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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT00909363 |
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Date of registration:
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27/05/2009 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
WAS |
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Scientific title:
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Effects Of Eltrombopag On Thrombocytopenia, Platelet Function and Bleeding In Patients With Wiskott-Aldrich Syndrome/X-Linked Thrombocytopenia. |
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Date of first enrolment:
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June 2009 |
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Target sample size:
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24 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT00909363 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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James B Bussel, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Weill Medical College of Cornell University |
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Key inclusion & exclusion criteria
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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
Age minimum:
3 Months
Age maximum:
80 Years
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Bleeding
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Thrombocytopenia
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Wiskott-Aldrich Syndrome
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Intervention(s)
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Diagnostic Test: blood drawing in patients with WAS
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Diagnostic Test: blood drawing in healthy controls
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Drug: Promacta
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Primary Outcome(s)
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How Many WAS Patients Will Achieve Platelet Counts Above 50,000/ul.
[Time Frame: 12 weeks]
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Secondary Outcome(s)
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How Many Patients With WAS Had Abnormal Platelet Function Including Activation
[Time Frame: 12 weeks]
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Number of Patients With Wiskott-Aldrich Syndrome (WAS) With Grade 3 or Higher Bleeding or SAE (on WHO Scale)
[Time Frame: 12 Weeks]
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How Many Patients With WAS Had Substantially Increased Platelet Production After Eltrombopag
[Time Frame: 12 weeks]
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Secondary ID(s)
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0801009600
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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