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

Learn more about:
Related Clinical Trial
Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome. Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome Bone Marrow Transplant With Abatacept for Non-Malignant Diseases Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies Alpha/Beta T and CD19+ Depleted Peripheral Stem Cells for Patients With Primary Immunodeficiencies Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP) Participation in a Research Registry for Immune Disorders Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome Gene Therapy for Wiskott-Aldrich Syndrome (WAS) Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS) Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990 A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome Interleukin-2 Treatment for Wiskott-Aldrich Syndrome Gene Therapy for WAS Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS) Molecular and Clinical Studies of Primary Immunodeficiency Diseases A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients

Brief Title

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

Official Title

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

Brief 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.

Detailed Description

      Wiskott Aldrich Syndrome is an X-linked disease characterized by immunodeficiency, eczema and
      thrombocytopenia; a milder form of the disease known as X-Linked thrombocytopenia also
      exists. The thrombocytopenia in both WAS and XLT is characterized by: severe thrombocytopenia
      with platelet counts frequently less than 10-30,000/ul; small platelets which may be
      dysfunctional; and, as a result, a high rate of serious bleeding including intracranial

      Because eltrombopag has been shown to be remarkably efficacious in substantially increasing
      platelet counts in a high percentage of ITP patients, this study seeks to effectively treat
      patients who exhibit similar pathologies, as well as evaluate the state of platelets in
      patients with WAS and relate it to clinical bleeding. It also aims to demonstrate whether
      eltrombopag administered daily will enhance stem cell function, increase platelet production
      and platelet count, and reduce bleeding in patients with WAS.

Study Phase

Phase 2

Study Type


Primary Outcome

How Many WAS Patients Will Achieve Platelet Counts Above 50,000/ul.

Secondary Outcome

 Number of Patients With Wiskott-Aldrich Syndrome (WAS) With Grade 3 or Higher Bleeding or SAE (on WHO Scale)


Wiskott-Aldrich Syndrome



Study Arms / Comparison Groups

 WAS patients receiving Promacta
Description:  Promacta® is commercially available in 12.5 mg, 25 mg, 50 mg, and 75 mg tablets. For this study, for young children unable to swallow a tablet, eltrombopag powder for oral suspension (Eltrombopag PfOS) will be used. PfOS is only available for investigational use at 20mg. Each sachet contains eltrombopag equivalent to 20mg per gm of powder and is reconstituted to a total of 10 ml so that the concentration is 2 mg/ml.


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

June 2009

Completion Date

June 30, 2017

Primary Completion Date

May 15, 2017

Eligibility 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




3 Months - 80 Years

Accepts Healthy Volunteers

Accepts Healthy Volunteers


James B Bussel, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Responsible Party


Study Sponsor

Weill Medical College of Cornell University


 Novartis Pharmaceuticals

Study Sponsor

James B Bussel, MD, Principal Investigator, Weill Medical College of Cornell University

Verification Date

March 2019