Efficacy of Eltrombopag to Improve Thrombocytopenia of MYH9-related Disease
|ClinicalTrials.gov Identifier: NCT01133860|
Recruitment Status : Completed
First Posted : May 31, 2010
Results First Posted : July 21, 2011
Last Update Posted : July 26, 2011
|Condition or disease||Intervention/treatment||Phase|
|Blood Platelet Disorders||Drug: eltrombopag||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Exploratory Phase II Dose Escalation Study of Eltrombopag in MYH9 Related Disease|
|Study Start Date :||January 2009|
|Actual Primary Completion Date :||June 2010|
|Actual Study Completion Date :||June 2010|
Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 will continue eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 will receive eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 will stop therapy.
- Response to Drug Based on Platelet Count at the End of Therapy [ Time Frame: 21 days and/or 42 days of therapy, 15 and 30 days after the end of therapy ]The primary endpoints were the achievement of a platelet count over 100 x10e9/L or at least 3 times the baseline value (major response), or at least twice the baseline value but less than major response (minor response). The overall response to therapy is reported. Platelet count was measured at the end of therapy (21 or 42 days, see study design) by phase-contrast microscopy.
- Bleeding Tendency Assessed by WHO Bleeding Score [ Time Frame: 21 days and/or 42 days of therapy, 15 and 30 days after the end of therapy ]The percentage of patients with bleeding diathesis (grade 1, i.e. cutaneous bleeding, or grade 2, i.e. mild blood loss, according to WHO bleeding score) was calculated at baseline and at the end of therapy. The results are expressed as the mean change in the percentage of patients with bleeding diathesis (95%CI).
- All Types of Adverse Events [ Time Frame: 21 days and/or 42 days of therapy, 15 and 30 days after the end of therapy ]All type of adverse events were registered.Results indicate the number of participants who experience a side effect of the drug.
- in Vitro Function of Platelets Produced During Therapy in Responding Patients [ Time Frame: 21 days or 42 days of therapy ]in vitro platelet function will be assessed in patients achieving a platelet count of 100 x10e9/L or more at the end of the therapy
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01133860
|Azienda Ospedaliero-Universitaria di Padova, Unità di Medicina Generale e Patologia Speciale|
|Padova, Italy, 35128|
|Fondazione IRCCS Policlinico San Matteo, Unità di Medicina III|
|Pavia, Italy, 27100|
|Policlinico Monteluce, Sezione di Medicina Interna e Cardiovascolare|
|Perugia, Italy, 06122|
|Principal Investigator:||Carlo Balduini, MD||IRCCS Policlinico San Matteo Foundation, Pavia, Italy|