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An exploratory phase II dose escalation study of eltrombopag in MYH9 related disease - ND

Conditions
MYH9 related disease
MedDRA version: 9.1Level: LLTClassification code 10043551Term: Thrombocytopaenia
Registration Number
EUCTR2008-001903-42-IT
Lead Sponsor
OSPEDALE POLICLINICO S. MATTEO
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
Not specified
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

- 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.

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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