Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04364269 | Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia | PHASE2 | COMPLETED | 35 | — | — | Jun 11, 2020 | Nov 3, 2021 | Dec 14, 2023 | 16 | Greece, Israel +3 |
Please note that in this section we are presenting just the overview of the adverse events experienced by the trial participants, in particular, the number of participants with at least one TEAE. Please refer to the detailed tables included on the Adverse Event Module for specifics.
Summary of the values by visit from baseline and changes from baseline by post-baseline visit.
Summary of the values by visit from baseline and changes from baseline by post-baseline visit.
Values by visit from baseline and changes from baseline by post-baseline visit. The following ECG parameters were recorded: PR interval, QRS duration, QT interval, RR interval and QTcF interval. PR interval represents the time from the onset of the P wave to the start of the QRS complex. QRS duration represents the time required for a stimulus to spread through the ventricles (ventricular depolarization). QT interval represents the time from the start of the Q wave to the end of the T wave. RR interval represents the time from the onset of one R wave to the onset of the next one, one complete cardiac cycle. QT corrected for heart rate (QTc) interval reflects ventricular repolarization.
| Arm | Type | Description |
|---|---|---|
| VIT-2763 Once a day (QD) | EXPERIMENTAL | Participants will be assigned to receive VIT-2763 once a day (QD) in a total daily dose of 60 mg or 120 mg depending on their body weight. The study medication (VIT-2763 and/or matching placebo) will be administered for all participants twice a day to maintain the blind. |
| VIT-2763 Twice a day (BID) | EXPERIMENTAL | Participants will be assigned to receive VIT-2763 Twice a day (BID) in a total daily dose of 60 mg or 120 mg depending on their body weight. |
| Placebo | PLACEBO_COMPARATOR | Participants will be assigned to receive Placebo, Twice a day. |
| Name | Type | Description |
|---|---|---|
| VIT-2763 once a day (QD) | DRUG | Participants will receive VIT-2763 QD at a dose of 60 mg if their body weight is between 40 kg to 59 kg or at a dose of 120 mg if their body weight is between 60 kg and 100 kg, during 12 weeks. |
| VIT-2763 twice a day (BID) | DRUG | Participants will receive VIT-2763 BID at a dose of 60 mg if their body weight is between 40 kg to 59 kg or at a dose of 120 mg if their body weight is between 60 kg and 100 kg, during 12 weeks. |
| Placebo | DRUG | Participants will receive hard capsules of Placebo, twice a day. |
Inclusion Criteria: * Documented diagnosis of NTDT, including a β-thalassemia intermedia-phenotype. * NTDT is defined as subjects having received less than 5 units of red blood cells (RBCs) during the 24-week period prior to randomisation/first drug administration of VIT-2763 or placebo (Day 1; 1 u...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Agios Pharmaceuticals, Inc. | AGIO | 5 | PHASE3 | Mitapivat, AG-348 |
| Bristol-Myers Squibb Company | BMY | 6 | PHASE3 | Luspatercept, ACE-536 |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 2 | PHASE3 | Etavopivat A, Etavopivat B, Etavopivat C |
| Regeneron Pharmaceuticals, Inc. | REGN | 1 | PHASE2 | REGN7999 |
| ICON Plc | ICLR | 1 | PHASE2 | SP-420 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
| Illumina, Inc. | ILMN | 1 | — | Undisclosed |