A phase III, randomized, multicenter, subject- and sponsor-blinded, placebo controlled study to compare the efficacy and safety of Anagrelide retard” versus placebo in at risk” subjects with Essential Thrombocythaemia
- Conditions
- male and female at risk subjects with Essential ThrombocythaemiaMedDRA version: 12.1Level: LLTClassification code 10015493Term: Essential thrombocythaemia
- Registration Number
- EUCTR2009-017095-24-SK
- Lead Sponsor
- AOP Orphan Pharmaceuticals AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 240
• 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 < 1.000 G/L
additionally at least ONE of the following criteria has to be fulfilled:
- Subjects aged between 40 and 60 years
or
- Subjects with ET and disease duration > 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 < 4); Hormone replacement therapy; hormonal contraception
Are the trial subjects under 18? no
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) no
F.1.3.1 Number of subjects for this age range
• 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 <30 ml/min)
• Severe liver impairment (ALT or AST >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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 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:<br>- reduction of platelet count <br>- response rates<br>- change to high risk status<br>- cardiovascular safety<br>- Quality of Life;Primary end point(s): Time to the 1st clinically significant ET related event
- Secondary Outcome Measures
Name Time Method