Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia
Study Details
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
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 Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: VIT-2763 60 mg QD VIT-2763 60 mg administered once daily |
Drug: VIT-2763 60 mg QD
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:
|
| Experimental: VIT-2763 60 mg BID VIT-2763 60 mg administered twice daily |
Drug: VIT-2763 60 mg BID
Participants receive one VIT-2763 60 mg capsule plus one Placebo capsule orally, twice daily for 24 weeks.
Other Names:
|
| Experimental: VIT-2763 120 mg BID VIT-2763 120 mg administered twice daily |
Drug: VIT-2763 120 mg BID
Participants receive two VIT-2763 60 mg capsules orally, twice daily for 24 weeks.
Other Names:
|
| Placebo Comparator: Placebo Placebo capsule administered twice daily |
Drug: Placebo
Participants receive two Placebo capsules matching VIT-2763 orally, twice daily for 24 weeks.
|
Outcome Measures
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 [Week 13 to Week 24 comparing to Baseline (Day -83 to Day 1)]
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. [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)]
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).
Eligibility Criteria
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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Investigator site #710 | Whittier | California | United States | 90603-2137 |
| 2 | Investigational site #802 | Plovdiv | Bulgaria | ||
| 3 | Investigational site #801 | Sofia | Bulgaria | ||
| 4 | Investigational site #804 | Stara Zagora | Bulgaria | ||
| 5 | Investigator Site 404 | Jerusalem | Israel | ||
| 6 | Investigator Site 406 | Petah tikva | Israel | ||
| 7 | Investigator Site 405 | Safed | Israel |
Sponsors and Collaborators
- Vifor (International) Inc.
- Labcorp Drug Development, Inc.
Investigators
- Study Director: Peter Szecsödy, Vifor (International) Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- VIT-2763-THAL-203
- 2021-001639-23