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

• Status: Terminated
• Trial end date: 2017-06-30
• Target enrollment: 0
• Participant gender: Male

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 vitro and in vivo.

• Phase: Phase 2
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Weill Medical College of Cornell University
• Collaborator: Novartis Pharmaceuticals

Criteria

Inclusion Criteria:

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 Criteria:

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