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Activator

AG-348 for Pyruvate Kinase Deficiency

Phase 2
Waitlist Available
Research Sponsored by Agios Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Males Hb ≤ 12.0 g/dL, females Hb ≤ 11 g/dL
Must have completed 24 weeks of treatment during the Core Period and tolerated AG-348
Must not have
Iron overload sufficiently severe to result in cardiac, hepatic or pancreatic insufficiency
Major surgery in the last 6 months
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to approximately 8.5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new drug, AG-348, to see if it is safe and effective in people with PK deficiency.

Who is the study for?
Adults with Pyruvate Kinase (PK) Deficiency who are not pregnant or breastfeeding, have a hemoglobin level below set thresholds, and haven't had recent blood transfusions can join. They must be in stable health, not on certain medications, and agree to use contraception. Those with psychiatric disorders or other conditions that could affect study participation are excluded.
What is being tested?
The trial is testing the safety and effectiveness of AG-348 (mitapivat) at various doses for treating PK deficiency. It's a multicenter study where participants receive different amounts of this medication to see how well it works and what side effects occur.
What are the potential side effects?
Specific side effects of AG-348 aren't listed here, but generally such trials monitor for any adverse reactions including digestive issues, allergic responses, changes in liver or kidney function, blood-related problems, fatigue or potential interactions with other drugs.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My hemoglobin level is low, below 12 g/dL if I'm male, or below 11 g/dL if I'm female.
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I completed 24 weeks of treatment with AG-348 and tolerated it well.
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I have had 3 or fewer blood transfusions in the last year and none in the past 4 months.
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I can take care of myself and am up and about more than half of my waking hours.
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I have been diagnosed with PK deficiency.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have iron overload affecting my heart, liver, or pancreas.
Select...
I have not had major surgery in the past 6 months.
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I have another blood disorder besides my current condition.
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I have symptoms of gallstones or gallbladder inflammation.
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I have had a bone marrow or stem cell transplant.
Select...
My hemoglobin level is above 12 g/dL if I am male, or above 11 g/dL if I am female.
Select...
I have heart rhythm problems or take medication that affects my heart's rhythm.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to approximately 8.5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to approximately 8.5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Percentage of Participants Experiencing at Least One Adverse Event (AEs) in the Core Period
Secondary study objectives
Apparent Clearance at Steady-State (Clss/F) for AG-348 and Its Metabolite AGI-8702
Area Under the Concentration-time Curve From Time Zero to the Last Non-zero Concentration (AUC0-t) for AG-348 and Its Metabolite AGI-8702
Change From Baseline in Carbon Monoxide Over the Duration of the Extension Period
+28 more

Side effects data

From 2020 Phase 3 trial • 80 Patients • NCT03548220
67%
Arthralgia
67%
Diarrhoea
67%
Nausea
67%
Nasopharyngitis
33%
Fatigue
33%
Headache
33%
Dyspnoea
33%
Hypertension
33%
Dyspepsia
33%
Musculoskeletal pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Experimental: AG-348, 20 mg
Experimental: AG-348, 5 mg
Experimental: AG-348, 50 mg
Placebo Comparator: Placebo

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups
Experimental Treatment
Group I: AG-348 50 mg BIDExperimental Treatment1 Intervention
Participants with PK deficiency received AG-348, 50 milligrams (mg), as initial dose, twice daily (BID) for the Core Period (Week 24).
Group II: AG-348 300 mg BIDExperimental Treatment1 Intervention
Participants with PK deficiency received AG-348, 300 mg, as initial dose, BID for the Core Period (Week 24).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AG-348
2014
Completed Phase 3
~380

Find a Location

Who is running the clinical trial?

Agios Pharmaceuticals, Inc.Lead Sponsor
54 Previous Clinical Trials
4,131 Total Patients Enrolled
9 Trials studying Pyruvate Kinase Deficiency
1,085 Patients Enrolled for Pyruvate Kinase Deficiency

Media Library

AG-348 (Activator) Clinical Trial Eligibility Overview. Trial Name: NCT02476916 — Phase 2
Pyruvate Kinase Deficiency Research Study Groups: AG-348 50 mg BID, AG-348 300 mg BID
Pyruvate Kinase Deficiency Clinical Trial 2023: AG-348 Highlights & Side Effects. Trial Name: NCT02476916 — Phase 2
AG-348 (Activator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02476916 — Phase 2
~5 spots leftby Jan 2026
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