current international recommendations (standard care). Are the trial subjects under 18? yes Number of subjects for this age range: 25 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 25 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 25
Exclusion criteria
Exclusion criteria: • age 75 yrs • personal history of arterial or venous thromboembolic events • association with another acquired or constitutional hemorrhagic diathesis • chronic hepatitis, cirrhosis, with moderate to severe liver failure (Child-Pugh score =5) • previous or concurrent myeloid malignancy, including myelodysplastic syndrome • alanine aminotransferase (ALT) or bilirubin levels 2 times the upper limit of normal • severe altered renal function (creatinin clearance <30 ml/min) • pregnancy (negative test required before inclusion in fertile women) or lactating women • refusal of safe contraception • ocular lenses opacity • hypersensitivity to eltrombopag or one of excipients • previous participation to the present study • current treatment with antiplatelet drugs, anticoagulants or direct acting antiviral agents approved for treatment of chronic hepatitis C infection • psychiatric, social or behavioral condition judged to be non-compatible with the respect of the protocol, including good observance of treatment and compliance to follow-up. • adult protected by the law.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to estimate the response to eltrombopag based on platelet count increase and lack of requirement for PC transfusion for performing invasive acts at mild or high bleeding risk;Secondary Objective: The secondary objective is to document in this particular setting the safety profile of eltrombopag, the kinetics of platelet recovery and the predictive value Inherited thrombocytopenia (IT) type, serum thrombopoietin (TPO) level and baseline platelet count on the response to eltrombopag.;Primary end point(s): Full response (“success”) is defined as an increase in platelet count above the safety level and a procedure performed without PC transfusion,;Timepoint(s) of evaluation of this end point: 4 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Adverse effects recorded during treatment and the 4-week follow-up after discontinuation, including excessive bleeding or any complication related to bleeding or thrombotic events. Serial platelet counts and change in platelet size ;Timepoint(s) of evaluation of this end point: 1/week during 8 weeks | — |
Countries
France
Contacts
Public ContactCLINICAL RESEARCH ASSISTANT
CHU Toulouse
Outcome results
None listed