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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-06-30

Study Completion Date

2017-06-30

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Wiskott-Aldrich Syndrome Thrombocytopenia Bleeding

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

1. group of 11 WAS patients treated with eltrombopag
2. WAS patients who had their blood drawn once but did not receive eltrombopag treatment
3. healthy children as controls for testing only

3\) healthy volunteers who had their blood drawn once

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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WAS patients receiving 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.

Group Type EXPERIMENTAL

Promacta

Intervention Type DRUG

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

WAS patients for blood drawing only

WAS patients not receiving treatment to serve as subjects for platelet parameter studies blood drawing once only

Group Type EXPERIMENTAL

blood drawing in patients with WAS

Intervention Type DIAGNOSTIC_TEST

blood will be drawn for platelet parameters in WAS patients not receiving treatment either because they declined or because they were ineligible

healthy children for blood drawing only

healthy children having blood obtained once as controls for platelet parameters study

Group Type PLACEBO_COMPARATOR

blood drawing in healthy controls

Intervention Type DIAGNOSTIC_TEST

blood will be drawn once in healthy children as controls for platelet parameters

Interventions

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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

Intervention Type DRUG

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

Intervention Type DIAGNOSTIC_TEST

blood drawing in healthy controls

blood will be drawn once in healthy children as controls for platelet parameters

Intervention Type DIAGNOSTIC_TEST

Other Intervention Names

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eltrombopag, revolade

Eligibility 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

Minimum Eligible Age

3 Months

Maximum Eligible Age

80 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

Yes