Clinical trial NCT04938635: 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
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Beta-Thalassemia
Keywords: Beta-Thalassemia
Transfusion-dependent Thalassemia
Lead sponsor: Vifor Pharma
Sponsor class: INDUSTRY
Collaborators: name: Labcorp Corporation of America Holdings, Inc
First posted: 2021-06-24
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: 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
timeFrame: Week 13 to Week 24 comparing to Baseline (Day -83 to Day 1)
Secondary outcomes:
measure: 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.
timeFrame: Week 13 to Week 24 comparing to Baseline (Day -83 to Day 1)
measure: Proportion of patients achieving ≥50% reduction of RBC transfusions and ≥2 units assessed over any consecutive 12-week interval from Week 1 to 24.
timeFrame: Any consecutive 12-week interval from Week 1 to Week 24 comparing to Baseline (Day -83 to Day 1)
measure: Proportion of patients achieving ≥33% reduction of RBC transfusions and ≥2 units assessed over any consecutive 12-week interval from Week 1 to 24.
timeFrame: Any consecutive 12-week interval from Week 1 to Week 24 comparing to Baseline (Day -83 to Day 1)
measure: Mean change from baseline in Quality of Life (QoL) total score
timeFrame: Week 15 and Week 24 comparing to Baseline (Day 1)
Other outcomes:
Locations:
city: Whittier
country: United States
facility: Investigator site #710
state: California
city: Plovdiv
country: Bulgaria
facility: Investigational site #802
city: Sofia
country: Bulgaria
facility: Investigational site #801
city: Stara Zagora
country: Bulgaria
facility: Investigational site #804
city: Jerusalem
country: Israel
facility: Investigator Site 404
city: Petah tikva
country: Israel
facility: Investigator Site 406
city: Safed
country: Israel
facility: Investigator Site 405
Study officials:
affiliation: Vifor (International) Inc.
name: Peter Szecsödy
role: STUDY_DIRECTOR
References: