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| ID | Type | Description | Link |
|---|---|---|---|
| 2021-001639-23 | EudraCT Number |
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Strategic reasons
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| Name | Class |
|---|---|
| Labcorp Corporation of America Holdings, Inc | INDUSTRY |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| VIT-2763 60 mg QD | Experimental | VIT-2763 60 mg administered once daily |
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| VIT-2763 60 mg BID | Experimental | VIT-2763 60 mg administered twice daily |
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| VIT-2763 120 mg BID | Experimental | VIT-2763 120 mg administered twice daily |
|
| Placebo | Placebo Comparator | Placebo capsule administered twice daily |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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) |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Peter Szecsödy | Vifor (International) Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigator site #710 | Whittier | California | 90603-2137 | United States | ||
| Investigational site #802 |
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| VIT-2763 60 mg BID | Drug | Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally, twice daily for 24 weeks. |
|
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| VIT-2763 120 mg BID | Drug | Participants receive two VIT-2763 60 mg capsules orally, twice daily for 24 weeks. |
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| Placebo | Drug | Participants receive two Placebo capsules matching VIT-2763 orally, twice daily for 24 weeks. |
|
| 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 | 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) |
| Plovdiv |
| Bulgaria |
| Investigational site #801 | Sofia | Bulgaria |
| Investigational site #804 | Stara Zagora | Bulgaria |
| Investigator Site 404 | Jerusalem | Israel |
| Investigator Site 406 | Petah Tikva | Israel |
| Investigator Site 405 | Safed | Israel |
| ID | Term |
|---|---|
| D017086 | beta-Thalassemia |
| ID | Term |
|---|---|
| D013789 | Thalassemia |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C000708305 | VIT-2763 |
| C494814 | BID protein, human |
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