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Trial record 28 of 456 for:    Inherited Bleeding Disorder

Efficacy of Eltrombopag to Improve Thrombocytopenia of MYH9-related Disease

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ClinicalTrials.gov Identifier: NCT01133860
Recruitment Status : Completed
First Posted : May 31, 2010
Results First Posted : July 21, 2011
Last Update Posted : July 26, 2011
Sponsor:
Collaborators:
University of Pavia
GlaxoSmithKline
Azienda Ospedaliera di Padova
Azienda Ospedaliera di Perugia
Fondazione Telethon
Information provided by:
IRCCS Policlinico S. Matteo

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Blood Platelet Disorders
Intervention Drug: eltrombopag
Enrollment 12
Recruitment Details Patients enrolled between January 2009 and January 2010 as outpatients in medical clinic
Pre-assignment Details  
Arm/Group Title Eltrombopag
Hide Arm/Group Description Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 continued eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 received eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 stopped therapy.
Period Title: Overall Study
Started 12
Completed 12
Not Completed 0
Arm/Group Title Eltrombopag
Hide Arm/Group Description Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 continued eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 received eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 stopped therapy.
Overall Number of Baseline Participants 12
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
<=18 years
1
   8.3%
Between 18 and 65 years
11
  91.7%
>=65 years
0
   0.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 12 participants
35.0  (14.3)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
Female
7
  58.3%
Male
5
  41.7%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Italy Number Analyzed 12 participants
12
1.Primary Outcome
Title Response to Drug Based on Platelet Count at the End of Therapy
Hide Description The primary endpoints were the achievement of a platelet count over 100 x10e9/L or at least 3 times the baseline value (major response), or at least twice the baseline value but less than major response (minor response). The overall response to therapy is reported. Platelet count was measured at the end of therapy (21 or 42 days, see study design) by phase-contrast microscopy.
Time Frame 21 days and/or 42 days of therapy, 15 and 30 days after the end of therapy
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eltrombopag
Hide Arm/Group Description:
Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 continued eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 received eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 stopped therapy.
Overall Number of Participants Analyzed 12
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
91.6
(61.5 to 99.7)
2.Secondary Outcome
Title Bleeding Tendency Assessed by WHO Bleeding Score
Hide Description The percentage of patients with bleeding diathesis (grade 1, i.e. cutaneous bleeding, or grade 2, i.e. mild blood loss, according to WHO bleeding score) was calculated at baseline and at the end of therapy. The results are expressed as the mean change in the percentage of patients with bleeding diathesis (95%CI).
Time Frame 21 days and/or 42 days of therapy, 15 and 30 days after the end of therapy
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eltrombopag
Hide Arm/Group Description:
Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 continued eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 received eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 stopped therapy.
Overall Number of Participants Analyzed 12
Mean (95% Confidence Interval)
Unit of Measure: participants
66.7
(31.7 to 100)
3.Secondary Outcome
Title All Types of Adverse Events
Hide Description All type of adverse events were registered.Results indicate the number of participants who experience a side effect of the drug.
Time Frame 21 days and/or 42 days of therapy, 15 and 30 days after the end of therapy
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eltrombopag
Hide Arm/Group Description:
Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 continued eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 received eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 stopped therapy.
Overall Number of Participants Analyzed 12
Measure Type: Number
Unit of Measure: number of participants
2
4.Secondary Outcome
Title in Vitro Function of Platelets Produced During Therapy in Responding Patients
Hide Description in vitro platelet function will be assessed in patients achieving a platelet count of 100 x10e9/L or more at the end of the therapy
Time Frame 21 days or 42 days of therapy
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Eltrombopag
Hide Arm/Group Description:
In patients with more than 100 x10e9 platelets/L at the end of therapy, we evaluated also the in vitro platelet aggregation after stimulation with adenosine diphosphate (5 and 20 mcM), collagen (5 and 20 mg/mL) and ristocetin (3 mg/mL) by the densitometric method of Born in native platelet rich plasma. The extent of platelet aggregation was measured 5 minutes after the addition of stimulating agents and results obtained in patients were compared with the normal ranges in the laboratories where the assay was performed. Results are reported as the number of patients with normal platelet aggregation.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: participants
5
Time Frame 51 or 72 days, i.e. during the administration of Eltrombopag (21 or 42 days, see study design) and until 30 days after the end of treatment (see study design).
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Eltrombopag
Hide Arm/Group Description Eltrombopag, administered orally, 50 mg/daily for 21 days. Patients with platelet counts between 100 and 150x10e9/L at day 21 continued eltrombopag 50 mg/daily for 21 additional days. Patients with platelet count lower than 100x10e9/L at day 21 received eltrombopag 75 mg/daily for additional 21 days. Patients with more than 150x10e9 platelets/L at day 21 stopped therapy.
All-Cause Mortality
Eltrombopag
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Eltrombopag
Affected / at Risk (%) # Events
Total   0/12 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Eltrombopag
Affected / at Risk (%) # Events
Total   2/12 (16.67%)    
General disorders   
headache [1]  2/12 (16.67%)  2
dry mouth [2]  1/12 (8.33%)  1
[1]
mild and transient headache at the beginning of the treatment, duration 3-4 days, spontaneous remission
[2]
transient sensation o dry mouth at the beginning of the treatment, duration 3-4 days, spontaneous remission
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Prof. Carlo Balduini
Organization: IRCCS Policlinico San Matteo Foundation
Phone: 0039.0382.502580
EMail: c.balduini@smatteo.pv.it
Publications:
Layout table for additonal information
Responsible Party: Prof. Carlo Balduini, Head of the Unit of Internal Medicine III, IRCCS Policlinico San Matteo Foundation, Pavia, Italy, IRCCS Policlinico San Matteo Foundation, Pavia, Italy
ClinicalTrials.gov Identifier: NCT01133860     History of Changes
Other Study ID Numbers: Eltrombopag-MYH9-2008
First Submitted: May 28, 2010
First Posted: May 31, 2010
Results First Submitted: June 22, 2011
Results First Posted: July 21, 2011
Last Update Posted: July 26, 2011