Beta-Thalassemia

Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia

NCT04364269 | PHASE 2 | INTERVENTIONAL

This is a randomised, double-blind, placebo-controlled parallel group trial to investigate the safety, tolerability and efficacy of multiple doses of VIT-2763 versus placebo in participants with non-transfusion-dependent Beta-thalassemia (NTDT).

16 Sites

35 Participants

COMPLETED

12 Years to 65 Years

If interested, contact clinicaltrials@cslbehring.com or +1 610-878-4697 for more information

CSL Behring respects your privacy. For an explanation of how CSL Behring will use the information you are submitting, please view our Privacy Policy

Find closest sites:

SUBMIT

Clinical Site #

Athens,Attiki,Greece,11527

Clinical Site #

Rio,Attiki,Greece,26504

Clinical Site #

Thessaloniki,Attiki,Greece,54642

Clinical Site #

Afula,Attiki,Israel,18411

Clinical Site #

Haifa,Attiki,Israel,34362

Clinical Site #

Petah Tikva,Attiki,Israel,49100

Clinical Site #

Milan,MI,Italy,20122

Clinical Site #

Napoli,MI,Italy,80131

Clinical Site #

Napoli,MI,Italy,80138

Clinical Site #

Orbassano,MI,Italy,10043

Clinical Site #

Palermo,MI,Italy,90146

Clinical Site #

Verona,MI,Italy,37134

Clinical Site #

Beirut,MI,Lebanon,11-0236b

Clinical Site #

Bangkok,MI,Thailand,10700

Clinical Site #

Chiang Mai,MI,Thailand,50200

Clinical Site #

Phitsanulok,MI,Thailand,65000

Study Eligibility Criteria

Inclusion Criteria

* Documented diagnosis of NTDT, including a β-thalassemia intermedia-phenotype.
* NTDT is defined as subjects having received less than 5 units of red blood cells (RBCs) during the 24-week period prior to randomisation/first drug administration of VIT-2763 or placebo (Day 1; 1 unit is defined as 200 to 350 ml of transfused packed RBCs and last RBC transfusion must have been received at east 14 days prior to randomisation).
* Male and female adult NTDT subjects, 18-65 years of age inclusive (Cohort I only) at time of screening.
* Male and female adolescent NTDT subjects, 12-17 years of age inclusive (Cohort II only) at time of screening.
* Subjects must have a mean baseline hemoglobin (Hb) equal to or lower than 11 g/dl, based on at least 2 consecutive measurements with at least 1 week apart within 6 weeks prior to randomisation/baseline.

Exclusion Criteria

* Documented diagnosis of transfusion dependent thalassemia (TDT), including a beta-thalassemia major phenotype (including β0/β0, β+/β+, β0/β+ genotype), and mixed compound heterozygous for sickling phenotype variants such as Hb S/β- thalassemia, or transfusion dependent non-deletional Hb H disease (i.e., Hb constant spring) or Hb C disease.
* Subjects on concomitant iron chelation therapy (ICT) or subjects on prior ICT when discontinued less than 4 weeks prior randomisation. If ICT was discontinued at least 4 weeks prior randomization the subject is eligible.
* ICT naïve subjects or subjects who discontinued ICT therapy at least 6 months before the screening visit with serum ferritin lower than 150 ng/ml and/or documented liver iron concentration (LIC) equal to or lower than 1 mg/g liver dry weight assessed through magnetic resonance imaging (MRI), or subjects on prior ICT with serum ferritin lower than 300 ng/ml and/or documented LIC lower than 3 mg/g liver dry weight assessed through MRI.
* Subjects with transferrin saturation (TSAT) less than 30%.
* Subjects with documented LIC greater than 15 mg/g liver dry weight assessed through MRI, or a documented myocardial T2\* less than 20 ms, if available per local practice and retrieved within 24 months prior to randomization.
* Adult or adolescent subjects with body weight lower than 40.0 kg or greater than 100 kg at screening.
* Chronic liver disease and/or alanine transaminase (ALT), aspartate transaminase (AST) or gamma-glutamyl transpeptidase (GGT) above 3-fold the upper limit of normal (ULN) range at screening.
* Estimated glomerular filtration rate (eGFR) less than 30 ml/min/1.73 m2 (according to chronic kidney disease classification Stage 4 or higher), and/or significant albuminuria greater than 30 mg/mmol. eGFR should be estimated according to Chronic Kidney Disease Epidemiology Collaboration formula (CKI-EPI) in adults, and Schwartz formula in adolescents.
* Newly diagnosed folate deficiency anemia and/or Vitamin B12 megaloblastic anemia. Subjects with known folate deficiency anemia and/or Vitamin B12 megaloblastic anemia who are on at least 12 weeks stable replacement therapy are eligible.
* Any history or clinically important finding of cardiac disorders, such as clinically relevant cardiac arrhythmia, cardiomyopathy, coronary disease, valve disorder, or heart failure according to New York Heart Association classification 3-4.
* Subjects with history of partial or total splenectomy within 6 months prior to screening.

Additional Studies

Additional studies can be found at ClinicalTrials.gov