- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04938635
Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia
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 Overview
Status
Conditions
Intervention / Treatment
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 Type
Phase
- Phase 2
Contacts and Locations
Study Locations
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Plovdiv, Bulgaria
- Investigational site #802
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Sofia, Bulgaria
- Investigational site #801
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Stara Zagora, Bulgaria
- Investigational site #804
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Jerusalem, Israel
- Investigator Site 404
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Petah tikva, Israel
- Investigator Site 406
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Safed, Israel
- Investigator Site 405
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California
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Whittier, California, United States, 90603-2137
- Investigator site #710
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: RANDOMIZED
- Interventional Model: PARALLEL
- Masking: TRIPLE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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EXPERIMENTAL: VIT-2763 60 mg QD
VIT-2763 60 mg administered once daily
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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.
Other Names:
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EXPERIMENTAL: VIT-2763 60 mg BID
VIT-2763 60 mg administered twice daily
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Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally, twice daily for 24 weeks.
Other Names:
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EXPERIMENTAL: VIT-2763 120 mg BID
VIT-2763 120 mg administered twice daily
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Participants receive two VIT-2763 60 mg capsules orally, twice daily for 24 weeks.
Other Names:
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PLACEBO_COMPARATOR: Placebo
Placebo capsule administered twice daily
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Participants receive two Placebo capsules matching VIT-2763 orally, twice daily for 24 weeks.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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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)
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Week 13 to Week 24 comparing to Baseline (Day -83 to Day 1)
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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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)
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Week 13 to Week 24 comparing to Baseline (Day -83 to Day 1)
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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)
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Any consecutive 12-week interval from Week 1 to Week 24 comparing to Baseline (Day -83 to Day 1)
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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)
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Any consecutive 12-week interval from Week 1 to Week 24 comparing to Baseline (Day -83 to Day 1)
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Mean change from baseline in Quality of Life (QoL) total score
Time Frame: Week 15 and Week 24 comparing to Baseline (Day 1)
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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).
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Week 15 and Week 24 comparing to Baseline (Day 1)
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Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Study Director: Peter Szecsödy, Vifor (International) Inc.
Study record dates
Study Major Dates
Study Start (ACTUAL)
Primary Completion (ANTICIPATED)
Study Completion (ANTICIPATED)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ACTUAL)
Study Record Updates
Last Update Posted (ACTUAL)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- VIT-2763-THAL-203
- 2021-001639-23 (EUDRACT_NUMBER)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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