Status:
COMPLETED
Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia Major
Lead Sponsor:
University of Washington
Collaborating Sponsors:
National Heart, Lung, and Blood Institute (NHLBI)
George Papanicolaou Hospital
Conditions:
Beta-Thalassemia
Eligibility:
All Genders
18-50 years
Phase:
NA
Brief Summary
Thalassemia is considered the most common genetic disorder worldwide, occurring with high frequency in Mediterranean areas, the Middle East, Southeast Asia, and the Pacific Islands. Currently, the onl...
Detailed Description
The purpose of this study is to optimize blood stem cell mobilization in adults with beta thalassemia major. We seek a method of mobilization that will be safe, with minimum side effects, and that wil...
Eligibility Criteria
Inclusion
- Βeta- thalassemia major
- Age \>18\<50
- Karnofsky performance status ³80%
- Splenectomized patients or patients with spleen volume \<800cm3 (only for the non splenectomized patients who will receive Mozobil + G-CSF)
- Compliant with regular transfusions and regular chelation
- Liver iron by MRI \<280μmol/gr or ³1.7msec by T2\*MRI
- Heart iron by MRI \>2.8 (SI/SD) or ³9msec by T2\*MRI
- Hepatitis B or C virus load negative by PCR (polymerase chain reaction)
- Left ventricular ejection fraction (LVEF) \>45% by echocardiogram
- Adequate respiratory function with DLCO \>50%
- Negative pregnancy test, if female
- Ability to give informed consent and willingness to meet all the expected requirements of the protocol for the duration of the study
Exclusion
- History of thrombosis or known thrombophilia
- Symptomatic viral, bacterial or fungal infection within 6 weeks prior eligibility evaluation
- Pregnancy or lactation
- HIV positivity
- History of malignancy, other than local skin cancer
- Other systematic disease non thalassemia-associated
- Splenectomized patients with platelet count \>900,000 (only for the splenectomized patients who will receive low dose G-CSF+ Mozobil)
- Additional risk factors for thrombosis, including Factor V Leiden; antiphospholipid antibodies and less than 50% of the lowest normal value for the following procoagulants: antithrombin 3, protein C, or protein S.
Key Trial Info
Start Date :
October 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2014
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT01206075
Start Date
October 1 2010
End Date
December 1 2014
Last Update
December 30 2014
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
George Papanicolaou Hospital
Thessaloniki, Greece