Trial · NCT03853798
Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007
NCT03853798, Phase 3, Completed, Agios Pharmaceuticals, Inc.; results first posted 2025-11-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007. Trials, record NCT03853798. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03853798
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007
- NCT number
- NCT03853798
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 90 participants, actual
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Conditions
- Pyruvate Kinase Deficiency
- Interventions
- Mitapivat
- First posted
- 2019-02-26
- Last updated
- 2025-11-18
- Other registries' IDs
- eudract:2018-003459-39
- Results first posted
- 2025-11-18
Source ClinicalTrials.gov
Registered outcomes28
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | All Cohorts: Number of Participants With at Least One Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3 | Up to 197 weeks | A clinical adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the study drug. TEAEs are AEs with an initial onset date during the on-treatment period or worsening from baseline and include both serious and 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. |
| Primary | All Cohorts: Number of Participants With TEAEs Leading to Dose Reduction, Treatment Interruption and Treatment Discontinuation | Up to 197 weeks | A clinical AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the study drug. TEAEs are AEs with an initial onset date during the on-treatment period or worsening from baseline and include both serious and non-serious TEAEs. Number of participants with TEAEs leading to dose reduction, treatment interruption and treatment discontinuation are reported. |
| Primary | All Cohorts: Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters Reported as Grade Greater Than or Equal to (≥)3 TEAEs | Up to 197 weeks | Clinical laboratory assessments including hematology, clinical chemistry, triglycerides, urate, coagulation, urinalysis, and liver function tests were performed in the study. Clinically significant treatment-emergent laboratory abnormalities were graded according to the 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. Clinical significance was determined based on the judgment of the Investigator. |
| Primary | All Cohorts: Number of Participants With Clinically Significant Abnormalities in Vital Signs Measurements and Physical Examinations Reported as TEAEs | Up to 197 weeks | Vital signs and physical examinations including height, weight, body mass index (BMI), systolic blood pressure, diastolic blood pressure, pulse rate, temperature were assessed. Number of participants who experienced clinically significant abnormalities in vital signs and physical examinations as TEAEs were reported. Clinical significance was determined based on the judgment of the Investigator. |
| Primary | All Cohorts: Number of Participants With Clinically Significant Abnormalities in Bone Mineral Density (BMD) | Up to 192 weeks | BMD was measured by dual-energy X-ray absorptiometry (DXA) scans during the study. Number of participants with clinically significant abnormalities were reported. Clinical significance was determined based on the judgment of the Investigator. |
| Primary | All Cohorts: Change From Baseline in Adjusted Spine T-score | Baseline, Week 192 | T-score of adjusted spine were assessed by DXA scans. The T-score is the number of standard deviations that bone density is above or below the average. A score of ≥ -1 indicates normal bone density; score between \< -1 and \> -2.5 indicates a sign of osteopenia (bone density below normal), and score of ≤ -2.5 indicates a sign of osteoporosis. |
| Primary | All Cohorts: Change From Baseline in Adjusted Spine Z-score | Baseline, Week 192 | The Z-score is a statistical measure to describe whether a value was above or below the standard. A Z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. |
| Primary | All Cohorts: Change From Baseline in Femoral Total T-score | Baseline, Week 192 | T-score of femoral total were assessed by DXA scans. The T-score is the number of standard deviations that bone density is above or below the average. A score of ≥ -1 indicates normal bone density; score between \< -1 and \> -2.5 indicates a sign of osteopenia (bone density below normal), and score of ≤ -2.5 indicates a sign of osteoporosis. |
| Primary | All Cohorts: Change From Baseline in Femoral Total Z-score | Baseline, Week 192 | The Z-score is a statistical measure to describe whether a value was above or below the standard. A Z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. |
| Primary | All Cohorts: Number of Participants With Clinically Significant Abnormalities in Electrocardiogram (ECG) Parameters Reported as TEAEs | Up to 197 weeks | The following ECG parameters including RR, PR, heart rate-corrected QT interval using the Fridericia's formula (QRS), QT, and QTc were assessed during the study. Number of Participants with clinically significant abnormalities in ECG parameters as TEAEs were reported. Clinical significance was determined based on the judgment of the Investigator. |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 registered outcomesOutcomes28
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | All Cohorts: Number of Participants With at Least One Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3 | Up to 197 weeks | The safety analysis set included all participants who received at least 1 dose of study treatment. Due to limited number of participants with optimal dose of 5 mg or 20 mg in antecedent studies AG348-C-006 (NCT03548220) and AG348-C-007 (NCT03559699), it was pre-specified in the SAP to report data for this outcome measure (OM) by Cohort. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | All Cohorts: Number of Participants With TEAEs Leading to Dose Reduction, Treatment Interruption and Treatment Discontinuation | Up to 197 weeks | The safety analysis set included all participants who received at least 1 dose of study treatment. Due to limited number of participants with optimal dose of 5 mg or 20 mg in antecedent studies AG348-C-006 (NCT03548220) and AG348-C-007 (NCT03559699), it was pre-specified in the SAP to report data for this OM by Cohort. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | All Cohorts: Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters Reported as Grade Greater Than or Equal to (≥)3 TEAEs | Up to 197 weeks | The safety analysis set included all participants who received at least 1 dose of study treatment. Due to limited number of participants with optimal dose of 5 mg or 20 mg in antecedent studies AG348-C-006 (NCT03548220) and AG348-C-007 (NCT03559699), it was pre-specified in the SAP to report data for this OM by Cohort. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | All Cohorts: Number of Participants With Clinically Significant Abnormalities in Vital Signs Measurements and Physical Examinations Reported as TEAEs | Up to 197 weeks | The safety analysis set included all participants who received at least 1 dose of study treatment. Due to limited number of participants with optimal dose of 5 mg or 20 mg in antecedent studies AG348-C-006 (NCT03548220) and AG348-C-007 (NCT03559699), it was pre-specified in the SAP to report data for this OM by Cohort. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | All Cohorts: Number of Participants With Clinically Significant Abnormalities in Bone Mineral Density (BMD) | Up to 192 weeks | The safety analysis set included all participants who received at least 1 dose of study treatment. Due to limited number of participants with optimal dose of 5 mg or 20 mg in antecedent studies AG348-C-006 (NCT03548220) and AG348-C-007 (NCT03559699), it was pre-specified in the SAP to report data for this OM by Cohort. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | All Cohorts: Change From Baseline in Adjusted Spine T-score | Baseline, Week 192 | The safety analysis set included all participants who received at least 1 dose of study treatment. Here, overall number of participants analyzed indicates the number of participants who were evaluable for this OM. Due to limited number of participants with optimal dose of 5 mg or 20 mg in antecedent studies AG348-C-006 (NCT03548220) and AG348-C-007 (NCT03559699), it was pre-specified in the SAP to report data for this OM by Cohort. | T-score | MEAN |
| PRIMARY | All Cohorts: Change From Baseline in Adjusted Spine Z-score | Baseline, Week 192 | The safety analysis set included all participants who received at least 1 dose of study treatment. Here, overall number of participants analyzed indicates the number of participants who were evaluable for this OM. Due to limited number of participants with optimal dose of 5 mg or 20 mg in antecedent studies AG348-C-006 (NCT03548220) and AG348-C-007 (NCT03559699), it was pre-specified in the SAP to report data for this OM by Cohort. | Z-score | MEAN |
| PRIMARY | All Cohorts: Change From Baseline in Femoral Total T-score | Baseline, Week 192 | The safety analysis set included all participants who received at least 1 dose of study treatment. Here, overall number of participants analyzed indicates the number of participants who were evaluable for this OM. Due to limited number of participants with optimal dose of 5 mg or 20 mg in antecedent studies AG348-C-006 (NCT03548220) and AG348-C-007 (NCT03559699), it was pre-specified in the SAP to report data for this OM by Cohort. | T-score | MEAN |
| PRIMARY | All Cohorts: Change From Baseline in Femoral Total Z-score | Baseline, Week 192 | The safety analysis set included all participants who received at least 1 dose of study treatment. Here, overall number of participants analyzed indicates the number of participants who were evaluable for this OM. Due to limited number of participants with optimal dose of 5 mg or 20 mg in antecedent studies AG348-C-006 (NCT03548220) and AG348-C-007 (NCT03559699), it was pre-specified in the SAP to report data for this OM by Cohort. | Z- score | MEAN |
| PRIMARY | All Cohorts: Number of Participants With Clinically Significant Abnormalities in Electrocardiogram (ECG) Parameters Reported as TEAEs | Up to 197 weeks | The safety analysis set included all participants who received at least 1 dose of study treatment. Due to limited number of participants with optimal dose of 5 mg or 20 mg in antecedent studies AG348-C-006 (NCT03548220) and AG348-C-007 (NCT03559699), it was pre-specified in the SAP to report data for this OM by Cohort. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 outcomesResults by group101
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| All Cohorts: Number of Participants With at Least One Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3 | Cohort 1 | Participants with TEAEs | 37 | Participants | |
| All Cohorts: Number of Participants With at Least One Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3 | Cohort 2 | Participants with TEAEs | 33 | Participants | |
| All Cohorts: Number of Participants With at Least One Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3 | Cohort 3 | Participants with TEAEs | 15 | Participants | |
| All Cohorts: Number of Participants With at Least One Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3 | Cohort 1 | Participants with Serious TEAEs | 11 | Participants | |
| All Cohorts: Number of Participants With at Least One Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3 | Cohort 2 | Participants with Serious TEAEs | 8 | Participants | |
| All Cohorts: Number of Participants With at Least One Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3 | Cohort 3 | Participants with Serious TEAEs | 4 | Participants | |
| All Cohorts: Number of Participants With at Least One Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3 | Cohort 1 | Participants with TEAEs related to study drug | 22 | Participants | |
| All Cohorts: Number of Participants With at Least One Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3 | Cohort 2 | Participants with TEAEs related to study drug | 14 | Participants | |
| All Cohorts: Number of Participants With at Least One Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3 | Cohort 3 | Participants with TEAEs related to study drug | 4 | Participants | |
| All Cohorts: Number of Participants With at Least One Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3 | Cohort 1 | Participants with any TEAE of Grade ≥ 3 | 16 | Participants |
1 to 10 of 101Source ClinicalTrials.gov
See all 101 results by groupParticipant flow27
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1: 5 mg | 1 | |
| Overall Study | STARTED | Cohort 1: 20 mg | 1 | |
| Overall Study | STARTED | Cohort 1: 50 mg | 36 | |
| Overall Study | STARTED | Cohort 2: 5 mg | 2 | |
| Overall Study | STARTED | Cohort 2: 20 mg | 3 | |
| Overall Study | STARTED | Cohort 2: 50 mg | 30 | |
| Overall Study | STARTED | Cohort 3: 5 mg | 0 | |
| Overall Study | STARTED | Cohort 3: 20 mg | 1 | |
| Overall Study | STARTED | Cohort 3: 50 mg | 16 | |
| Overall Study | COMPLETED | Cohort 1: 5 mg | 0 |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 participant flowAdverse events1,692
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | COVID-19 | Infections and infestations | Cohort 1: 5 mg | 0 | 1 | |
| other | COVID-19 | Infections and infestations | Cohort 1: 20 mg | 0 | 1 | |
| other | COVID-19 | Infections and infestations | Cohort 1: 50 mg | 12 | 36 | |
| other | COVID-19 | Infections and infestations | Cohort 2: 5 mg | 1 | 2 | |
| other | COVID-19 | Infections and infestations | Cohort 2: 20 mg | 2 | 3 | |
| other | COVID-19 | Infections and infestations | Cohort 2: 50 mg | 13 | 30 | |
| other | COVID-19 | Infections and infestations | Cohort 3: 5 mg | 0 | 0 | |
| other | COVID-19 | Infections and infestations | Cohort 3: 20 mg | 1 | 1 | |
| other | COVID-19 | Infections and infestations | Cohort 3: 50 mg | 5 | 16 | |
| other | Nasopharyngitis | Infections and infestations | Cohort 1: 5 mg | 0 | 1 |
1 to 10 of 1,692Source ClinicalTrials.gov
See all 1,692 adverse eventsAdverse event totals27
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1: 5 mg | 0 | 1 |
| Total, all-cause mortality | Cohort 1: 20 mg | 0 | 1 |
| Total, all-cause mortality | Cohort 1: 50 mg | 0 | 36 |
| Total, all-cause mortality | Cohort 2: 5 mg | 0 | 2 |
| Total, all-cause mortality | Cohort 2: 20 mg | 0 | 3 |
| Total, all-cause mortality | Cohort 2: 50 mg | 1 | 30 |
| Total, all-cause mortality | Cohort 3: 5 mg | 0 | 0 |
| Total, all-cause mortality | Cohort 3: 20 mg | 0 | 1 |
| Total, all-cause mortality | Cohort 3: 50 mg | 0 | 16 |
| Total, other adverse events | Cohort 1: 5 mg | 0 | 1 |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 adverse event totalsPapers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
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 trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03853798
GET https://conductscience.org/api/trials/v1/NCT03853798/registered?page=2