A Phase 3 Double-Blind Randomized Placebo-Controlled Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects with Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T) AG348-C-018
Recruiting
99 years or below
All
Phase
3
5 participants needed
1 Location
Brief description of study
To compare the effect of
mitapivat versus placebo on
transfusion burden in subjects
with alpha- or beta-transfusiondependent
thalassemia (TDT) Transfusion reduction response (TRR), defined as a
50% reduction in transfused red blood cell (RBC)
units with a reduction of greater that or equal to 2 units of transfused
RBCs in any consecutive 12-week period through
Week 48 compared with baseline - greater than or equal to 33 percent reduction in transfused RBC units from
Week 13 through Week 48 compared with baseline
(TRR3)
- greater than or equal to 50 percent reduction in transfused RBC units in any
consecutive 24-week period through Week 48
compared with baseline (TRR2)
- greater than or equal to 50 percent reduction in transfused RBC units from
Week 13 through Week 48 compared with baseline
(TRR4)
- Change from baseline in transfused RBC units from
Week 13 through Week 48
- Transfusion independence, defined as transfusion-free for greater than or equal to 8 consecutive weeks through
Week 48
- Change from baseline in iron, serum ferritin, total
iron binding capacity, and transferrin saturation
through Week 48
- Type, severity, and relationship of adverse events
(AEs) and serious adverse events
- Plasma or blood concentrations and pharmacokinetic
parameters of mitapivat and pharmacodynamic
parameters, including adenosine triphosphate (ATP)
and 2,3-diphosphoglycerate (2,3-DPG)
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Medical Research
-
Age: 99 years or below
-
Gender: All
Updated on
08 Feb 2023.
Study ID: 850647