Wiskott-Aldrich Syndrome | Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
Wiskott-Aldrich Syndrome research study
What is the primary objective of this study?
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.
Who is eligible to participate?
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
Which medical condition, disease, disorder, syndrome, illness, or injury is researched?
Interventions can include giving participants drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available or noninvasive approaches such as surveys, education, and interviews.
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.
Drug:Eltrombopag/promactaPatients 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.
Research studies and clinical trials typically have two or more research arms. An arm is a group of people who receive the same treatment in the study.
PromactaPromacta® 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.
Active, not recruiting
Start Date: June 2009
Completed Date: June 2020
Phase: Phase 2
Primary Outcome: To increase platelet counts of WAS and XLT patients to above 30,000/ul.
Secondary Outcome: 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.
Study sponsors, principal investigator, and references
Principal Investigator: James B Bussel, MD
Lead Sponsor: Weill Medical College of Cornell University
Collaborator: Novartis Pharmaceuticals