male and female "at risk" subjects with Essential Thrombocythaemia MedDRA version: 17.1 Level: PT Classification code 10015493 Term: Essential thrombocythaemia System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Conditions
Interventions
Product Name: Anagrelide retard 2mg tablets
Product Code: not applicable
Pharmaceutical Form: Modified-release tablet
INN or Proposed INN: Anagrelide hydrochloride monohydrate
CAS Number: 58579-51-4
C
Sponsors
AOP Orphan Pharmaceuticals AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Willing and able to give written informed consent prior to any study specific procedures and able to comply with this protocol • Male or female subjects aged between 18 and 60 years, • Confirmed diagnosis of ET according to WHO-criteria (2008) including assessment of JAK-2 status. • Presence of predisposing risk factors for ET related events confirmed by clinical or laboratory results: - Platelet count 3 years (Diagnosis of ET has to be at least 3 years ago and confirmed at time of screening) or - Subjects with ONE of the following risk factors for thrombotic complications: a) JAK- 2 positivity, b) Protein C and/or Protein S deficiency, c) Antithrombin III deficiency, d) Factor V Leiden or Prothrombin mutation, e) Cardiovascular risk factors: Essential hypertension; Smoking (>5 cigarettes/d), Obesity (BMI>30), Cholesterol (HDL/LDL ratio =65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: • Diagnosis of any other myeloproliferative disorder • High-risk status (age > 60 years, platelet count = 1.000 G/L, increase of platelet count > 300 G/L within 3 month, history of thrombotic/haemorhagic or ischemic complications). • Any known cause for a secondary thrombocytosis • Previous or current treatment of ET with cytoreductive therapy • Diagnosis of any malignancy, apart from ET, within the last 3 years • Known or suspected intolerance to the investigational products • Known or suspected congestive heart failure • WBC = 15 G/L • Severe renal impairment (creatinine clearance 5 times normal) • Clinically significant abnormal laboratory values (excluding markers of essential thrombocythaemia) • Poorly controlled diabetes mellitus • Infection with hepatitis B, hepatitis C or HIV • Subjects with a history of drug/alcohol abuse (within the previous 2 years) • Participation in another investigational study within 6 months prior to enrolment or for a longer duration if specified in local regulations • Women of childbearing potential with inadequate contraception • Pregnant or lactating women (pregnancy test to be assessed within 7 days prior to study treatment start) • Any significant psychiatric disorder that, in the opinion of the investigator, might prohibit the understanding and giving of informed consent or that might prevent the subject from completing the trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine whether "Anagrelide retard" compared to placebo will reduce the rate of ET-related complications in subjects with potential risk for ET-related Events;Secondary Objective: to assess the effect of "Anagrelide retard" compared to placebo on the following: - reduction of platelet count - response rates - change to "high risk status" - cardiovascular safety - Quality of Life;Primary end point(s): Time to the 1st clinically significant ET related event;Timepoint(s) of evaluation of this end point: Assessed at each visit during 12 months of primary trial and for up to 3 years in extensions study. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Efficacy - reduction of platelet counts - occurrence of change to “high risk” status (i.e. platelets > 1.000 G/L or occurrence of an ET related event) - number of subjects achieving a complete response Safety - Adverse Events - cardiovascular safety (assessed by ECG, ECHO, NT-proBNP or BNP) Quality of Life: SF-36;Timepoint(s) of evaluation of this end point: All Secondary endpoints: Assessed at each visit during 12 months of primary trial and for up to 3 years in extensions study. except cardiovascular safety (assessed by ECG, ECHO, NT-proBNP or BNP): Assessed at screening, month 6 and month 12. ECH & ECG are also assessed every three months for a maximum of 3 years extension study Quality of Life: SF-36 assessed at baseline, month 6 and month 12 | — |
Countries
Austria, Bulgaria, Croatia, Germany, Hungary, Israel, Lithuania, Poland, Romania, Russian Federation, Serbia, Slovakia, Slovenia, Turkey, Ukraine
Contacts
Public ContactMag. Petra Merz
AOP Orphan Pharmaceuticals AG
Outcome results
None listed