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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to approximately 8.5 years

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to approximately 8.5 years

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Percentage of Participants Experiencing at Least One Adverse Event (AEs) in the Core Period

Secondary outcome measures

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

50%

Gastroenteritis

100%

80%

60%

40%

20%

Study treatment Arm

Experimental: AG-348, 5 mg

Experimental: AG-348, 20 mg

Experimental: AG-348, 50 mg

Placebo Comparator: Placebo

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

~290

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

Agios Pharmaceuticals, Inc.Lead Sponsor

52 Previous Clinical Trials

4,154 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many individuals are being assisted by this trial?

"This particular trial has concluded recruitment and is no longer searching for patients. The original posting was on 6/26/2015, with the final update taking place on 11/8/2022. However, if you are interested in other studies, there are 5 trials related to pyruvate metabolism and 9 AG-348 trials that remain active."

Answered by AI

Are there any spaces left in this trial for new test subjects?

"No, according to the listing on clinicaltrials.gov this study is not recruiting patients at this time. The trial was first posted on June 26th, 2015 and ended recruitment on November 8th, 2020. There are, however, 14 other ongoing trials that may be of interest."

Answered by AI

In how many different research facilities is this experiment being conducted today?

"At the moment, there are 8 different sites conducting this trial. They can be found in major cities such as New york, Detroit and Toronto as well as 8 other locations. If you decide to participate in this trial, try and select a location that is close to you to reduce travel time and expenses."

Answered by AI

What other similar trials exist for this disease?

"AG-348 has been studied since 2015, with the first 52-person Phase 1 trial sponsored by Agios Pharmaceuticals, Inc. After this initial study in 2015, AG-348 was approved for Phase 2 testing. Currently, there are 9 ongoing trials involving AG-348 being conducted in 61 cities and 28 countries around the world."

Answered by AI

Has AG-348 met the requirements for federal approval in the United States?

"AG-348 falls into the Phase 2 category, which means that while there is some evidence indicating that it is safe, none supports its efficacy. Our team at Power gave it a safety rating of 2."

Answered by AI

What other medical experiments has AG-348 been used in?

"There are nine ongoing clinical trials for AG-348, six of which are in Phase 3. New york has the most active trials with 339 locations running studies."

Answered by AI

Recent research and studies

New England Journal of MedicineJournal

Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency

Grace, Rachael F., Christian Rose, D. Mark Layton, Frédéric Galactéros, Wilma Barcellini, D. Holmes Morton, Eduard J. van Beers, et al.. 2019. “Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency”. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejmoa1902678.

HaematologicaJournal

AG-348 (mitapivat), an Allosteric Activator of Red Blood Cell Pyruvate Kinase, Increases Enzymatic Activity, Protein Stability, and ATP Levels over a Broad Range of PKLR Genotypes

Rab, Minke A.E., Brigitte A. Van Oirschot, Penelope A. Kosinski, Jeffrey Hixon, Kendall Johnson, Victor Chubukov, Lenny Dang, et al.. 2020. “AG-348 (mitapivat), an Allosteric Activator of Red Blood Cell Pyruvate Kinase, Increases Enzymatic Activity, Protein Stability, and ATP Levels over a Broad Range of PKLR Genotypes”. Haematologica. Ferrata Storti Foundation (Haematologica). doi:10.3324/haematol.2019.238865.

clinicaltrials.govStudy

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

2015. "A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02476916.