Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia Major

Plain-language summary

This study evaluates the safety and effectiveness of using the drug Mozobil to mobilize stem cells into the blood in adults with beta-thalassemia major. It included patients aged 18-50 with beta-thalassemia major who received regular transfusions and chelation therapy. It was carried out as a study with no assigned phase and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Not Applicable

Status

Completed

Sponsor

University of Washington

Intervention

Mozobil, Mozobil, Mozobil

Start date

10/1/2010

Estimated completion

12/1/2014

Countries

Greece

Related genes

Locations

• Thessaloniki, Greece

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* 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 Criteria:

* 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.