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

PHASE2TerminatedINTERVENTIONAL
Enrollment

24

Participants

Timeline

Start Date

June 30, 2009

Primary Completion Date

May 15, 2017

Study Completion Date

June 30, 2017

Conditions
Wiskott-Aldrich SyndromeThrombocytopeniaBleeding
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

Trial Locations (1)

10065

Weill Cornell Medical College, New York

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

Novartis Pharmaceuticals

INDUSTRY

lead

Weill Medical College of Cornell University

OTHER