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

• Status: Recruiting
• Trial end date: 2023-07-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Vifor (International) Inc.
• Collaborator: Covance

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.