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

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
James B. Bussel, Weill Medical College of Cornell University
ClinicalTrials.gov Identifier:
NCT00909363
First received: May 27, 2009
Last updated: January 31, 2017
Last verified: January 2017
  Purpose
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.

Condition Intervention Phase
Wiskott-Aldrich Syndrome
Thrombocytopenia
Bleeding
Drug: Promacta (eltrombopag)
Drug: Eltrombopag/promacta
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
Official Title: Effects Of Eltrombopag On Thrombocytopenia, Platelet Function and Bleeding In Patients With Wiskott-Aldrich Syndrome/X-Linked Thrombocytopenia.

Resource links provided by NLM:


Further study details as provided by Weill Medical College of Cornell University:

Primary Outcome Measures:
  • To increase platelet counts of WAS and XLT patients to above 30,000/ul. [ Time Frame: 12 weeks ]

Secondary Outcome Measures:
  • 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. [ Time Frame: 12 Weeks ]

Enrollment: 19
Study Start Date: June 2009
Estimated Study Completion Date: June 2020
Estimated Primary Completion Date: May 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Promacta
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.
Drug: Promacta (eltrombopag)
Patients 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.
Other Name: eltrombopag
Drug: Eltrombopag/promacta
Patients 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.

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.

  Eligibility

Ages Eligible for Study:   3 Months to 80 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   Yes
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00909363

Locations
United States, New York
Weill Cornell Medical College
New York, New York, United States, 10065
Sponsors and Collaborators
Weill Medical College of Cornell University
Novartis Pharmaceuticals
Investigators
Principal Investigator: James B Bussel, MD Weill Medical College of Cornell University
  More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: James B. Bussel, Professor of Pediatrics, Weill Medical College of Cornell University
ClinicalTrials.gov Identifier: NCT00909363     History of Changes
Other Study ID Numbers: 0801009600
Study First Received: May 27, 2009
Last Updated: January 31, 2017
Individual Participant Data  
Plan to Share IPD: Undecided

Additional relevant MeSH terms:
Wiskott-Aldrich Syndrome
Syndrome
Hemorrhage
Thrombocytopenia
Disease
Pathologic Processes
Blood Platelet Disorders
Hematologic Diseases
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hemorrhagic Disorders
Lymphopenia
Leukopenia
Leukocyte Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Immunologic Deficiency Syndromes
Immune System Diseases

ClinicalTrials.gov processed this record on March 22, 2017