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