NCT00909363 (WAS) is a Phase 2 clinical trial of Promacta in Wiskott-Aldrich Syndrome, sponsored by Weill Medical College of Cornell University.

Who is sponsoring NCT00909363?

NCT00909363 is sponsored by Weill Medical College of Cornell University.

What drugs are being tested in NCT00909363?

Interventions in NCT00909363: Promacta, blood drawing in patients with WAS, blood drawing in healthy controls.

What condition does NCT00909363 study?

NCT00909363 studies Wiskott-Aldrich Syndrome, Thrombocytopenia, Bleeding.

Is NCT00909363 still recruiting?

NCT00909363 is currently terminated. Last updated 15 March 2019.

Are results published for NCT00909363?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2019-03-15 · How we verify

NCT00909363: WAS

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Effects Of Eltrombopag On Thrombocytopenia, Platelet Function and Bleeding In Patients With Wiskott-Aldrich Syndrome/X-Linked Thrombocytopenia.

Terminated Phase 2 Results posted Last updated 15 March 2019

What this trial tests

Phase 2 trial testing Promacta in Wiskott-Aldrich Syndrome in 24 participants. Terminated before completion.

Timeline

1 June 2009

Primary endpoint
15 May 2017

30 June 2017

Quick facts

Lead sponsor	Weill Medical College of Cornell University
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	24
Start date	1 June 2009
Primary completion	15 May 2017
Estimated completion	30 June 2017
Sites	1 location across United States

Drugs / interventions tested

Promacta (ELTROMBOPAG) — full drug profile →
blood drawing in patients with WAS
blood drawing in healthy controls

Conditions studied

Wiskott-Aldrich Syndrome — all drugs for Wiskott-Aldrich Syndrome →
Thrombocytopenia — all drugs for Thrombocytopenia →
Bleeding — all drugs for Bleeding →

Sponsor

Weill Medical College of Cornell University

Who can join

Adults 3 Months to 80, male only, with Wiskott-Aldrich Syndrome or Thrombocytopenia. Healthy volunteers can join.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

How Many WAS Patients Will Achieve Platelet Counts Above 50,000/ul.
Time frame: 12 weeks
number of WAS patients achieving this increase to \> 50,000/uL without rescue medication in the previous 3 weeks during eltrombopag treatment

Sponsor's own description

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.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia.
Gerrits AJ, Leven EA, Frelinger AL, Brigstocke SL, et al · · 2015 · cited 70× · PMID 26224646 · DOI 10.1182/blood-2014-09-602573
Off-Label Use of Thrombopoietin Receptor Agonists: Case Series and Review of the Literature.
Capecchi M, Serpenti F, Giannotta J, Pettine L, et al · · 2021 · cited 6× · PMID 34650908 · DOI 10.3389/fonc.2021.680411

Verify or expand the search:

Other Weill Medical College of Cornell University trials

Trials by the same sponsor.

NCT07485712 — Center for Research and Education on Aging and Technology Enhancement - CREATE V - Project 2 · NA · not yet recruiting
NCT05811338 — Center for Research and Education on Aging and Technology Enhancement - CREATE V - Project 1 · NA · not yet recruiting
NCT06118983 — Improving TRansitions ANd OutcomeS for Heart FailurE Patients in Home Health CaRe (I-TRANSFER-HF) · NA · recruiting
NCT06818734 — Implementation of a Clinical Decision Support Tool for Postpartum Depression · NA · not yet recruiting
NCT07485725 — Center for Research and Education on Aging and Technology Enhancement - CREATE V - Project 3 · NA · not yet recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT00909363 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Weill Medical College of Cornell University
Last refreshed: 15 March 2019

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00909363.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing