Trial · NCT03559699
A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
NCT03559699, Phase 3, Completed, Agios Pharmaceuticals, Inc.; results first posted 2021-12-10
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD). Trials, record NCT03559699. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03559699
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
- NCT number
- NCT03559699
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 27 participants, actual
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Conditions
- Pyruvate Kinase Deficiency, Anemia, Hemolytic
- Interventions
- AG-348
- First posted
- 2018-06-18
- Last updated
- 2022-01-04
- Other registries' IDs
- eudract:2017-003803-22
- Results first posted
- 2021-12-10
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Achieving a Reduction in Transfusion Burden in Part 2 | From Part 2, Day 1 to Part 2 Week 24 | Reduction in transfusion burden is defined as a ≥33% reduction in the number of RBC units transfused during the Fixed Dose Period standardized to 24 weeks compared with the historical transfusion burden standardized to 24 weeks (Standardized Control Period). The on-study (Fixed Dose Period) transfusion burden was calculated as the total number of transfused RBC units received in the Fixed Dose Period standardized to 24 weeks. |
| Secondary | Annualized Number of RBC Units Transfused During the Study | Part 1 Day 1 to Part 2 Week 24 | The annualized total number of RBC units transfused during the entire study (both Part 1 and Part 2) is reported. It was calculated as the total number of RBC units transfused up to the end of Fixed Dose Period divided by the total number of days from the first dose date until the end date of Fixed Dose Period × 52. |
| Secondary | Number of Transfusion Episodes in Part 2 | From Part 2 Day 1 to Part 2 Week 24 | This is the number of transfusion episodes in Part 2. The number of transfusion episodes were standardized to 24 weeks. Transfusions received over up to 3 consecutive days were counted as 1 episode. |
| Secondary | Percentage of Transfusion-Free Participants in Part 2 | From Part 2 Day 1 to Part 2 Week 24 | Transfusion-free responders were the participants who were transfusion-free in Part 2. |
| Secondary | Percentage of Participants Achieving Normal Hemoglobin (Hb) Concentrations in Part 2 | From Part 2 Day 1 to Part 2 Week 24 | This is the percentage of participants who achieved hemoglobin (Hb) concentrations in the normal range at least once, 8 weeks or more after a transfusion in Part 2. |
| Secondary | Percentage of Participants With Adverse Events | Through 4 weeks after last dose (approximately Part 2, Week 31) | An 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. An AE can, therefore, be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. |
| Other | Percentage of Participants Experiencing an Adverse Event of Special Interest (AESI) | From Part 1 Day 1 to end of Part 2, including follow-up (Day 197) | An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An AESI can be serious or non-serious. |
| Other | Bone Mineral Density Z-Score | Part 1, Day 1, Part 2, Day 1 and Part 2, Week 24 | |
| Other | Bone Mineral Density T-Score | Part 1, Day 1, Part 2, Day 1 and Part 2, Week 24 |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants Achieving a Reduction in Transfusion Burden in Part 2 | From Part 2, Day 1 to Part 2 Week 24 | Full analysis set included all participants who received at least 1 dose of study drug. | percentage of participants | NUMBER |
| SECONDARY | Annualized Number of RBC Units Transfused During the Study | Part 1 Day 1 to Part 2 Week 24 | Full analysis set included all participants who received at least 1 dose of study drug. | RBC units | MEAN |
| SECONDARY | Number of Transfusion Episodes in Part 2 | From Part 2 Day 1 to Part 2 Week 24 | Full analysis set included all participants who received at least 1 dose of study drug. Number analyzed is the number of participants evaluated for the outcome measure. | transfusion episodes | MEAN |
| SECONDARY | Percentage of Transfusion-Free Participants in Part 2 | From Part 2 Day 1 to Part 2 Week 24 | Full analysis set included all participants who received at least 1 dose of study drug. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Achieving Normal Hemoglobin (Hb) Concentrations in Part 2 | From Part 2 Day 1 to Part 2 Week 24 | Full analysis set included all participants who received at least 1 dose of study drug. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Adverse Events | Through 4 weeks after last dose (approximately Part 2, Week 31) | Safety analysis set included all participants who received at least 1 dose of study drug. | percentage of participants | NUMBER |
| OTHER_PRE_SPECIFIED | Percentage of Participants Experiencing an Adverse Event of Special Interest (AESI) | From Part 1 Day 1 to end of Part 2, including follow-up (Day 197) | |||
| OTHER_PRE_SPECIFIED | Bone Mineral Density Z-Score | Part 1, Day 1, Part 2, Day 1 and Part 2, Week 24 | |||
| OTHER_PRE_SPECIFIED | Bone Mineral Density T-Score | Part 1, Day 1, Part 2, Day 1 and Part 2, Week 24 |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants Achieving a Reduction in Transfusion Burden in Part 2 | AG-348 | 37 | 95% Confidence Interval 19.4 to 57.6 | percentage of participants | |
| Annualized Number of RBC Units Transfused During the Study | AG-348 | 11.52 | Standard Deviation 10.543 | RBC units | |
| Number of Transfusion Episodes in Part 2 | AG-348 | 2.88 | Standard Deviation 2.694 | transfusion episodes | |
| Percentage of Transfusion-Free Participants in Part 2 | AG-348 | 22.2 | 95% Confidence Interval 8.6 to 42.3 | percentage of participants | |
| Percentage of Participants Achieving Normal Hemoglobin (Hb) Concentrations in Part 2 | AG-348 | 11.1 | 95% Confidence Interval 2.4 to 29.2 | percentage of participants | |
| Percentage of Participants With Adverse Events | AG-348 | 100 | percentage of participants |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | AG-348 | 27 | |
| Overall Study | COMPLETED | AG-348 | 20 | |
| Overall Study | NOT COMPLETED | AG-348 | 7 |
Source ClinicalTrials.gov
Adverse events29
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 | Nasopharyngitis | Infections and infestations | AG-348 | 4 | 27 | |
| other | Upper respiratory tract infection | Infections and infestations | AG-348 | 4 | 27 | |
| other | Viral upper respiratory tract infection | Infections and infestations | AG-348 | 4 | 27 | |
| other | Influenza | Infections and infestations | AG-348 | 2 | 27 | |
| other | Pharyngitis | Infections and infestations | AG-348 | 2 | 27 | |
| other | Suspected COVID-19 | Infections and infestations | AG-348 | 2 | 27 | |
| other | Nausea | Gastrointestinal disorders | AG-348 | 5 | 27 | |
| other | Vomiting | Gastrointestinal disorders | AG-348 | 4 | 27 | |
| other | Abdominal pain | Gastrointestinal disorders | AG-348 | 3 | 27 | |
| other | Diarrhoea | Gastrointestinal disorders | AG-348 | 3 | 27 |
1 to 10 of 29Source ClinicalTrials.gov
See all 29 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | AG-348 | 0 | 27 |
| Total, other adverse events | AG-348 | 27 | 27 |
| Total, serious adverse events | AG-348 | 3 | 27 |
Source ClinicalTrials.gov
Papers3
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/NCT03559699
GET https://conductscience.org/api/trials/v1/NCT03559699/events?page=2