Trial Outcomes & Findings for A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) (NCT NCT04770779)
NCT ID: NCT04770779
Last Updated: 2026-04-24
Results Overview
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 Participants withdrawn from the study before Week 12 (Day 85) were considered nonresponders.
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE3
Target enrollment
258 participants
Primary outcome timeframe
Double-blind Period: Baseline through Week 48
Results posted on
2026-04-24
Participant Flow
Participants took part at 81 study sites in Malaysia,Taiwan,Thailand,Bulgaria,Brazil,Lebanon,Saudi Arabia,Turkey,United Arab Emirates,Canada, the United States,Denmark,France,Germany,Greece,Italy,Netherlands,Spain and the United Kingdom.Results are reported for 48-week DB period until primary completion date.Analysis of data for OLE period is still ongoing with anticipated completion in June 2029.Results for OLE period will be reported by June 2030.
A total of 305 participants with a diagnosis of Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) were screened. Of which, 258 were randomized and 257 received study treatment in this study.
Participant milestones
| Measure |
Mitapivat
Participants randomized to receive mitapivat 100 milligrams (mg), orally, twice daily (BID) for 48 weeks in double-blind period.
|
Placebo
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in double-blind period.
|
|---|---|---|
|
Overall Study
STARTED
|
171
|
87
|
|
Overall Study
Safety Analysis Set
|
172
|
85
|
|
Overall Study
COMPLETED
|
155
|
83
|
|
Overall Study
NOT COMPLETED
|
16
|
4
|
Reasons for withdrawal
| Measure |
Mitapivat
Participants randomized to receive mitapivat 100 milligrams (mg), orally, twice daily (BID) for 48 weeks in double-blind period.
|
Placebo
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in double-blind period.
|
|---|---|---|
|
Overall Study
Randomized, never treated
|
0
|
1
|
|
Overall Study
Adverse Event
|
9
|
1
|
|
Overall Study
Withdrawal by Subject
|
7
|
1
|
|
Overall Study
Pregnancy
|
0
|
1
|
Baseline Characteristics
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
Baseline characteristics by cohort
| Measure |
Mitapivat
n=171 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in double-blind period.
|
Placebo
n=87 Participants
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in double-blind period.
|
Total
n=258 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
35.8 years
STANDARD_DEVIATION 11.61 • n=2 Participants
|
34.7 years
STANDARD_DEVIATION 9.77 • n=1 Participants
|
35.5 years
STANDARD_DEVIATION 11.02 • n=3 Participants
|
|
Sex: Female, Male
Female
|
93 Participants
n=2 Participants
|
43 Participants
n=1 Participants
|
136 Participants
n=3 Participants
|
|
Sex: Female, Male
Male
|
78 Participants
n=2 Participants
|
44 Participants
n=1 Participants
|
122 Participants
n=3 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=2 Participants
|
3 Participants
n=1 Participants
|
8 Participants
n=3 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
158 Participants
n=2 Participants
|
79 Participants
n=1 Participants
|
237 Participants
n=3 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
8 Participants
n=2 Participants
|
5 Participants
n=1 Participants
|
13 Participants
n=3 Participants
|
|
Race/Ethnicity, Customized
White
|
99 Participants
n=2 Participants
|
56 Participants
n=1 Participants
|
155 Participants
n=3 Participants
|
|
Race/Ethnicity, Customized
Asian
|
56 Participants
n=2 Participants
|
22 Participants
n=1 Participants
|
78 Participants
n=3 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
1 Participants
n=2 Participants
|
1 Participants
n=1 Participants
|
2 Participants
n=3 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 Participants
n=2 Participants
|
0 Participants
n=1 Participants
|
0 Participants
n=3 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=2 Participants
|
0 Participants
n=1 Participants
|
0 Participants
n=3 Participants
|
|
Race/Ethnicity, Customized
Multiracial
|
2 Participants
n=2 Participants
|
0 Participants
n=1 Participants
|
2 Participants
n=3 Participants
|
|
Race/Ethnicity, Customized
Unknown
|
7 Participants
n=2 Participants
|
3 Participants
n=1 Participants
|
10 Participants
n=3 Participants
|
|
Race/Ethnicity, Customized
Not reported
|
6 Participants
n=2 Participants
|
5 Participants
n=1 Participants
|
11 Participants
n=3 Participants
|
PRIMARY outcome
Timeframe: Double-blind Period: Baseline through Week 48Population: FAS included all participants who were randomized.
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.
Outcome measures
| Measure |
Mitapivat
n=171 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
n=87 Participants
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Percentage of Participants Who Achieved Transfusion Reduction Response (TRR)
|
30.4 percentage of participants
|
12.6 percentage of participants
|
SECONDARY outcome
Timeframe: Double-blind period: Baseline through Week 48Population: FAS included all participants who were randomized.
TRR2, defined as a ≥50% reduction in transfused RBC units in any consecutive 24-week period through Week 48 compared with the 24-week baseline transfusion burden, is reported. Baseline transfusion burden standardized to 24 weeks is the total number of RBC units transfused during the 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 24 (Day 169) were considered nonresponders.
Outcome measures
| Measure |
Mitapivat
n=171 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
n=87 Participants
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Percentage of Participants Who Achieved TRR2
|
13.5 percentage of participants
|
2.3 percentage of participants
|
SECONDARY outcome
Timeframe: Double-blind period: Baseline up to Week 13 through Week 48Population: FAS included all participants who were randomized.
TRR3, defined as a ≥33% reduction in transfused RBC units from Week 13 through Week 48 compared with the baseline transfusion burden standardized to 36 weeks, is reported. Baseline transfusion burden standardized to 36 weeks= (36/24) × total number of RBC units transfused during the 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 48 were considered nonresponders.
Outcome measures
| Measure |
Mitapivat
n=171 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
n=87 Participants
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Percentage of Participants Who Achieved TRR3
|
14.6 percentage of participants
|
1.1 percentage of participants
|
SECONDARY outcome
Timeframe: Double-blind period: Baseline up to Week 13 through Week 48Population: FAS included all participants who were randomized.
TRR4, defined as a ≥50% reduction in transfused RBC units from Week 13 through Week 48 compared with the baseline transfusion burden standardized to 36 weeks, is reported. Baseline transfusion burden standardized to 36 weeks = (36/24) × total number of RBC units transfused during the 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 48 were considered nonresponders.
Outcome measures
| Measure |
Mitapivat
n=171 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
n=87 Participants
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Percentage of Participants Who Achieved TRR4
|
7.6 percentage of participants
|
1.1 percentage of participants
|
SECONDARY outcome
Timeframe: Double-blind Period: Baseline, Week 13 through Week 48Population: FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure.
Transfusion burden from Week 13 through Week 48 standardized to 36 weeks is defined as number of transfused RBC units from Day 85 through Week 48 in the Double-blind Period ×36/(Number of days from Day 85 through Week 48 in the Double-blind Period/7). Baseline transfusion burden standardized to 36 weeks= (36/24)× is the total number of RBC units transfused during the 24-week period before the randomization date for participants randomized and not dosed or within 168 days before the start of study treatment for participants randomized and dosed.
Outcome measures
| Measure |
Mitapivat
n=155 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
n=83 Participants
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Percent Change From Baseline in Transfused RBC Units
|
10.96 percent change
Standard Error 2.149
|
3.32 percent change
Standard Error 2.846
|
SECONDARY outcome
Timeframe: Double-blind Period: Baseline through Week 48Population: FAS included all participants who were randomized.
Transfusion-independence is defined as transfusion-free for ≥8 consecutive weeks through Week 48 in the double-blind period.
Outcome measures
| Measure |
Mitapivat
n=171 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
n=87 Participants
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Percentage of Participants Who Achieved Transfusion-Independence
|
9.9 percentage of participants
|
1.1 percentage of participants
|
SECONDARY outcome
Timeframe: Double-blind Period: Baseline through Week 48Population: FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure.
Iron metabolism was assessed based on Iron levels.
Outcome measures
| Measure |
Mitapivat
n=149 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
n=75 Participants
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Change From Baseline in Iron Levels
|
-4.57 micromoles per liter (µmol/L)
Standard Error 0.949
|
2.06 micromoles per liter (µmol/L)
Standard Error 1.280
|
SECONDARY outcome
Timeframe: Double-blind Period: Baseline through Week 48Population: FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure.
Iron metabolism was assessed based on serum ferritin levels.
Outcome measures
| Measure |
Mitapivat
n=148 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
n=75 Participants
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Change From Baseline in Serum Ferritin Levels
|
45.3 micrograms per liter (µg/L)
Standard Error 88.90
|
76.3 micrograms per liter (µg/L)
Standard Error 119.84
|
SECONDARY outcome
Timeframe: Double-blind Period: Baseline through Week 48Population: FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure.
Iron metabolism was assessed based on total iron binding capacity.
Outcome measures
| Measure |
Mitapivat
n=80 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
n=41 Participants
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Change From Baseline in Total Iron Binding Capacity
|
-21.80 µmol/L
Standard Error 3.597
|
-1.23 µmol/L
Standard Error 5.053
|
SECONDARY outcome
Timeframe: Double-blind Period: Baseline through Week 48Population: FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure.
Iron metabolism was assessed based on TSAT levels. Transferrin saturation is reported by dividing value of serum iron by total iron binding capacity.
Outcome measures
| Measure |
Mitapivat
n=80 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
n=40 Participants
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Change From Baseline in Transferrin Saturation (TSAT) Levels
|
0.079 Ratio
Standard Error 0.0261
|
-0.013 Ratio
Standard Error 0.0371
|
SECONDARY outcome
Timeframe: Double-blind Period: From first dose of study drug up to week 48Population: Safety Analysis Set (SAS) included all participants who had received at least 1 dose of study treatment. Participants were classified according to the treatment received. If a participant was randomized to placebo and received at least 1 dose of mitapivat in the double-blind period, then the participant was classified to the mitapivat arm. One subject, randomized to placebo, received mitapivat and was classified in the mitapivat arm in the safety analysis set.
An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have a causal relationship with the study drug. A serious adverse event (SAE) is any untoward medical occurrence that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. TEAEs are AEs with an initial onset date during the on-treatment period or worsening from baseline and includes both serious \& non-serious TEAEs. Severity of AEs was evaluated using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE; Version 4.03): grade 1: mild; grade 2: moderate; grade 3: severe or medically significant but not immediately life-threatening; grade 4: life threatening or disabling; grade 5: death related to AE.
Outcome measures
| Measure |
Mitapivat
n=172 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
n=85 Participants
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3
Participants with any TEAE of Grade ≥ 3
|
32 Participants
|
12 Participants
|
|
Double-blind Period: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3
Participants with TEAEs
|
155 Participants
|
71 Participants
|
|
Double-blind Period: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3
Participants with serious TEAEs
|
19 Participants
|
13 Participants
|
|
Double-blind Period: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3
Participants with TEAEs related to study drug
|
65 Participants
|
16 Participants
|
SECONDARY outcome
Timeframe: Open-Label Extension Period: From Week 48 up to end of study (approximately 5 years)An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have a causal relationship with the study drug. A serious adverse event (SAE) is any untoward medical occurrence that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. TEAEs are AEs with an initial onset date during the on-treatment period or worsening from baseline and includes both serious \& non-serious TEAEs. Severity of abnormalities was evaluated using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE; Version 4.03): grade 1: mild; grade 2: moderate; grade 3: severe or medically significant but not immediately life-threatening; grade 4: life threatening or disabling; grade 5: death related to AE.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Double-blind Period: Pre-dose at Week 12; pre-dose at Week 24; pre-dose, 0.5, 1, 3, 5, 7 hours post-dose at Week 36Population: Pharmacokinetic (PK) Analysis Set included a subset of the safety analysis set and included all participants with at least 1 mitapivat plasma concentration measurement ≥ lower limit of quantification (LLQ). Number analyzed signifies those participants who were evaluable at specified time point.
Outcome measures
| Measure |
Mitapivat
n=165 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Plasma Concentrations of Mitapivat
Pre-dose at Week 12
|
122.90 nanograms per milliliter (ng/mL)
Standard Deviation 252.666
|
—
|
|
Double-blind Period: Plasma Concentrations of Mitapivat
Pre-dose at Week 24
|
96.55 nanograms per milliliter (ng/mL)
Standard Deviation 184.835
|
—
|
|
Double-blind Period: Plasma Concentrations of Mitapivat
Pre-dose at Week 36
|
129.31 nanograms per milliliter (ng/mL)
Standard Deviation 260.610
|
—
|
|
Double-blind Period: Plasma Concentrations of Mitapivat
0.5 Hour Post-dose at Week 36
|
1282.21 nanograms per milliliter (ng/mL)
Standard Deviation 1007.068
|
—
|
|
Double-blind Period: Plasma Concentrations of Mitapivat
1 Hour Post-dose at Week 36
|
1468.69 nanograms per milliliter (ng/mL)
Standard Deviation 713.248
|
—
|
|
Double-blind Period: Plasma Concentrations of Mitapivat
3 Hours Post-dose at Week 36
|
765.32 nanograms per milliliter (ng/mL)
Standard Deviation 372.719
|
—
|
|
Double-blind Period: Plasma Concentrations of Mitapivat
5 Hours Post-dose at Week 36
|
371.11 nanograms per milliliter (ng/mL)
Standard Deviation 232.556
|
—
|
|
Double-blind Period: Plasma Concentrations of Mitapivat
7 Hours Post-dose at Week 36
|
221.78 nanograms per milliliter (ng/mL)
Standard Deviation 172.845
|
—
|
SECONDARY outcome
Timeframe: Double-blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose at Week 36Population: PK analysis set is a subset of the safety analysis set and included all participants with at least 1 plasma mitapivat concentration measurement ≥LLQ. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure.
Area under the concentration-time curve from time zero to Tlast on dosing day, calculated using the linear-log trapezoidal rule.
Outcome measures
| Measure |
Mitapivat
n=141 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Area Under the Plasma Concentration-time Curve From Time Zero to the Last Measurable Concentration (AUC0-last) of Mitapivat
|
4365.84 hours*nanograms per milliliter (h*ng/mL)
Geometric Coefficient of Variation 35.9
|
—
|
SECONDARY outcome
Timeframe: Double-blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose Week 36Population: PK analysis set is a subset of the safety analysis set and included all participants with at least 1 plasma mitapivat concentration measurement ≥LLQ. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure.
Outcome measures
| Measure |
Mitapivat
n=149 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Maximum Observed Plasma Concentration (Cmax) of Mitapivat
|
1603.00 nanograms per milliliter (ng/mL)
Geometric Coefficient of Variation 45.6
|
—
|
SECONDARY outcome
Timeframe: Double-blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose Week 36Population: PK analysis set is a subset of the safety analysis set and included all participants with at least 1 plasma mitapivat concentration measurement ≥LLQ. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure.
Outcome measures
| Measure |
Mitapivat
n=149 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Time to Reach of Maximum Observed Plasma Concentration (Tmax) of Mitapivat
|
1.000 Hours
Interval 0.42 to 5.0
|
—
|
SECONDARY outcome
Timeframe: Double-blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose Week 36Population: PK analysis set is a subset of the safety analysis set and included all participants with at least 1 plasma mitapivat concentration measurement ≥LLQ. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure.
Outcome measures
| Measure |
Mitapivat
n=147 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Time of Last Quantifiable Concentration (Tlast) of Mitapivat
|
6.750 Hours
Interval 4.83 to 7.17
|
—
|
SECONDARY outcome
Timeframe: Double-blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose Week 36Population: PK analysis set is a subset of the safety analysis set and included all participants with at least 1 plasma mitapivat concentration measurement ≥LLQ. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure.
Clast is the last quantifiable concentration after a single dose or within the dosing interval (tau) for multiple doses.
Outcome measures
| Measure |
Mitapivat
n=147 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Last Quantifiable Plasma Concentration (Clast) of Mitapivat
|
175.76 ng/mL
Geometric Coefficient of Variation 85.1
|
—
|
SECONDARY outcome
Timeframe: Double-blind Period: Pre-dose at Day 1; pre-dose at Week 12; pre-dose at Week 24; pre-dose, 0.5, 1, 3, 5, 7 hours post-dose at Week 36Population: Pharmacodynamic (PD) analysis set is a subset of the safety analysis set and included all participants with at least 1 blood 2,3-diphosphoglycerate (DPG) or ATP concentration measurement ≥ LLQ. Number analyzed signifies those participants who were evaluable at specified time point.
Outcome measures
| Measure |
Mitapivat
n=169 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
n=85 Participants
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Blood Concentration of Adenosine Triphosphate (ATP)
1 Hour Post-dose at Week 36
|
277.45 µg/mL
Standard Deviation 58.668
|
209.92 µg/mL
Standard Deviation 38.597
|
|
Double-blind Period: Blood Concentration of Adenosine Triphosphate (ATP)
3 Hours Post-dose at Week 36
|
281.70 µg/mL
Standard Deviation 61.262
|
213.63 µg/mL
Standard Deviation 43.949
|
|
Double-blind Period: Blood Concentration of Adenosine Triphosphate (ATP)
Pre-dose at Day 1
|
224.33 µg/mL
Standard Deviation 40.031
|
236.23 µg/mL
Standard Deviation 47.368
|
|
Double-blind Period: Blood Concentration of Adenosine Triphosphate (ATP)
Pre-dose at Week 12
|
283.39 µg/mL
Standard Deviation 59.217
|
204.19 µg/mL
Standard Deviation 33.207
|
|
Double-blind Period: Blood Concentration of Adenosine Triphosphate (ATP)
Pre-dose at Week 24
|
279.16 µg/mL
Standard Deviation 53.652
|
200.84 µg/mL
Standard Deviation 30.333
|
|
Double-blind Period: Blood Concentration of Adenosine Triphosphate (ATP)
Pre-dose at Week 36
|
280.72 µg/mL
Standard Deviation 61.834
|
210.47 µg/mL
Standard Deviation 43.330
|
|
Double-blind Period: Blood Concentration of Adenosine Triphosphate (ATP)
0.5 Hour Post-dose at Week 36
|
274.75 µg/mL
Standard Deviation 63.513
|
206.83 µg/mL
Standard Deviation 39.389
|
|
Double-blind Period: Blood Concentration of Adenosine Triphosphate (ATP)
5 Hours Post-dose at Week 36
|
283.76 µg/mL
Standard Deviation 61.216
|
208.18 µg/mL
Standard Deviation 40.965
|
|
Double-blind Period: Blood Concentration of Adenosine Triphosphate (ATP)
7 Hours Post-dose at Week 36
|
281.47 µg/mL
Standard Deviation 57.163
|
212.02 µg/mL
Standard Deviation 45.767
|
SECONDARY outcome
Timeframe: Double-blind Period: Pre-dose at Day 1; pre-dose at Week 12; pre-dose at Week 24; pre-dose, 0.5, 1, 3, 5, 7 hours post-dose at Week 36Population: PD analysis set is a subset of the safety analysis set and included all participants with at least 1 blood 2,3-diphosphoglycerate (2,3-DPG) or adenosine triphosphate (ATP) concentration measurement ≥LLQ. Number analyzed signifies those participants who were evaluable at specified time point.
Outcome measures
| Measure |
Mitapivat
n=169 Participants
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
n=85 Participants
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Double-blind Period: Blood Concentration of 2,3 - Diphosphoglycerate (2,3-DPG)
Pre-dose at Day 1
|
426.67 µg/mL
Standard Deviation 75.055
|
417.13 µg/mL
Standard Deviation 87.448
|
|
Double-blind Period: Blood Concentration of 2,3 - Diphosphoglycerate (2,3-DPG)
Pre-dose at Week 12
|
274.25 µg/mL
Standard Deviation 76.114
|
365.88 µg/mL
Standard Deviation 67.238
|
|
Double-blind Period: Blood Concentration of 2,3 - Diphosphoglycerate (2,3-DPG)
Pre-dose at Week 24
|
275.60 µg/mL
Standard Deviation 78.307
|
355.57 µg/mL
Standard Deviation 59.420
|
|
Double-blind Period: Blood Concentration of 2,3 - Diphosphoglycerate (2,3-DPG)
Pre-dose at Week 36
|
289.11 µg/mL
Standard Deviation 78.658
|
368.81 µg/mL
Standard Deviation 71.481
|
|
Double-blind Period: Blood Concentration of 2,3 - Diphosphoglycerate (2,3-DPG)
0.5 Hour Post-dose at Week 36
|
286.06 µg/mL
Standard Deviation 73.409
|
367.74 µg/mL
Standard Deviation 62.673
|
|
Double-blind Period: Blood Concentration of 2,3 - Diphosphoglycerate (2,3-DPG)
1 Hour Post-dose at Week 36
|
281.73 µg/mL
Standard Deviation 80.276
|
368.67 µg/mL
Standard Deviation 66.037
|
|
Double-blind Period: Blood Concentration of 2,3 - Diphosphoglycerate (2,3-DPG)
5 Hours Post-dose at Week 36
|
272.73 µg/mL
Standard Deviation 65.327
|
359.09 µg/mL
Standard Deviation 72.144
|
|
Double-blind Period: Blood Concentration of 2,3 - Diphosphoglycerate (2,3-DPG)
7 Hours Post-dose at Week 36
|
268.17 µg/mL
Standard Deviation 65.942
|
369.19 µg/mL
Standard Deviation 75.546
|
|
Double-blind Period: Blood Concentration of 2,3 - Diphosphoglycerate (2,3-DPG)
3 Hours Post-dose at Week 36
|
280.93 µg/mL
Standard Deviation 76.916
|
383.00 µg/mL
Standard Deviation 91.727
|
Adverse Events
Mitapivat
Serious events: 19 serious events
Other events: 122 other events
Deaths: 0 deaths
Placebo
Serious events: 13 serious events
Other events: 50 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Mitapivat
n=172 participants at risk
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
n=85 participants at risk
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Infections and infestations
Pneumonia
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
2.4%
2/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Infections and infestations
Viral infection
|
0.00%
0/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
1.2%
1/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Blood and lymphatic system disorders
Splenic haematoma
|
0.00%
0/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
1.2%
1/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Blood and lymphatic system disorders
Hypersplenism
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Blood and lymphatic system disorders
Mesenteric lymphadenitis
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Blood and lymphatic system disorders
Pancytopenia
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Infections and infestations
Gastroenteritis
|
1.2%
2/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Infections and infestations
COVID-19 pneumonia
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Infections and infestations
Cellulitis
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Infections and infestations
Dengue fever
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Infections and infestations
Influenza
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Infections and infestations
Lower respiratory tract infection
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Hepatobiliary disorders
Cholecystitis
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
1.2%
1/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Hepatobiliary disorders
Cholecystitis acute
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
1.2%
1/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Hepatobiliary disorders
Cholangitis acute
|
0.00%
0/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
1.2%
1/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Cardiac disorders
Arrhythmia supraventricular
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Cardiac disorders
Supraventricular tachycardia
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Cardiac disorders
Arrhythmia
|
0.00%
0/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
1.2%
1/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Cardiac disorders
Left ventricular dysfunction
|
0.00%
0/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
1.2%
1/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Injury, poisoning and procedural complications
Radius fracture
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
0.00%
0/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
1.2%
1/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Gastrointestinal disorders
Proctitis
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
General disorders
Asthenia
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Hepatic cancer
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Nervous system disorders
Dizziness
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Renal and urinary disorders
Renal mass
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Reproductive system and breast disorders
Ovarian cyst ruptured
|
0.58%
1/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Eye disorders
Cataract
|
0.00%
0/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
1.2%
1/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Investigations
Blood creatine phosphokinase increased
|
0.00%
0/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
1.2%
1/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Musculoskeletal and connective tissue disorders
Limb deformity
|
0.00%
0/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
1.2%
1/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Pregnancy, puerperium and perinatal conditions
Abortion spontaneous
|
0.00%
0/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
1.2%
1/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
0.00%
0/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
1.2%
1/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
Other adverse events
| Measure |
Mitapivat
n=172 participants at risk
Participants randomized to receive mitapivat 100 mg, orally, BID for 48 weeks in the double-blind period.
|
Placebo
n=85 participants at risk
Participants randomized to receive placebo matching mitapivat, orally, BID for 48 weeks in the double-blind period.
|
|---|---|---|
|
Infections and infestations
COVID-19
|
4.7%
8/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
9.4%
8/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Infections and infestations
Upper respiratory tract infection
|
15.7%
27/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
16.5%
14/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Infections and infestations
Influenza
|
8.1%
14/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
4.7%
4/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Infections and infestations
Nasopharyngitis
|
6.4%
11/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
4.7%
4/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Infections and infestations
Urinary tract infection
|
6.4%
11/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
0.00%
0/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Nervous system disorders
Headache
|
26.7%
46/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
11.8%
10/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
General disorders
Fatigue
|
10.5%
18/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
2.4%
2/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
General disorders
Pyrexia
|
9.9%
17/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
7.1%
6/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
General disorders
Asthenia
|
5.8%
10/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
4.7%
4/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
General disorders
Influenza like illness
|
5.2%
9/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
2.4%
2/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Gastrointestinal disorders
Diarrhoea
|
11.0%
19/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
8.2%
7/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Gastrointestinal disorders
Nausea
|
7.6%
13/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
5.9%
5/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Gastrointestinal disorders
Vomiting
|
4.7%
8/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
8.2%
7/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Psychiatric disorders
Initial insomnia
|
14.0%
24/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
4.7%
4/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Psychiatric disorders
Middle insomnia
|
5.8%
10/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
3.5%
3/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
7.6%
13/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
5.9%
5/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.8%
10/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
4.7%
4/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
3.5%
6/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
5.9%
5/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
8.7%
15/172 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
3.5%
3/85 • Double-blind period: From first dose of study drug up to week 48
AEs: Safety Analysis Set (SAS) was used. One participant, randomized to placebo, received mitapivat and is classified in the mitapivat arm in the SAS. All-Cause Mortality: FAS was used. Safety data are presented for Double-blind Period only. The OLE period is ongoing with anticipated completion in June 2029. Results for OLE will be reported separately by June 2030.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place