Trial · NCT04770779
Outcomes20
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
1 to 20 of 20
Download this page as JSON| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Double-blind Period: Percentage of Participants Who Achieved Transfusion Reduction Response (TRR) | Double-blind Period: Baseline through Week 48 | FAS included all participants who were randomized. | percentage of participants | NUMBER |
| SECONDARY | Double-blind Period: Percentage of Participants Who Achieved TRR2 | Double-blind period: Baseline through Week 48 | FAS included all participants who were randomized. | percentage of participants | NUMBER |
| SECONDARY | Double-blind Period: Percentage of Participants Who Achieved TRR3 | Double-blind period: Baseline up to Week 13 through Week 48 | FAS included all participants who were randomized. | percentage of participants | NUMBER |
| SECONDARY | Double-blind Period: Percentage of Participants Who Achieved TRR4 | Double-blind period: Baseline up to Week 13 through Week 48 | FAS included all participants who were randomized. | percentage of participants | NUMBER |
| SECONDARY | Double-blind Period: Percent Change From Baseline in Transfused RBC Units | Double-blind Period: Baseline, Week 13 through Week 48 | FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Double-blind Period: Percentage of Participants Who Achieved Transfusion-Independence | Double-blind Period: Baseline through Week 48 | FAS included all participants who were randomized. | percentage of participants | NUMBER |
| SECONDARY | Double-blind Period: Change From Baseline in Iron Levels | Double-blind Period: Baseline through Week 48 | FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure. | micromoles per liter (µmol/L) | LEAST_SQUARES_MEAN |
| SECONDARY | Double-blind Period: Change From Baseline in Serum Ferritin Levels | Double-blind Period: Baseline through Week 48 | FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure. | micrograms per liter (µg/L) | LEAST_SQUARES_MEAN |
| SECONDARY | Double-blind Period: Change From Baseline in Total Iron Binding Capacity | Double-blind Period: Baseline through Week 48 | FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure. | µmol/L | LEAST_SQUARES_MEAN |
| SECONDARY | Double-blind Period: Change From Baseline in Transferrin Saturation (TSAT) Levels | Double-blind Period: Baseline through Week 48 | FAS included all participants who were randomized. Overall number of participants analyzed indicates number of participants who were evaluable for this outcome measure. | Ratio | LEAST_SQUARES_MEAN |
| 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 | 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. | Participants | COUNT_OF_PARTICIPANTS |
| 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) | |||
| 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 | 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. | nanograms per milliliter (ng/mL) | MEAN |
| 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 | 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. | hours*nanograms per milliliter (h*ng/mL) | GEOMETRIC_MEAN |
| 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 | 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. | nanograms per milliliter (ng/mL) | GEOMETRIC_MEAN |
| 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 | 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. | Hours | MEDIAN |
| 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 | 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. | Hours | MEDIAN |
| 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 | 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. | ng/mL | GEOMETRIC_MEAN |
| 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 | 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. | µg/mL | MEAN |
| 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 | 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. | µg/mL | MEAN |
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.
For software
Read this list from software.
Twenty rows a page, as JSON. No key.
GET https://conductscience.org/api/trials/v1/NCT04770779/outcomes