Status:
TERMINATED
Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
Lead Sponsor:
Weill Medical College of Cornell University
Collaborating Sponsors:
Novartis Pharmaceuticals
Conditions:
Wiskott-Aldrich Syndrome
Thrombocytopenia
Eligibility:
MALE
3-80 years
Phase:
PHASE2
Brief Summary
The purpose of this project is to describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in v...
Detailed Description
Wiskott Aldrich Syndrome is an X-linked disease characterized by immunodeficiency, eczema and thrombocytopenia; a milder form of the disease known as X-Linked thrombocytopenia also exists. The thrombo...
Eligibility Criteria
Inclusion
- In order to be eligible for study entry, subjects must comply with the following:
- Males from 3 months old to 80 years old
- Signed written informed consent obtained prior to study entry
- Clinical diagnosis of WAS or XLT
- Platelet levels less than 100 x 109/L
- Adequate renal and hepatic function (creatinine and bilirubin less than or equal to 1.5 x IULN, AST and ALT less than or equal to 2.5 x IULN)
Exclusion
- Any patient is ineligible for study entry if he/she:
- Over the age of 80
- Women (only males are eligible)
- fertile men who are not practicing or who are unwilling to practice birth control while enrolled in the study or until at least 6 months after treatment
- Aspirin, aspirin-containing compounds, salicylates, non-steroidal anti-inflammatory medications (NSAIDS), clopidogrel or ticlopidine, warfarin or other vitamin K antagonists, unfractionated or low molecular heparin within 7 days of first infusion
- Red blood cell transfusion in the past four weeks
- Elevated (\> 1.5 x ULN) prothrombin time (PT) or partial thromboplastin time (PTT)
- New York Heart Classification III or IV heart disease. Other severe cardiovascular or cardiopulmonary disease, including COPD.
- Known HIV infection, hepatitis B or C infection
- Any infection requiring antibiotic treatment within 3 days
- Other concurrent medical or psychiatric conditions that, in the Investigator's opinion, may be likely to confound study interpretation or prevent completion of study procedures and follow-up examinations.
- Prior malignancy with less than a 5-year disease-free interval, excluding nonmelanoma skin cancers and carcinoma in situ of the cervix
Key Trial Info
Start Date :
June 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 30 2017
Estimated Enrollment :
24 Patients enrolled
Trial Details
Trial ID
NCT00909363
Start Date
June 1 2009
End Date
June 30 2017
Last Update
March 18 2019
Active Locations (1)
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1
Weill Cornell Medical College
New York, New York, United States, 10065