transfusion dependent beta thalassemia MedDRA version: 14.0 Level: PT Classification code 10043391 Term: Thalassaemia beta System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Pharmaceutical Form: Solution for injection
INN or Proposed INN: plerixafor
Concentration unit: mg milligram(s)
Concentration number: 20-
Pharmaceutical Form: Solution for injection
INN or Proposed I
Sponsors
FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Transfusion-dependent beta-thalassemia (any genotype) • = 18 years • Karnofsky Index > 80 % • Adequate cardiac, renal, hepatic and pulmonary functions as evidenced by: o LVEF greater than 45% by echo and normal ECG or presence of abnormalities not significant for cardiac disease o DLCO diffusing capacity > 50% and FEV1 and FVC > 60% predicted o Serum creatinine =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents) • Symptomatic viral, bacterial, or fungal infection within 6 weeks of eligibility evaluation or active infection (included fever of unknown origin) • Neoplasia (except local skin cancer or cervical intraepithelial neoplasia) or history of “familial” cancer • Myelodysplasia or other serious hematological disorder than thalassemia • History of uncontrolled seizures • Other systemic disease judged non compatible with the procedure • Positivity for HIV and/or HCV RNA and/or HbsAg and/or HBV DNA • Active alcohol or substance abuse within 6 months of the study • Pregnancy or lactation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 1. To explore the safety of Plerixafor administration to patients affected by transfusion dependent betathalassemia Efficacy 2. To explore the efficacy of Plerixafor in mobilizing CD34+ cells in patients affected by transfusion dependent beta-thalassemia;Secondary Objective: 1. To explore the efficiency of transduction of Plerixafor mobilized CD34+ cells and Plerixafor primed BM cells from patients with beta-thalassemia using a lentiviral vector encoding for human beta globin 2. To evaluate yield of CD34+ cells harvest from BM following administration of Plerixafor (primed-BM). 3. To characterize Plerixafor mobilized CD34+ cells from thalassemic patients.;Primary end point(s): Safety 1.Incidence and severity of adverse events following Plerixafor administration (any grade) occurring between day 0 and day 30. Efficacy 2.Harvest of > 5x10^6 CD34/kg body weight in at least 3 out of 6 patients. | — |
Countries
Italy
Outcome results
None listed