Trial · NCT02476916

A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency

NCT02476916, Phase 2, Completed, Agios Pharmaceuticals, Inc.; results first posted 2020-06-11

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency. Trials, record NCT02476916. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02476916

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency
NCT number: NCT02476916
Status: Completed
Phases: Phase 2
Enrollment: 52 participants, actual
Lead sponsor: Agios Pharmaceuticals, Inc.
Conditions: Pyruvate Kinase Deficiency
Interventions: AG-348
First posted: 2015-06-22
Last updated: 2026-05-01
Other registries' IDs: eudract:2015-000484-13
Results first posted: 2020-06-11

Source ClinicalTrials.gov

Registered outcomes34

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Percentage of Participants Experiencing at Least One Adverse Event (AEs) in the Core Period	Up to Week 24	An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered study drug-related.
Secondary	Percentage of Participants Experiencing at Least One AE up to Month 102	Up to Month 102	An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered study drug-related. Safety data for cumulative period (Core period and Extension period) has been reported in this outcome measure.
Secondary	Change From Baseline in Hemoglobin (Hb) Value at Week 24	Baseline and Week 24	Change (absolute change) from baseline was calculated as post-baseline value - baseline value. Increased Hb values indicate improvement.
Secondary	Change From Baseline Hb Value up to Month 102	Baseline, Months 12, 36, 60, 84, and 102	Change (absolute change) from baseline was calculated as post-baseline value - baseline value. Increased Hb values indicate improvement.
Secondary	Change From Baseline in Hematocrit at Week 24	Baseline and Week 24	Change (absolute change) from baseline was calculated as post-baseline value - baseline value. Increased hematocrit values indicate improvement.
Secondary	Change From Baseline in Hematocrit up to Month 102	Baseline, Months 12, 36, 60, 84, and 102	Change (absolute change) from baseline was calculated as post-baseline value - baseline value. Increased hematocrit values indicate improvement.
Secondary	Change From Baseline in Reticulocyte Count at Week 24	Baseline and Week 24	Change (absolute change) from baseline was calculated as post-baseline value - baseline value. Decreased reticulocyte count values indicate improvement.
Secondary	Change From Baseline in Reticulocyte Count up to Month 102	Baseline, Months 12, 36, 60, 84, and 102	Change (absolute change) from baseline will be calculated as post-baseline value - baseline value. Decreased reticulocyte count values indicate improvement.
Secondary	Change From Baseline in Haptoglobin at Week 24	Baseline and Week 24	Change (absolute change) from baseline was calculated as post-baseline value - baseline value. Increased haptoglobin values indicate improvement.
Secondary	Change From Baseline in Haptoglobin up to Month 102	Baseline, Months 12, 36, 60, 84, and 102	Change (absolute change) from baseline was calculated as post-baseline value - baseline value. Increased haptoglobin values indicate improvement.

1 to 10 of 34Source ClinicalTrials.gov

See all 34 registered outcomes

Outcomes32

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Percentage of Participants Experiencing at Least One Adverse Event (AEs) in the Core Period	Up to Week 24	The Safety Analysis Set included all participants who had received at least one dose of study drug.	percentage of participants	NUMBER
SECONDARY	Percentage of Participants Experiencing at Least One AE Over the Duration of the Extension Period	Up to approximately 8.5 years
SECONDARY	Change From Baseline in Hemoglobin (Hb) Value at Week 24	Baseline and Week 24	The Efficacy Analysis Set included all participants who enrolled and received any study treatment for at least 3 weeks. Number analyzed is the number of participants with evaluable data at the given time-point.	grams per deciliter (g/dL)	MEAN
SECONDARY	Change From Baseline in Hb Value Over the Duration of the Extension Period	Up to approximately 8.5 years
SECONDARY	Change From Baseline in Hematocrit at Week 24	Baseline and Week 24	The Safety Analysis Set included all participants who had received at least one dose of study drug. Number analyzed is the number of participants with evaluable data at the given time-point.	volume per volume (V/V)	MEAN
SECONDARY	Change From Baseline in Hematocrit Over the Duration of the Extension Period	Up to approximately 8.5 years
SECONDARY	Change From Baseline in Reticulocyte Count at Week 24	Baseline and Week 24	The Safety Analysis Set included all participants who had received at least one dose of study drug. Number analyzed is the number of participants with evaluable data at the given time-point.	cells * 10^9/liter	MEAN
SECONDARY	Change From Baseline in Reticulocyte Count Over the Duration of the Extension Period	Up to approximately 8.5 years
SECONDARY	Change From Baseline in Haptoglobin at Week 24	Baseline and Week 24	The Safety Analysis Set included all participants who had received at least one dose of study drug. Number analyzed is the number of participants with evaluable data at the given time-point.	gram per liter (g/L)	MEAN
SECONDARY	Change From Baseline in Haptoglobin Over the Duration of the Extension Period	Up to approximately 8.5 years

1 to 10 of 32Source ClinicalTrials.gov

See all 32 outcomes

Results by group86

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Percentage of Participants Experiencing at Least One Adverse Event (AEs) in the Core Period	AG-348 50 mg BID		96.3		percentage of participants
Percentage of Participants Experiencing at Least One Adverse Event (AEs) in the Core Period	AG-348 300 mg BID		100.0		percentage of participants
Change From Baseline in Hemoglobin (Hb) Value at Week 24	AG-348 50 mg BID	Baseline	9.243	Standard Deviation 1.4757	grams per deciliter (g/dL)
Change From Baseline in Hemoglobin (Hb) Value at Week 24	AG-348 300 mg BID	Baseline	8.636	Standard Deviation 1.1664	grams per deciliter (g/dL)
Change From Baseline in Hemoglobin (Hb) Value at Week 24	AG-348 50 mg BID	Change at Week 24	1.205	Standard Deviation 1.4181	grams per deciliter (g/dL)
Change From Baseline in Hemoglobin (Hb) Value at Week 24	AG-348 300 mg BID	Change at Week 24	1.611	Standard Deviation 1.7058	grams per deciliter (g/dL)
Change From Baseline in Hematocrit at Week 24	AG-348 50 mg BID	Baseline	0.289	Standard Deviation 0.0470	volume per volume (V/V)
Change From Baseline in Hematocrit at Week 24	AG-348 300 mg BID	Baseline	0.268	Standard Deviation 0.0367	volume per volume (V/V)
Change From Baseline in Hematocrit at Week 24	AG-348 50 mg BID	Change at Week 24	0.033	Standard Deviation 0.0414	volume per volume (V/V)
Change From Baseline in Hematocrit at Week 24	AG-348 300 mg BID	Change at Week 24	0.045	Standard Deviation 0.0499	volume per volume (V/V)

1 to 10 of 86Source ClinicalTrials.gov

See all 86 results by group

Participant flow12

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Core Period	STARTED	AG-348 50 mg BID	27
Core Period	STARTED	AG-348 300 mg BID	25
Core Period	COMPLETED	AG-348 50 mg BID	21
Core Period	COMPLETED	AG-348 300 mg BID	22
Core Period	NOT COMPLETED	AG-348 50 mg BID	6
Core Period	NOT COMPLETED	AG-348 300 mg BID	3
Extension Period	STARTED	AG-348 50 mg BID	18
Extension Period	STARTED	AG-348 300 mg BID	18
Extension Period	COMPLETED	AG-348 50 mg BID	0
Extension Period	COMPLETED	AG-348 300 mg BID	0

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events94

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
other	Nausea	Gastrointestinal disorders	AG-348 50 mg BID	10	27
other	Nausea	Gastrointestinal disorders	AG-348 300 mg BID	10	25
other	Vomiting	Gastrointestinal disorders	AG-348 50 mg BID	2	27
other	Vomiting	Gastrointestinal disorders	AG-348 300 mg BID	5	25
other	Diarrhoea	Gastrointestinal disorders	AG-348 50 mg BID	3	27
other	Diarrhoea	Gastrointestinal disorders	AG-348 300 mg BID	3	25
other	Dyspepsia	Gastrointestinal disorders	AG-348 50 mg BID	2	27
other	Dyspepsia	Gastrointestinal disorders	AG-348 300 mg BID	2	25
other	Abdominal pain	Gastrointestinal disorders	AG-348 50 mg BID	0	27
other	Abdominal pain	Gastrointestinal disorders	AG-348 300 mg BID	3	25

1 to 10 of 94Source ClinicalTrials.gov

See all 94 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	AG-348 50 mg BID	0	27
Total, all-cause mortality	AG-348 300 mg BID	0	25
Total, other adverse events	AG-348 50 mg BID	26	27
Total, other adverse events	AG-348 300 mg BID	25	25
Total, serious adverse events	AG-348 50 mg BID	5	27
Total, serious adverse events	AG-348 300 mg BID	3	25

Source ClinicalTrials.gov

Papers3

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
31483964	RESULT	PubMed
31974203	DERIVED	PubMed
36594181	DERIVED	PubMed

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 comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
AG-348 50 mg BID vs AG-348 300 mg BID: deaths from any cause	Trial comparisons
AG-348 50 mg BID vs AG-348 300 mg BID: serious adverse events	Trial comparisons

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT02476916

GET https://conductscience.org/api/trials/v1/NCT02476916/registered?page=2

API reference For AI assistants