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Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT00909363
Other study ID # 0801009600
Secondary ID
Status Terminated
Phase Phase 2
First received
Last updated
Start date June 2009
Est. completion date June 30, 2017

Study information

Verified date March 2019
Source Weill Medical College of Cornell University
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Terminated
Enrollment 24
Est. completion date June 30, 2017
Est. primary completion date May 15, 2017
Accepts healthy volunteers Accepts Healthy Volunteers
Gender Male
Age group 3 Months to 80 Years
Eligibility 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

Study Design


Intervention

Drug:
Promacta
WAS Patients receiving treatment 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 as well as on liver tests. they will also have diagnostic blood testing prior to initiating treatment
Diagnostic Test:
blood drawing in patients with WAS
blood will be drawn for platelet parameters in WAS patients not receiving treatment either because they declined or because they were ineligible
blood drawing in healthy controls
blood will be drawn once in healthy children as controls for platelet parameters

Locations

Country Name City State
United States Weill Cornell Medical College New York New York

Sponsors (2)

Lead Sponsor Collaborator
Weill Medical College of Cornell University Novartis Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary How Many WAS Patients Will Achieve Platelet Counts Above 50,000/ul. number of WAS patients achieving this increase to > 50,000/uL without rescue medication in the previous 3 weeks during eltrombopag treatment 12 weeks
Secondary Number of Patients With Wiskott-Aldrich Syndrome (WAS) With Grade 3 or Higher Bleeding or SAE (on WHO Scale) number of patients with bleeding SAEs while on treatment and/or number of patients with grade 3 or higher bleeding on WHO (World Health Organization) scale: the scale is from 1 to 5 with 5 = fatality and 1=very little bleeding 12 Weeks
Secondary How Many Patients With WAS Had Abnormal Platelet Function Including Activation in how many patients with WAS were platelets dysfunctional or activated before treatment as measured by flow cytometry to a substantial degree and the same after treatment with eltrombopag 12 weeks
Secondary How Many Patients With WAS Had Substantially Increased Platelet Production After Eltrombopag in how many patients with WAS did eltrombopag increase platelet production as measured by the immature platelet fraction (IPF), a variable derived from the Sysmex auto analyzer, which is considered to be a measure of newly formed platelets ie reticulated platelets 12 weeks
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