Mitapivat for Non-Transfusion-dependent Alpha-Thalassemia
Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Non-Transfusion-dependent Alpha-Thalassemia+1 MoreMitapivat - Drug
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Eligibility
18+
All Sexes
What conditions do you have?
Select
Eligibility
18+
All Sexes
What conditions do you have?
Select
Study Summary
This trial will compare the effect of the drug mitapivat versus placebo on anemia in people with either alpha- or beta-non-transfusion dependent thalassemia.
Change From Baseline in Average Functional Assessment of Chronic Illness Therapy (FACIT) -Fatigue Subscale Score from Week 12 through Week 24
Change From Baseline in Average Hb Concentration From Week 12 through Week 24
Percentage of Participants With Hb 1.5+ Response
Percentage of Participants With Hemoglobin (Hb) Response
Baseline, Week 24
Change From Baseline in Erythropoietin at Week 24
Change From Baseline in Haptoglobin at Week 24
Change From Baseline in Indirect Bilirubin at Week 24
Change From Baseline in Lactate Dehydrogenase (LDH) at Week 24
Change From Baseline in Reticulocytes at Week 24
Change From Baseline in Serum Ferritin at Week 24
Change From Baseline in Transferrin Saturation (TSAT) at Week 24
Change From Baseline in the 6-minute Walk Test (6MWT) Distance at Week 24
Week 12
Percentage of Participants With Improvement in the Patient Global Impression of Severity (PGIS) -Fatigue by at Least 1 Category at Weeks 12, 16, 20, and 24 Compared With Baseline, or "No Change" if No or Mild Fatigue at Baseline
Week 20
Blood Concentration of 2,3 - diphosphoglycerate (2,3-DPG)
Blood Concentration of Adenosine Triphosphate (ATP)
Week 20
Plasma or Blood Concentrations Over Time for Mitapivat
Week 20
Area Under the Concentration-time Curve From Time Zero to the Last Measurable Concentration (AUClast) of Mitapivat
Maximum Plasma Concentration (Cmax) of Mitapivat
Time of Maximum Plasma Concentration (Tmax) of Mitapivat
Up to Week 293
Percentage of Participants With Adverse Events (AEs) Considered by the Investigator to be Related to Study Drug
Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs), Graded by Severity
Percentage of Participants With Serious Adverse Events (SAEs) Considered by the Investigator to be Related to Study Drug
Week 12
Percentage of Participants With Improvement in the Patient Global Impression of Change (PGIC) -Fatigue at Weeks 12, 16, 20, and 24, or "No Change" if No or Mild Fatigue at Baseline