Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients

Stopped early · Phase 2

Conditions studied: Wiskott-Aldrich Syndrome, Thrombocytopenia, Bleeding

In brief

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 vitro and in vivo.

Key facts

Study ID

NCT00909363

Run by

Weill Medical College of Cornell University

People needed

24

Starts

2009-06-01

Expected to finish

2017-06-30

Last updated by the study team

2019-03-18

Who can join

Age: 0 and older, up to 80. Sex: male. Healthy volunteers: accepted.

You may qualify if…

• 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)

You may not qualify if…

• 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

Where it is running

• Weill Cornell Medical College — New York, New York, United States

Full record on ClinicalTrials.gov

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