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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

Must not have

Prior exposure to sotatercept (ACE-011), luspatercept (ACE-536), ruxolitinib, or gene therapy

Currently receiving medications that are strong inhibitors of cytochrome P450 (CYP)3A4, strong inducers of CYP3A4, strong inhibitors of P-glycoprotein (P-gp), or digoxin (a P-gp sensitive substrate medication) that have not been stopped for a duration of at least 5 days or a timeframe equivalent to 5 half-lives (whichever is longer) prior to the first day of study drug

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from signing the inform consent form up to data cut-off date: 20 august 2020 (up to approximately 19 months)

Awards & highlights

No Placebo-Only Group

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.

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.

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

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

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

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I have a type of thalassemia.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have previously been treated with sotatercept, luspatercept, ruxolitinib, or gene therapy.

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I am not currently on strong medication that affects liver enzymes or certain heart meds.

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I am not allergic to AG-348 or its ingredients.

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I have not had major surgery in the last 6 months.

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I have had a bone marrow or stem cell transplant.

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I have been diagnosed with Hb S or Hb C thalassemia.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from signing the inform consent form up to data cut-off date: 20 august 2020 (up to approximately 19 months)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from signing the inform consent form up to data cut-off date: 20 august 2020 (up to approximately 19 months)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from signing the inform consent form up to data cut-off date: 20 august 2020 (up to approximately 19 months) 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 Hemoglobin Response (HR)

Secondary study objectives

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

Percentage of Participants Achieving a Delayed Hb Response

Hemoglobin, Sickle

+1 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%

Hypertension

33%

Dyspnoea

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.

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

~380

0 / 9Eligibility criteria met