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

NCT00909363 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2019-03-18

Study results available
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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.

Conditions

Interventions

DRUG

Promacta

WAS Patients receiving treatment will start on 1 mg/kg of eltrombopag daily and be seen weekly for 12 weeks. Dose adjustment will be based on the weekly monitoring of the platelet count as utilized in ongoing studies in ITP as well as on liver tests. they will also have diagnostic blood testing prior to initiating treatment

DIAGNOSTIC_TEST

blood drawing in patients with WAS

blood will be drawn for platelet parameters in WAS patients not receiving treatment either because they declined or because they were ineligible

DIAGNOSTIC_TEST

blood drawing in healthy controls

blood will be drawn once in healthy children as controls for platelet parameters

Sponsors & Collaborators

Principal Investigators

  • James B Bussel, MD · Weill Medical College of Cornell University

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Months
Max Age
80 Years
Sex
MALE
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2009-06-30
Primary Completion
2017-05-15
Completion
2017-06-30

Countries

  • United States

Study Locations

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Diseases
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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00909363 on ClinicalTrials.gov