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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07506863 | A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) | PHASE3 | NOT YET_RECRUITING | 54 | — | — | Sep 1, 2026 | Jun 1, 2032 | May 29, 2026 | - | — |
| NCT04770779 | A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) | PHASE3 | ACTIVE NOT_RECRUITING | 258 | — | — | Nov 30, 2021 | Jun 1, 2029 | Jun 1, 2026 | 75 | United States, Brazil +17 |
TRR is defined as ≥50 percent (%) reduction in transfused red blood cells (RBC) volume (normalized by weight) in any consecutive 12-week period through Week 48 compared with historical RBC transfusion volume (normalized by weight) and standardized to 12 weeks.
TRR is defined as ≥50% reduction in transfused red blood cells (RBC) units with a reduction of ≥2 units of transfused RBCs in any consecutive 12-week period through Week 48 compared with baseline transfusion burden standardized to 12 weeks. Baseline transfusion burden standardized to 12 weeks= (12/24) × total number of RBC units transfused during 24-week period before the randomization date for participants randomized and not dosed or the start of study treatment for participants randomized and dosed. Participants withdrawn from the study before Week 12 (Day 85) were considered nonresponders.
| Arm | Type | Description |
|---|---|---|
| Mitapivat | EXPERIMENTAL | Participants will receive oral mitapivat twice daily (BID), with dose determined by age and weight, for 48 weeks during the double blind (DB) period. Enrollment is conducted sequentially by age cohort, beginning with the oldest cohort. Cohort 1: 12 to \<18 years Cohort 2: 6 to \<12 years Cohort 3: 1 to \<6 years Participants who complete the DB period may continue receiving mitapivat in the open label extension (OLE) period for up to 144 weeks. |
| Placebo | PLACEBO_COMPARATOR | Participants will receive oral placebo matching mitapivat, administered BID, with dosing based on age and weight, for 48 weeks during the DB period. Enrollment is conducted sequentially by age cohort, beginning with the oldest cohort. Cohort 1: 12 to \<18 years Cohort 2: 6 to \<12 years Cohort 3: 1 to \<6 years Participants who complete the DB period may transition to receive mitapivat in the OLE period for up to 144 weeks. |
| Name | Type | Description |
|---|---|---|
| Mitapivat Matched Placebo | DRUG | Tablets or Granules |
| Mitapivat | DRUG | Tablets or Granules |
| Placebo Matching Mitapivat | DRUG | Tablets |
Inclusion Criteria: * Written informed consent/assent from the participant (or their legally authorized representative, parent(s), or legal guardian) must be obtained before any study-related procedures are conducted and participants must be willing to comply with all study procedures for the durat...