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

Recruiting

12 Years to 65 Years

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Clinical Site #

Athens,Attiki,Greece,11527

Clinical Site #

Rio,Greece,26504

Clinical Site #

Thessaloniki,Greece,54642

Clinical Site #

Afula,Israel,18411

Clinical Site #

Haifa,Israel,34362

Clinical Site #

Petah Tikva,Israel,49100

Clinical Site #

Milan,MI,Italy,20122

Clinical Site #

Napoli,Italy,80131

Clinical Site #

Napoli,Italy,80138

Clinical Site #

Orbassano,Italy,10043

Clinical Site #

Palermo,Italy,90146

Clinical Site #

Verona,Italy,37134

Clinical Site #

Beirut,Lebanon,11-0236b

Clinical Site #

Bangkok,Thailand,10700

Clinical Site #

Chiang Mai,Thailand,50200

Clinical Site #

Phitsanulok,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