← Back to Search

Activator

AG-348 for Pyruvate Kinase Deficiency

Phase 3

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

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 12: pre-dose, post-dose at 30 minutes, 1 h, 2 h, 4 h, 8 h

Awards & highlights

Study Summary

This trial is testing a drug to see if it is safe and effective long-term for people who have already taken it.

Who is the study for?

Adults with Pyruvate Kinase Deficiency who've been in previous AG-348 studies can join. They must use effective contraception, have a negative pregnancy test if applicable, and be able to follow the study plan. Those with new significant medical conditions or on certain drugs are excluded.Check my eligibility

What is being tested?

The trial is testing the long-term effects of AG-348 for treating Pyruvate Kinase Deficiency. It's an extension of earlier trials (AG348-C-006/007), focusing on safety, tolerability, and efficacy over a longer period.See study design

What are the potential side effects?

While specific side effects aren't listed here, participants will monitor for any adverse reactions due to extended use of AG-348 as it relates to their condition and overall health.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 12: pre-dose, post-dose at 30 minutes, 1 h, 2 h, 4 h, 8 h

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 12: pre-dose, post-dose at 30 minutes, 1 h, 2 h, 4 h, 8 h

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 12: pre-dose, post-dose at 30 minutes, 1 h, 2 h, 4 h, 8 h for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Number of Participants with AEs Leading to Dose Reduction, Treatment Interruption and Treatment Discontinuation

Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

Secondary outcome measures

Area Under the Concentration-Time Curve (AUC) of AG-348 in Participants Who Previously Received Placebo in Study AG348-C-006

Change from Baseline in Bilirubin

Change from Baseline in HRQoL PRO Scores: Pyruvate Kinase Deficiency Impact Assessment (PKDIA)

+9 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

3Treatment groups

Experimental Treatment

Group I: Cohort 3Experimental Treatment1 Intervention

Participants who received AG-348 in Study AG348-C-007 will enroll in Cohort 3. Participants will continue the AG-348 dose regimen they were receiving at the last visit of Study AG348-C-007.

Group II: Cohort 2Experimental Treatment1 Intervention

Participants who received AG-348 in Study AG348-C-006 will enroll in Cohort 2. Participants will continue the AG-348 dose regimen they were receiving at the last visit of Study AG348-C-006.

Group III: Cohort 1Experimental Treatment1 Intervention

Participants who received placebo in Study AG348-C-006 will enroll in Cohort 1. Part 1 (Dose Optimization Period, 12 weeks): Participants will begin by receiving 5 milligrams (mg) orally, twice a day. Each participant's dose of AG-348 may be increased to 20 mg twice a day and then to 50 mg twice a day depending on their response to AG-348 and tolerability. Part 2 (Fixed Dose Period, 12 weeks): Last dose received in Part 1, twice a day. After completion of Part 2, participants who, in the opinion of the Investigator, have demonstrated clinical benefit from AG-348 treatment will continue AG-348 treatment in the Continued Treatment Period.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

AG-348

2014

Completed Phase 3

~290

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Agios Pharmaceuticals, Inc.Lead Sponsor

52 Previous Clinical Trials

4,116 Total Patients Enrolled

9 Trials studying Pyruvate Kinase Deficiency

1,047 Patients Enrolled for Pyruvate Kinase Deficiency

Medical AffairsStudy ChairAgios Pharmaceuticals, Inc.

39 Previous Clinical Trials

8,470 Total Patients Enrolled

4 Trials studying Pyruvate Kinase Deficiency

135 Patients Enrolled for Pyruvate Kinase Deficiency

Media Library

AG-348 (Activator) Clinical Trial Eligibility Overview. Trial Name: NCT03853798 — Phase 3

Pyruvate Kinase Deficiency Research Study Groups: Cohort 1, Cohort 2, Cohort 3

Pyruvate Kinase Deficiency Clinical Trial 2023: AG-348 Highlights & Side Effects. Trial Name: NCT03853798 — Phase 3

AG-348 (Activator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03853798 — Phase 3

Frequently Asked Questions

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

Do AG-348's results stand up to scientific scrutiny?

"There are a total of 9 studies being conducted on AG-348 at the moment. Of those, 6 are in Phase 3 clinical trials. Most of the research is centralized in Ribeirão Preto and Massachusetts; however, there are 330 medical sites running AG-348 trials in total."

Answered by AI

Is this a current study that is looking for new participants?

"This information can be found on clinicaltrials.gov. The trial in question is not presently recruiting participants. The study was initially posted on 3/21/2019 and was last updated on 10/28/2022. Although this clinical trial is not recruiting patients anymore, there are 14 other trials actively recruiting patients at this time."

Answered by AI

What makes this trial unique in comparison to others?

"AG-348 has undergone 9 trials in 54 cities and 28 countries since 2015. The first trial, which had 52 patients and was sponsored by Agios Pharmaceuticals, Inc., completed its Phase 2 drug approval stage. Subsequently, 12 more studies have been conducted."

Answered by AI