← Back to Search

Activator

AG-348 for Hemolytic Anemia

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 from part 1 day 1 to end of part 2, including follow-up (day 197)

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

Study AG348-C-007 was a multicenter study designed to evaluate the efficacy and safety of treatment with AG-348 in a minimum of 20, with up to 40, participants with pyruvate kinase (PK) deficiency, who were regularly receiving blood transfusions. The study was composed of two parts. During Part 1, Dose Optimization Period, participants started on a dose of 5 mg AG-348 administered twice daily. Over the course of Part 1 each participant's dose of AG-348 was sequentially increased to 20 mg twice a day, followed by 50 mg twice a day depending on their tolerance. During Part 2, Fixed-Dose Period, participants received AG-348 at their optimized dose from Part 1.

Eligible Conditions

Hemolytic Anemia
Pyruvate Kinase Deficiency

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from part 1 day 1 to end of part 2, including follow-up (day 197)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from part 1 day 1 to end of part 2, including follow-up (day 197)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from part 1 day 1 to end of part 2, including follow-up (day 197) 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 Achieving a Reduction in Transfusion Burden in Part 2

Secondary study objectives

Blood Transfusion

Hemoglobin, Sickle

+2 more

Other study objectives

Percentage of Participants Experiencing an Adverse Event of Special Interest (AESI)

Side effects data

From 2020 Phase 3 trial • 80 Patients • NCT03548220

67%

Arthralgia

67%

Diarrhoea

67%

Nausea

67%

Nasopharyngitis

33%

Fatigue

33%

Dyspnoea

33%

Headache

33%

Hypertension

33%

Dyspepsia

33%

Musculoskeletal pain

100%

80%

60%

40%

20%

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.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: AG-348Experimental Treatment1 Intervention

Participants received AG-348 tablets, administered orally, at a starting dose of 5 milligrams (mg), twice daily (BID), followed by two sequential dose level increases to 20 mg and 50 mg BID, for a period of 16 weeks in Part 1. This was followed by optimized dose BID, as determined by the investigator in Part 1, for a period of 24 weeks in Part 2.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

AG-348

2014

Completed Phase 3

~380

Do I qualify?Apply below to find out