Beta-Thalassemia

Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia

NCT04938635 | PHASE 2 | INTERVENTIONAL

The main purpose of this study is to evaluate the efficacy of 3 multiple doses of VIT-2763 as measured by the reduction in red blood cell (RBC) transfusion burden from Week 13 to Week 24, to identify the most efficacious and safe dose.

7 Sites

0 Participants

Recruiting

18 Years to 65 Years

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Investigator site #

Whittier,California,United States,90603

Investigational site #

Plovdiv,Bulgaria

Investigational site #

Sofia,Bulgaria

Investigational site #

Stara Zagora,Bulgaria

Investigator Site

Jerusalem,Israel

Investigator Site

Petah tikva,Israel

Investigator Site

Safed,Israel

Study Eligibility Criteria

Inclusion Criteria

* Body weight ≥40.0 kg and ≤100 kg at screening
* Documented diagnosis of beta-thalassemia or Hb E/beta-thalassemia
* Red blood cell (RBC) transfusion dependence, defined as at least 6 RBC units in the 24 weeks prior to randomization and no transfusion-free period for ≥35 days during that period
* Ability to understand the requirements of the study and provide written informed consent

Exclusion Criteria

* Documented diagnosis of Hb S/beta-thalassemia, alpha-thalassemia, or delta beta (δβ)-thalassemia, or hereditary persistence of foetal Hb.
* History of partial or total splenectomy within 4 months prior to screening.
* History of myocardial iron overload
* Chronic liver disease or history of liver cirrhosis
* Clinically relevant renal disease
* History or clinically important finding of cardiac disorders
* History of clinically significant lung disease
* Uncontrolled hypertension (\> Grade 1 according to NCI CTCAE current version)
* Unable to take and absorb oral medications.
* Pregnancy or breastfeeding
* History of drug or alcohol abuse within 2 years prior to screening
* History or concomitant solid tumors and/or hematological malignancies unless resolved in the ≥5 past years.

Additional Studies

Additional studies can be found at ClinicalTrials.gov