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

NCT00909363 | Terminated Phase 2 Results DMC

• 1 other identifier: 0801009600
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Wiskott-Aldrich Syndrome; Thrombocytopenia; Bleeding

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Number of Patients With Wiskott-Aldrich Syndrome (WAS) With Grade 3 or Higher Bleeding or SAE (on WHO Scale)

  12 Weeks

• How Many Patients With WAS Had Abnormal Platelet Function Including Activation

  12 weeks

• How Many Patients With WAS Had Substantially Increased Platelet Production After Eltrombopag

  12 weeks

Study Arms (3)

WAS patients receiving Promacta

EXPERIMENTAL

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

WAS patients for blood drawing only

EXPERIMENTAL

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

Diagnostic Test: blood drawing in patients with WAS

healthy children for blood drawing only

PLACEBO COMPARATOR

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

Diagnostic Test: blood drawing in healthy controls

Interventions

Promacta 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

Also known as: eltrombopag, revolade

WAS patients receiving Promacta

blood drawing in patients with WAS DIAGNOSTIC_TEST

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

WAS patients for blood drawing only

blood drawing in healthy controls DIAGNOSTIC_TEST

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

healthy children for blood drawing only

Eligibility Criteria

• Age: 3 Months - 80 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Weill Medical College of Cornell University: lead
• Novartis Pharmaceuticals: collaborator

Study Sites (1)

Weill Cornell Medical College

New York, New York, 10065, United States

Location

Related Publications (1)

• Gerrits AJ, Leven EA, Frelinger AL 3rd, Brigstocke SL, Berny-Lang MA, Mitchell WB, Revel-Vilk S, Tamary H, Carmichael SL, Barnard MR, Michelson AD, Bussel JB. Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia. Blood. 2015 Sep 10;126(11):1367-78. doi: 10.1182/blood-2014-09-602573. Epub 2015 Jul 29.

  PMID: 26224646 DERIVED

MeSH Terms

Conditions

Wiskott-Aldrich Syndrome; Thrombocytopenia; Hemorrhage

Interventions

eltrombopag; Blood Specimen Collection

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphopenia; Leukopenia; Cytopenia; Hemorrhagic Disorders; Leukocyte Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked; Primary Immunodeficiency Diseases; Immunologic Deficiency Syndromes; Immune System Diseases; Blood Platelet Disorders; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Results Point of Contact

• Title: Dr james B Bussel
• Organization: Weill Cornell Medicine

jbussel@med.cornell.edu

917-291-5091

Study Officials

• James B Bussel, MD

  Weill Medical College of Cornell University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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

Study Record Dates

• First Submitted: May 27, 2009
• First Posted: May 28, 2009
• Study Start: June 1, 2009
• Primary Completion: May 15, 2017
• Study Completion: June 30, 2017
• Last Updated: March 18, 2019
• Results First Posted: March 18, 2019

Record last verified: 2019-03

Locations

US (1)