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

NCT04938635 | Withdrawn Phase 2 DMC FDA Drug

• 2 other identifiers: VIT-2763-THAL-2032021-001639-23
• Study Type: interventional
• Target: N/A
• Locations: 3 countries
• Sites: 7

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.

Conditions

Beta-Thalassemia

Keywords

Beta-Thalassemia; Transfusion-dependent Thalassemia

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• 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)

• 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)

• 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)

• Mean change from baseline in Quality of Life (QoL) total score

  Week 15 and Week 24 comparing to Baseline (Day 1)

Study Arms (4)

VIT-2763 60 mg QD

EXPERIMENTAL

VIT-2763 60 mg administered once daily

Drug: VIT-2763 60 mg QD

VIT-2763 60 mg BID

EXPERIMENTAL

VIT-2763 60 mg administered twice daily

Drug: VIT-2763 60 mg BID

VIT-2763 120 mg BID

EXPERIMENTAL

VIT-2763 120 mg administered twice daily

Drug: VIT-2763 120 mg BID

Placebo

PLACEBO COMPARATOR

Placebo capsule administered twice daily

Drug: Placebo

Interventions

VIT-2763 60 mg QD DRUG

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.

Also known as: Vamifeport 60 mg QD

VIT-2763 60 mg QD

VIT-2763 60 mg BID DRUG

Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally, twice daily for 24 weeks.

Also known as: Vamifeport 60 mg BID

VIT-2763 60 mg BID

VIT-2763 120 mg BID DRUG

Participants receive two VIT-2763 60 mg capsules orally, twice daily for 24 weeks.

Also known as: Vamifeport 120 mg BID

VIT-2763 120 mg BID

Placebo DRUG

Participants receive two Placebo capsules matching VIT-2763 orally, twice daily for 24 weeks.

Placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Vifor (International) Inc.: lead
• Labcorp Corporation of America Holdings, Inc: collaborator

Study Sites (7)

Investigator site #710

Whittier, California, 90603-2137, United States

Location

Investigational site #802

Plovdiv, Bulgaria

Location

Investigational site #801

Sofia, Bulgaria

Location

Investigational site #804

Stara Zagora, Bulgaria

Location

Investigator Site 404

Jerusalem, Israel

Location

Investigator Site 406

Petah Tikva, Israel

Location

Investigator Site 405

Safed, Israel

Location

MeSH Terms

Conditions

beta-Thalassemia

Interventions

VIT-2763; BID protein, human

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Peter Szecsödy

  Vifor (International) Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 16, 2021
• First Posted: June 24, 2021
• Study Start: September 20, 2021
• Primary Completion: July 1, 2023
• Study Completion: July 1, 2023
• Last Updated: November 10, 2022

Record last verified: 2022-03

Data Sharing

• IPD Sharing: Will not share

Locations

IL (3); US (1); BU (3)