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

Beta-Thalassemia Clinical Trial

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT04938635
• Other study ID #: VIT-2763-THAL-203
• Secondary ID: 2021-001639-23
• Status: Withdrawn
• Phase: Phase 2
• Start date: September 20, 2021
• Est. completion date: July 2023

Study information

• Verified date: March 2022
• Source: Vifor Pharma
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: July 2023
• Est. primary completion date: July 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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 (dß)-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.

Related Conditions & MeSH terms

• Beta-Thalassemia
• Thalassemia

Intervention

Drug:
• VIT-2763 60 mg QD
Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally in the morning, and 2 Placebo capsules orally in the evening, for 24 weeks.
• VIT-2763 60 mg BID
Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally, twice daily for 24 weeks.
• VIT-2763 120 mg BID
Participants receive two VIT-2763 60 mg capsules orally, twice daily for 24 weeks.
• Placebo
Participants receive two Placebo capsules matching VIT-2763 orally, twice daily for 24 weeks.

Locations

Country	Name	City	State
Bulgaria	Investigational site #802	Plovdiv
Bulgaria	Investigational site #801	Sofia
Bulgaria	Investigational site #804	Stara Zagora
Israel	Investigator Site 404	Jerusalem
Israel	Investigator Site 406	Petah tikva
Israel	Investigator Site 405	Safed
United States	Investigator site #710	Whittier	California

Sponsors (2)

Lead Sponsor	Collaborator
Vifor (International) Inc.	Labcorp Drug Development Inc

Countries where clinical trial is conducted

United States,  Bulgaria,  Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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		Week 13 to Week 24 comparing to Baseline (Day -83 to Day 1)
Secondary	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.		Week 13 to Week 24 comparing to Baseline (Day -83 to Day 1)
Secondary	Proportion of patients achieving =50% reduction of RBC transfusions and =2 units assessed over any consecutive 12-week interval from Week 1 to 24.		Any consecutive 12-week interval from Week 1 to Week 24 comparing to Baseline (Day -83 to Day 1)
Secondary	Proportion of patients achieving =33% reduction of RBC transfusions and =2 units assessed over any consecutive 12-week interval from Week 1 to 24.		Any consecutive 12-week interval from Week 1 to Week 24 comparing to Baseline (Day -83 to Day 1)
Secondary	Mean change from baseline in Quality of Life (QoL) total score	Transfusion-dependent QoL Questionnaire (TranQuol): a disease-specific, validated, QoL measure developed for thalassemia patients. The adult version includes 36 questions grouped into 5 domains: physical health, emotional health, sexual health, family functioning, and school/career functioning. The total score ranges from 0 (worst) to 100 (best).	Week 15 and Week 24 comparing to Baseline (Day 1)