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

Primary Purpose

Beta-Thalassemia

Status
Withdrawn
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
VIT-2763 60 mg QD
VIT-2763 60 mg BID
VIT-2763 120 mg BID
Placebo
Sponsored by
Vifor (International) Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Beta-Thalassemia focused on measuring Beta-Thalassemia, Transfusion-dependent Thalassemia

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Investigator site #710
  • Investigational site #802
  • Investigational site #801
  • Investigational site #804
  • Investigator Site 404
  • Investigator Site 406
  • Investigator Site 405

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Placebo Comparator

Arm Label

VIT-2763 60 mg QD

VIT-2763 60 mg BID

VIT-2763 120 mg BID

Placebo

Arm Description

VIT-2763 60 mg administered once daily

VIT-2763 60 mg administered twice daily

VIT-2763 120 mg administered twice daily

Placebo capsule administered twice daily

Outcomes

Primary Outcome Measures

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 Measures

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.
Proportion of patients achieving ≥50% reduction of RBC transfusions and ≥2 units assessed over any consecutive 12-week interval from Week 1 to 24.
Proportion of patients achieving ≥33% reduction of RBC transfusions and ≥2 units assessed over any consecutive 12-week interval from Week 1 to 24.
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).

Full Information

First Posted
June 16, 2021
Last Updated
November 7, 2022
Sponsor
Vifor (International) Inc.
Collaborators
Labcorp Corporation of America Holdings, Inc
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1. Study Identification

Unique Protocol Identification Number
NCT04938635
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
Study Type
Interventional

2. Study Status

Record Verification Date
March 2022
Overall Recruitment Status
Withdrawn
Why Stopped
Strategic reasons
Study Start Date
September 20, 2021 (Actual)
Primary Completion Date
July 2023 (Anticipated)
Study Completion Date
July 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vifor (International) Inc.
Collaborators
Labcorp Corporation of America Holdings, Inc

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Beta-Thalassemia
Keywords
Beta-Thalassemia, Transfusion-dependent Thalassemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Arm Title
VIT-2763 60 mg QD
Arm Type
Experimental
Arm Description
VIT-2763 60 mg administered once daily
Arm Title
VIT-2763 60 mg BID
Arm Type
Experimental
Arm Description
VIT-2763 60 mg administered twice daily
Arm Title
VIT-2763 120 mg BID
Arm Type
Experimental
Arm Description
VIT-2763 120 mg administered twice daily
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo capsule administered twice daily
Intervention Type
Drug
Intervention Name(s)
VIT-2763 60 mg QD
Other Intervention Name(s)
Vamifeport 60 mg QD
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
VIT-2763 60 mg BID
Other Intervention Name(s)
Vamifeport 60 mg BID
Intervention Description
Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally, twice daily for 24 weeks.
Intervention Type
Drug
Intervention Name(s)
VIT-2763 120 mg BID
Other Intervention Name(s)
Vamifeport 120 mg BID
Intervention Description
Participants receive two VIT-2763 60 mg capsules orally, twice daily for 24 weeks.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Participants receive two Placebo capsules matching VIT-2763 orally, twice daily for 24 weeks.
Primary Outcome Measure Information:
Title
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
Time Frame
Week 13 to Week 24 comparing to Baseline (Day -83 to Day 1)
Secondary Outcome Measure Information:
Title
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.
Time Frame
Week 13 to Week 24 comparing to Baseline (Day -83 to Day 1)
Title
Proportion of patients achieving ≥50% reduction of RBC transfusions and ≥2 units assessed over any consecutive 12-week interval from Week 1 to 24.
Time Frame
Any consecutive 12-week interval from Week 1 to Week 24 comparing to Baseline (Day -83 to Day 1)
Title
Proportion of patients achieving ≥33% reduction of RBC transfusions and ≥2 units assessed over any consecutive 12-week interval from Week 1 to 24.
Time Frame
Any consecutive 12-week interval from Week 1 to Week 24 comparing to Baseline (Day -83 to Day 1)
Title
Mean change from baseline in Quality of Life (QoL) total score
Description
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).
Time Frame
Week 15 and Week 24 comparing to Baseline (Day 1)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Peter Szecsödy
Organizational Affiliation
Vifor (International) Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Investigator site #710
City
Whittier
State/Province
California
ZIP/Postal Code
90603-2137
Country
United States
Facility Name
Investigational site #802
City
Plovdiv
Country
Bulgaria
Facility Name
Investigational site #801
City
Sofia
Country
Bulgaria
Facility Name
Investigational site #804
City
Stara Zagora
Country
Bulgaria
Facility Name
Investigator Site 404
City
Jerusalem
Country
Israel
Facility Name
Investigator Site 406
City
Petah tikva
Country
Israel
Facility Name
Investigator Site 405
City
Safed
Country
Israel

12. IPD Sharing Statement

Plan to Share IPD
No

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

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