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

Phase 1

• Conditions: transfusiondependent ß-thalassaemia; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2021-001639-23-GR
• Lead Sponsor: Vifor (International) Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-10-22
• Last Update Posted: 18 July 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 0

Inclusion Criteria

• Body weight =40.0 kg and =100 kg at screening
• Documented diagnosis of beta-thalassaemia or Hb E/beta-thalassaemia
• Red blood cell (RBC) transfusion dependence, defined as at least 6 RBC units in the 24 weeks prior to randomisation and no transfusion-free period for =35 days during that period
• Ability to understand the requirements of the study and provide written informed consent
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 79
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

• Documented diagnosis of Hb S/beta-thalassaemia, alfa-thalassaemia, or delta beta (dß)- thalassaemia, 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 tumours and/or haematological malignancies unless resolved in the =5 past years.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified