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

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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
      hemorrhage.

      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

Interventional


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)

Condition

Wiskott-Aldrich Syndrome

Intervention

Promacta

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.

Publications

* 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

Drug

Estimated Enrollment

24

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
      

Gender

Male

Ages

3 Months - 80 Years

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Contacts

James B Bussel, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00909363

Organization ID

0801009600


Responsible Party

Sponsor

Study Sponsor

Weill Medical College of Cornell University

Collaborators

 Novartis Pharmaceuticals

Study Sponsor

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


Verification Date

March 2019