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

• Status: Completed
• Trial end date: 2014-12-01
• Target enrollment: 0
• Participant gender: All

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 only cure for thalassemia is bone marrow transplantation from a related, compatible donor. Gene transfer, achieved by transplantation of the patient's own blood stem cells that have been genetically-modified with the corrected gene, could potentially cure thalassemia.

The first step in developing gene transfer for treatment of thalassemia is to develop a safe and effective method to obtain blood stem cells from thalassemia patients. Eventually, high numbers of genetically modified cells will need to be infused into the patient for clinical gene transfer to be effective. The blood stem cells are obtained by giving a "mobilization" agent to the patients. This causes the stem cells to leave the bone marrow and go into the blood. The purpose of this study is to test the safety and effectiveness of the new mobilization agent, Mozobil, in causing mobilization of blood stem cells for patients with beta-thalassemia major.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Washington
• Collaborators: Genzyme, a Sanofi Company; George Papanicolaou Hospital; National Heart, Lung, and Blood Institute (NHLBI)
• Treatments: JM 3100; Lenograstim; Plerixafor

Criteria

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.