← Back to Search

Activator

AG-348 for Thalassemia

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

Hb concentration ≤10.0 grams per deciliter (g/dL), regardless of sex, based on an average of at least 2 Hb measurements (separated by a minimum of 7 days) during the screening period

Considered non-transfusion-dependent, defined as having no more than 5 units of red blood cells (RBCs) transfused during the 24-week period up to the first day of study drug and no RBC transfusions in the 8 weeks prior to the first day of study drug

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to approximately 10.5 years

Awards & highlights

Study Summary

This trial is testing a new drug, AG-348, for adults with non-transfusion-dependent thalassemia, a genetic blood disorder. The trial includes a core period of 24 weeks followed by an extension period of up to 10 years for eligible participants.

Who is the study for?

Adults with non-transfusion-dependent thalassemia (NTDT) who haven't had more than 5 blood transfusions in the last 24 weeks and none in the past 8 weeks. Participants must have a documented history of thalassemia, adequate organ function, and agree to use two forms of contraception if applicable. Exclusions include prior treatments like gene therapy or certain drugs, recent major surgery, or serious allergies.Check my eligibility

What is being tested?

The trial is testing AG-348's effectiveness for NTDT over up to 10 years. Starting with a dose of 50 mg twice daily, which may increase to 100 mg based on safety and hemoglobin levels after six weeks. The study aims to understand how well AG-348 works and its effects on the body.See study design

What are the potential side effects?

While specific side effects are not listed here, common ones associated with new medications can include digestive issues, headaches, fatigue, potential allergic reactions or skin rashes. Organ function will be monitored due to possible impacts from long-term medication use.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My average hemoglobin level is 10 g/dL or less, based on two tests.

Select...

I haven't needed more than 5 blood transfusions in the last 6 months and none in the last 2 months.

Select...

I have a type of thalassemia.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to approximately 10.5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to approximately 10.5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to approximately 10.5 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 Achieving a Hemoglobin Response (HR)

Secondary outcome measures

AUC0-8h: Area Under the Plasma Concentration-time Curve From Time 0 to 8 Hours of AG-348

AUC0-t: Area Under the Plasma Concentration-time Curve From Time 0 to Time of the Last Quantifiable Concentration of AG-348

Average Change From Baseline in Hb Concentrations From Week 12 to Week 24

+17 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

1Treatment groups

Experimental Treatment

Group I: AG-348Experimental Treatment1 Intervention

Participants with alpha or beta thalassemia received AG-348 50 mg twice daily (BID), orally up to Week 6. Following Week 6, depending on the participants' safety and hemoglobin (Hb) concentrations, they could undergo one potential dose-level increase from 50 to 100 mg BID. After completion of the Core Period of 24 weeks, participants were eligible to continue to receive AG-348 in the Extension Period which is up to 10 years.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

AG-348

2014

Completed Phase 3

~290

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Agios Pharmaceuticals, Inc.Lead Sponsor

52 Previous Clinical Trials

4,186 Total Patients Enrolled

2 Trials studying Thalassemia

452 Patients Enrolled for Thalassemia

Medical AffairsStudy ChairAgios Pharmaceuticals, Inc.

39 Previous Clinical Trials

8,540 Total Patients Enrolled

2 Trials studying Thalassemia

452 Patients Enrolled for Thalassemia

Media Library

AG-348 (Activator) Clinical Trial Eligibility Overview. Trial Name: NCT03692052 — Phase 2

Thalassemia Research Study Groups: AG-348

Thalassemia Clinical Trial 2023: AG-348 Highlights & Side Effects. Trial Name: NCT03692052 — Phase 2

AG-348 (Activator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03692052 — Phase 2

Frequently Asked Questions

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

Are we still enrolling individuals for this research project?

"This study has completed patient recruitment and is no longer enrolling new participants. If you are interested in other clinical trials, there are 61 thalassemia studies and 9 AG-348 trials that are still looking for patients."

Answered by AI

How many people total will be in this experiment?

"This study has completed recruitment for participants. The trial was first posted on December 28th, 2018 and was last edited on July 21st, 2022. For individuals interested in other studies, 61 trials for thalassemia and 9 trials AG-348 are currently open."

Answered by AI

What is the uniqueness of this clinical trial?

"AG-348 has had a long journey from its first clinical trial in 2015 to the present day, where there are 9 different ongoing trials involving the drug. The original study was conducted by Agios Pharmaceuticals, Inc. and 52 patients were enrolled. AG-348 received Phase 2 approval after this initial success. Now, the medication is being tested in 61 cities across 28 countries."

Answered by AI

What other tests has AG-348 undergone in the past?

"AG-348 was first pioneered in 2015 by researchers at Stanford University. So far, there have been 12 completed clinical trials with 9 more active ones. A considerable amount of these ongoing studies are based in Toronto, Canada."

Answered by AI

Has AG-348 undergone the FDA's regulatory process?

"While there is some evidence to support AG-348's safety, as this drug is still in Phase 2 of testing, the lack of data with regards to efficacy rates warrants a score of 2."

Answered by AI

Recent research and studies

The LancetJournal

Safety and Efficacy of Mitapivat, an Oral Pyruvate Kinase Activator, in Adults with Non-transfusion Dependent Α-thalassaemia or Β-thalassaemia: An Open-label, Multicentre, Phase 2 Study

Kuo, Kevin H M, D Mark Layton, Ashutosh Lal, Hanny Al-Samkari, Joy Bhatia, Penelope A Kosinski, Bo Tong, Megan Lynch, Katrin Uhlig, and Elliott P Vichinsky. 2022. “Safety and Efficacy of Mitapivat, an Oral Pyruvate Kinase Activator, in Adults with Non-transfusion Dependent Α-thalassaemia or Β-thalassaemia: An Open-label, Multicentre, Phase 2 Study”. The Lancet. Elsevier BV. doi:10.1016/s0140-6736(22)01337-x.

clinicaltrials.govStudy

A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia

2019. "A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03692052.

All > Thalassemia