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

Brief Title

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

Official Title

A Phase 2b, Double-blind, Randomised, Placebo-controlled, Multicentre Study to Assess the Efficacy and Safety of VIT-2763 Multiple Doses in Adults With Transfusion-dependent Beta-thalassaemia

Brief 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.
    

Detailed Description

      All patients giving written informed consent will undergo a 12-week screening period to
      determine eligibility for study entry. At Day 1, patients who meet the eligibility
      requirements will be randomized in a double-blind manner to 1 of 4 treatment groups: VIT-2763
      60 mg (once daily), 60 mg (twice daily), or 120 mg (twice daily) or placebo.

      The randomisation will be stratified (balanced allocation across treatment groups) according
      to β0/β0 genotype (yes/no).

      The duration of the treatment with VIT-2763 or placebo is 24 weeks, after which patients will
      undergo a 12-week post-treatment follow-up period.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Proportion of patients achieving ≥33% reduction of RBC transfusions from baseline and a reduction of ≥2 units assessed consecutively from Week 13 to Week 24 compared to the baseline transfusion

Secondary Outcome

 Change from baseline in RBC transfusions over Weeks 13 to 24 compared to the baseline RBC transfusion burden derived using the last 12 weeks prior to randomization.

Condition

Beta-Thalassemia

Intervention

VIT-2763 60 mg QD

Study Arms / Comparison Groups

 VIT-2763 60 mg QD
Description:  VIT-2763 60 mg administered once daily

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

80

Start Date

September 20, 2021

Completion Date

July 2023

Primary Completion Date

July 2023

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.
      

Gender

All

Ages

18 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

Peter Szecsödy, , 

Location Countries

Bulgaria

Location Countries

Bulgaria

Administrative Informations


NCT ID

NCT04938635

Organization ID

VIT-2763-THAL-203

Secondary IDs

2021-001639-23

Responsible Party

Sponsor

Study Sponsor

Vifor (International) Inc.

Collaborators

 Labcorp Drug Development, Inc.

Study Sponsor

Peter Szecsödy, Study Director, Vifor (International) Inc.


Verification Date

March 2022