MYH9 related disease MedDRA version: 9.1 Level: LLT Classification code 10043551 Term: Thrombocytopaenia
Conditions
Interventions
Product Name: Eltrombopag olamine
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: SB-497115-GR
CAS Number: 496775-62-3
Current Sponsor code: SB-497115-GR
Concentration unit: mg milligram(
Sponsors
OSPEDALE POLICLINICO S. MATTEO
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Age ≥ 16 years - Confirmed diagnosis of MYH9RD - Average platelet count for the previous year less than 50x10 exp9/L - Written informed consent Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Diseases known to involve the risk of thromboembolic events (e.g. atrial fibrillation) - History of thrombosis within 1 year - Use of drugs that affect platelet function (including but not limited to, aspirin, clopidogrel or NSAIDS) or anti-coagulants - Females who are pregnant or nursing (a negative pregnancy test in required before enrollment of fertile women) - Formal refusal of any recommendation of a safe contraception - Alcohol or drug addiction - Altered renal function as defined by creatinine ≥ 20 mg/L - Any other disease or condition that by the advise of the responsible physician would make the treatment dangerous for the patient or would make the patient ineligible for the study, including physical, psychiatric, social and behavioral problems. HCV positivity and liver diseases will not be considered an exclusion criterion since a phase II study showed that eltrombopag was effective and safe in this patient population.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: to test the hypothesis that eltrombopag, at the dose of 50 or 75 mg/daily for up to 6 weeks, increases platelet count in MYH9RD patients with less than 50x10 exp9 platelets/L.;Secondary Objective: a) to test the hypothesis that eltrombopag reduces bleeding tendency, measured with a bleeding scale; b) to evaluate in vitro function of platelets produced during eltrombopag therapy in patients responding to this drug; c) to evaluate safety and tolerability of eltrombopag in patients with MYH9RD;Primary end point(s): platelet count increase | — |
Countries
Italy
Outcome results
None listed