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AG-946 for Sickle Cell Anemia

Phase 1
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
Participant is a healthy male or a female of nonchildbearing potential aged 18 to 55 years inclusive at the time of consent;
Participant has a bodyweight ≥50 kilograms (kg) and body mass index ≥18.5 and ≤32 kilograms per square meter (kg/m^2) at screening;
Must not have
Used acetaminophen greater than 2 g in any 24-hour period up to 48 hours before first dose
Had surgery within 6 months before first dose
Timeline
Screening 3 weeks
Treatment Varies
Follow Up sad: up to day 8; mad: up to day 49; scd: up to day 56

Summary

This trial tests a new oral drug, AG-946, for safety and effectiveness. It involves healthy volunteers and people with sickle cell disease. Researchers will measure how the body processes the drug and its effects.

Who is the study for?
This trial is for healthy adults aged 18-55 and those with sickle cell disease (SCD) aged 18-70. Participants must be non-smokers, in good health, understand the study, and agree to use contraception. Those with SCD should have stable medication doses and adequate organ function. Exclusions include recent surgery, certain medical conditions or drug use that could affect results.
What is being tested?
AG-946 is being tested for safety and effectiveness in both healthy volunteers through single/multiple dose trials and participants with SCD in an open-label part of the study. The trial will explore how AG-946 behaves inside the body (pharmacokinetics) and its impact on the disease (pharmacodynamics).
What are the potential side effects?
While specific side effects are not listed here, common ones may include digestive issues, headaches, fatigue or allergic reactions. Side effects can vary based on individual responses to AG-946.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am a healthy adult between 18 and 55 years old and cannot become pregnant.
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I weigh at least 50 kg and my BMI is between 18.5 and 32.
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I am in good health with normal medical exams and tests.
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I agree to either abstain from sex or use contraception for 90 days after my last treatment dose.
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I am between 18 and 55 years old and cannot have children.
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I understand the study and can give my consent.
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I weigh at least 50 kg and my BMI is between 18.5 and 32.
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I agree not to donate sperm during and for 3 months after the study.
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I have been diagnosed with sickle cell anemia.
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I must take at least 0.8 mg of folic acid daily for the study.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have taken more than 2 grams of acetaminophen in a day within the last 2 days.
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I had surgery less than 6 months ago.
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I have no conditions affecting my stomach, liver, kidneys, or had my gallbladder removed that could affect medication absorption or removal.
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I haven't donated blood, lost more than 500 mL of blood, or received a blood transfusion in the last 3 months.
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I haven't taken prescription drugs within 2 weeks or 5 half-lives before starting.
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I have difficulty communicating with my doctor.
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I have a known G6PD deficiency.
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I am a candidate for a bone marrow transplant that could potentially cure my SCD, but participating in this study would delay or stop me from receiving it.
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I have a confirmed diagnosis of pyruvate kinase deficiency.
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I've had over 6 pain crises due to sickle cell disease that needed hospital care in the last year.
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I regularly get or got a blood transfusion in the last 3 months.
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I am currently on treatment to improve oxygen delivery by my red blood cells.
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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 haven't taken strong P-gp inhibitors recently.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~sad: up to day 8; mad: up to day 49; scd: up to day 56
This trial's timeline: 3 weeks for screening, Varies for treatment, and sad: up to day 8; mad: up to day 49; scd: up to day 56 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Area Under the Plasma Concentration-Time Curve from Time Zero to Last Quantifiable Concentration (AUC0-t), Time Curve from Time Zero to Infinity (AUC0-∞), and Time Curve from Time Zero to Time tau Over the Dosing Interval (AUC0-τ) for AG-946
Maximum Observed Concentration (Cmax) of AG-946
SAD: Apparent Clearance (CL/F) for AG-946
+2 more

Trial Design

3Treatment groups
Experimental Treatment
Group I: Part 3: Sickle Cell Disease (SCD) PhaseExperimental Treatment1 Intervention
Participants will receive a range of selected ascending doses of AG-946, orally, QD or using an alternative dosing regimen for 28 days.
Group II: Part 2: Multiple Ascending Dose (MAD) PhaseExperimental Treatment2 Interventions
Participants will receive a range of doses of AG-946 or placebo, orally, once daily (QD) for 14 days or using an alternative dosing regimen for up to 28 days under fasted conditions.
Group III: Part 1: Single Ascending Dose (SAD) PhaseExperimental Treatment2 Interventions
Participants will receive a range of doses of AG-946 or placebo, orally, once on Day 1. AG-946 will be given under fasted or fed conditions.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AG-946
2020
Completed Phase 1
~130
AG-946 Matched Placebo
2020
Completed Phase 1
~130

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Common treatments in healthy subjects often involve drugs that modulate metabolic pathways, enzyme activity, or receptor interactions to understand their pharmacokinetics and pharmacodynamics. For example, AG-946 is being studied to assess its safety and tolerability, which involves understanding how the drug is absorbed, distributed, metabolized, and excreted in the body. This is crucial for determining the appropriate dosage and identifying any potential side effects, ensuring that the drug is safe and effective before being administered to patients with specific conditions like sickle cell disease.
Primary prevention of stroke in children with sickle cell anemia in sub-Saharan Africa: rationale and design of phase III randomized clinical trial.Antibiotics for treating acute chest syndrome in people with sickle cell disease.Fluid replacement therapy for acute episodes of pain in people with sickle cell disease.

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Logistics

Participation is compensated

You will be compensated for participating in this trial.

Who is running the clinical trial?

Agios Pharmaceuticals, Inc.Lead Sponsor
54 Previous Clinical Trials
4,061 Total Patients Enrolled

Media Library

AG-946 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT04536792 — Phase 1
Sickle Cell Disease Research Study Groups: Part 2: Multiple Ascending Dose (MAD) Phase, Part 3: Sickle Cell Disease (SCD) Phase, Part 1: Single Ascending Dose (SAD) Phase
Sickle Cell Disease Clinical Trial 2023: AG-946 Highlights & Side Effects. Trial Name: NCT04536792 — Phase 1
AG-946 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04536792 — Phase 1
Sickle Cell Disease Patient Testimony for trial: Trial Name: NCT04536792 — Phase 1
~23 spots leftby Dec 2025