A phase I/II study evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment of patients affected by transfusion dependent beta-thalassemia - TIGET-BTHA

OSPEDALE SAN RAFFAELE0 sites10 target enrollmentNovember 21, 2014

Overview

No summary available.

Registry

who.int

Start Date

November 21, 2014

End Date

November 25, 2019

Last Updated

4 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Sponsor

OSPEDALE SAN RAFFAELE

•Written informed consent
•Transfusion\-dependent beta\-thalassemia (any genotype). Transfusion dependence is defined as receiving \= 8 transfusions of blood per year over a minimum of 2 years.
•Karnofsky Index or Lansky \> 80%
•Age \= 3 years and \< 65 years
•Adequate cardiac, renal, hepatic and pulmonary functions as evidenced by:
•?Left ventricular ejection fraction (LVEF) greater than 45% by echo and normal ECG or presence of abnormalities not significant for cardiac disease. Absence of severe pulmonary hypertension
•?Diffusing capacity of the lung for carbon monoxide (DLCO) \> 50% and forced expiratory volume in 1 sec (FEV1\) and forced expiratory vital capacity (FVC) \> 60% predicted
•(if non cooperative: pulse oximetry \> 95 % in room air)
•?Serum creatinine \< 1\.5 upper limit of normal
•?Absent\-mild\-moderate liver iron overload on T2\*MRI (less than 12 months before enrolment)

•Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long\-acting agents)
•Severe, active viral, bacterial, or fungal infection at eligibility evaluation
•Malignant neoplasia (except local skin cancer or cervical intraepithelial neoplasia) or exceptional family history of familial cancer syndromes
•Myelodysplasia, cytogenetic alterations associated with neoplasia, or other serious haematological disorder than thalassemia
•History of uncontrolled seizures
•Other clinical conditions judged non compatible with the procedure and/or the treatment
•Positivity for HIV (serology or RNA), and/or HbsAg and/or HBV DNA and/or HCV RNA (or negative HCV RNA but on antiviral treatment) and/or Treponema Pallidum or Mycoplasma active infection
•Active alcohol or substance abuse within 6 months of the study
•Pregnancy or lactation
•Previous allogeneic bone marrow transplantation or gene therapy