Trial · NCT04770779
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 20
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Double-blind Period: Percentage of Participants Who Achieved Transfusion Reduction Response (TRR) | Double-blind Period: Baseline through Week 48 | 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 Participants withdrawn from the study before Week 12 (Day 85) were considered nonresponders. |
| Secondary | Double-blind Period: Percentage of Participants Who Achieved TRR2 | Double-blind period: Baseline through Week 48 | 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 Participants withdrawn from the study before Week 24 (Day 169) were considered nonresponders. |
| Secondary | Double-blind Period: Percentage of Participants Who Achieved TRR3 | Double-blind period: Baseline up to Week 13 through Week 48 | 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 Participants withdrawn from the study before Week 48 were considered nonresponders. |
| Secondary | Double-blind Period: Percentage of Participants Who Achieved TRR4 | Double-blind period: Baseline up to Week 13 through Week 48 | 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. |
| Secondary | Double-blind Period: Percent Change From Baseline in Transfused RBC Units | Double-blind Period: Baseline, Week 13 through Week 48 | 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. |
| Secondary | Double-blind Period: Percentage of Participants Who Achieved Transfusion-Independence | Double-blind Period: Baseline through Week 48 | Transfusion-independence is defined as transfusion-free for ≥8 consecutive weeks through Week 48 in the double-blind period. |
| Secondary | Double-blind Period: Change From Baseline in Iron Levels | Double-blind Period: Baseline through Week 48 | Iron metabolism was assessed based on Iron levels. |
| Secondary | Double-blind Period: Change From Baseline in Serum Ferritin Levels | Double-blind Period: Baseline through Week 48 | Iron metabolism was assessed based on serum ferritin levels. |
| Secondary | Double-blind Period: Change From Baseline in Total Iron Binding Capacity | Double-blind Period: Baseline through Week 48 | Iron metabolism was assessed based on total iron binding capacity. |
| Secondary | Double-blind Period: Change From Baseline in Transferrin Saturation (TSAT) Levels | Double-blind Period: Baseline through Week 48 | Iron metabolism was assessed based on TSAT levels. Transferrin saturation is reported by dividing value of serum iron by total iron binding capacity. |
| Secondary | 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 | Double-blind Period: From first dose of study drug up to week 48 | 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. |
| Secondary | Open-label Extension Period: Number of Participants With TEAEs, Serious TEAEs, Related TEAEs, Related TEAEs and TEAEs With Severity of Greater Than or Equal to 3 | 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. |
| Secondary | Double-blind Period: Plasma Concentrations of Mitapivat | 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 36 | |
| Secondary | Double-blind Period: Area Under the Plasma Concentration-time Curve From Time Zero to the Last Measurable Concentration (AUC0-last) of Mitapivat | Double-blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose at Week 36 | Area under the concentration-time curve from time zero to Tlast on dosing day, calculated using the linear-log trapezoidal rule. |
| Secondary | Double-blind Period: Maximum Observed Plasma Concentration (Cmax) of Mitapivat | Double-blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose Week 36 | |
| Secondary | Double-blind Period: Time to Reach of Maximum Observed Plasma Concentration (Tmax) of Mitapivat | Double-blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose Week 36 | |
| Secondary | Double-blind Period: Time of Last Quantifiable Concentration (Tlast) of Mitapivat | Double-blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose Week 36 | |
| Secondary | Double-blind Period: Last Quantifiable Plasma Concentration (Clast) of Mitapivat | Double-blind Period: Pre-dose, 0.5, 1, 3, 5, 7 hours post-dose Week 36 | Clast is the last quantifiable concentration after a single dose or within the dosing interval (tau) for multiple doses. |
| Secondary | Double-blind Period: Blood Concentration of Adenosine Triphosphate (ATP) | 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 36 | |
| Secondary | Double-blind Period: Blood Concentration of 2,3 - Diphosphoglycerate (2,3-DPG) | 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 36 |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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