A pilot study on the safety and efficacy of haemopoietic stem cell mobilization (CD34+ cells) with MOZOBIL ± G-CSF, in adult patients diagnosed with beta-thalassaemia major.

• Conditions: Beta-thalassaemia major; MedDRA version: 12.0Level: LLTClassification code 10004505Term: Beta thalassaemia; MedDRA version: 12.0Level: LLTClassification code 10004514Term: Beta-thalassaemia; MedDRA version: 12.0Level: LLTClassification code 10043391Term: Thalassaemia beta

• Registration Number: EUCTR2009-014136-37-GR
• Lead Sponsor: niversity of Washington

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-06-15
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

a) ?eta- thalassemia major
b) Age >18<50
c) Karnofsky performance status ³80%
d) Splenectomized patients or patients with spleen volume <800cm3 (only for the non splenectomized patients
who will receive Mozobil + G-CSF) [V=0.523 x length x thickness x width (De Odorico I et al, 1999)]
e) Compliant with regular transfusions and regular chelation
f) Liver iron by MRI <280µmol/gr or 1.7msec by T2*MRI
g) Heart iron by MRI >2.8 (SI/SD) or 9msec by T2*MRI
h) Hepatitis B or C virus load negative by PCR
i) Left ventricular ejection fraction (LVEF) >45% by echocardiogram
j) Adequate respiratory function with DLCO >50%
k) Negative pregnancy test, if female
l) Ability to give informed consent and willingness to meet all the expected requirements of the protocol for the
duration of the study
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years)
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

a) History of thrombosis or known thrombophilia
b) Symptomatic viral, bacterial or fungal infection within 6 weeks prior eligibility evaluation
c) Pregnancy or lactation
d) HIV positivity
e) History of malignancy, other than local skin cancer
f) Other systematic disease non thalassemia-associated
g) Splenectomized patients with platelet count >900,000 (only for the splenectomized patients who will receive
low dose G-CSF+ Mozobil)
h) 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified